19. Lysosomal storage disease Disease details / Clinical trials / Drug dev / DR info
Clinical trials : 899 / Drugs : 684 - (DrugBank : 99) / Drug target genes : 51 - Drug target pathways : 182
Drugs and their primary sponsors and trial info
045251016
Recordati Rare Diseases
2020 Phase 3 EUCTR2018-002984-24-IT Belgium;France;Germany;Italy;United Kingdom;
1- Deoxygalactonojirimycin
Amicus Therapeutics
2005 Phase 2 EUCTR2005-004384-33-GB United Kingdom;
1-deoxygalactonojirimycin hydrochloride
AMICUS THERAPEUTICS, INC
2011 - EUCTR2010-022636-37-IT Argentina;Australia;Austria;Belgium;Brazil;Denmark;Germany;Greece;Italy;Japan;Mexico;Russian Federation;Switzerland;Taiwan;Turkey;United Kingdom;United States;
Amicus Therapeutics, Inc.
2012 - EUCTR2010-022709-16-GB Belgium;United Kingdom;
2012 - EUCTR2010-022636-37-DE Argentina;Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Hungary;Israel;Italy;Japan;Mexico;Poland;Russian Federation;Slovakia;Switzerland;Taiwan;Turkey;United Kingdom;United States;
2011 Phase 3 EUCTR2010-022636-37-GR Argentina;Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Hungary;Israel;Italy;Japan;Mexico;Poland;Russian Federation;Slovakia;Switzerland;Taiwan;Turkey;United Kingdom;United States;
2011 Phase 2 EUCTR2010-022709-16-BE Australia;Belgium;Canada;France;Netherlands;Switzerland;United Kingdom;
2011 - EUCTR2010-022636-37-GB Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;United Kingdom;United States;
2011 - EUCTR2010-022636-37-DK Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;United Kingdom;United States;
2011 - EUCTR2010-022636-37-BE Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;United Kingdom;United States;
2011 - EUCTR2010-022636-37-AT Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;United Kingdom;United States;
2010 Phase 3 EUCTR2009-013459-31-ES Belgium;Denmark;France;Germany;Italy;Netherlands;Spain;United Kingdom;
2010 Phase 3 EUCTR2009-013459-31-BE Belgium;Denmark;France;Germany;Italy;Netherlands;Spain;United Kingdom;
2010 - EUCTR2009-013459-31-NL Belgium;Denmark;France;Germany;Italy;Netherlands;Spain;United Kingdom;
2010 - EUCTR2009-013459-31-DK Argentina;Australia;Belgium;Brazil;Canada;Denmark;Egypt;France;Germany;Italy;Netherlands;South Africa;Spain;Turkey;United Kingdom;United States;
2009 Phase 3 EUCTR2009-013459-31-GB Argentina;Australia;Belgium;Brazil;Canada;Denmark;Egypt;France;Germany;Italy;Netherlands;South Africa;Spain;Turkey;United Kingdom;United States;
2009 Phase 3 EUCTR2009-013459-31-FR Belgium;Denmark;France;Germany;Italy;Netherlands;Spain;United Kingdom;
2009 Phase 2 EUCTR2007-001838-13-GB Australia;Brazil;United Kingdom;United States;
2006 Phase 2 EUCTR2006-000181-36-GB United Kingdom;
- - EUCTR2009-013459-31-DE Belgium;Denmark;France;Germany;Italy;Netherlands;Spain;United Kingdom;
12 lead electrocardiogram
Baylor Research Institute
2018 - NCT04002531 United States;
12S-IT
Shire HGT Inc
2012 Phase 1;Phase 2 EUCTR2011-000212-25-GB Canada;United Kingdom;United States;
15 O Water
National Institute of Neurological Disorders and Stroke (NINDS)
1994 - NCT00001972 United States;
2(S)-(ACETYLAMINO)-4-METHYLPENTANOIC ACID
IntraBio Ltd
2022 Phase 3 EUCTR2021-005356-10-SK Australia;Czechia;Germany;Netherlands;Slovakia;Switzerland;United Kingdom;United States;
2022 Phase 3 EUCTR2021-005356-10-DE Australia;Czechia;Germany;Netherlands;Slovakia;Spain;Switzerland;United Kingdom;United States;
2019 Phase 2 EUCTR2018-004406-25-ES Germany;Spain;United Kingdom;United States;
2019 Phase 2 EUCTR2018-004406-25-DE Germany;Spain;United Kingdom;United States;
2019 Phase 2 EUCTR2018-004331-71-SK Germany;Slovakia;Spain;United Kingdom;United States;
2019 Phase 2 EUCTR2018-004331-71-ES Germany;Slovakia;Spain;United Kingdom;United States;
2019 Phase 2 EUCTR2018-004331-71-DE Germany;Slovakia;Spain;United Kingdom;United States;
- Phase 3 EUCTR2021-005356-10-NL Australia;Czechia;Germany;Netherlands;Slovakia;Spain;Switzerland;United Kingdom;United States;
2-HYDROXYPROPYL-BETA-CYCLODEXTRIN
Jichi Medical University, Saitama Medical Center
2014 - JPRN-UMIN000020032 Japan;
Nara Medical University
2016 - JPRN-UMIN000023517 Japan;
2014 - JPRN-UMIN000023094 Japan;
2-hour Holter Monitor
Baylor Research Institute
2018 - NCT04002531 United States;
4D-310
4D Molecular Therapeutics
2020 Phase 1/Phase 2 NCT04519749 United States;
500 0500 0
Manchester University NHS Foundation Trust
2014 Phase 3 EUCTR2013-001479-18-GB United Kingdom;
89 Zirconium Recombinant Human Alpha-N-Acetylglucosaminidase (rhNAGLU) (radiolabeled)
Alexion Pharmaceuticals, Inc.
2016 Phase 2 EUCTR2015-000753-20-NL Netherlands;
89Zr-SBC-103
Alexion Pharmaceuticals, Inc.
2016 Phase 2 EUCTR2015-000753-20-NL Netherlands;
A (migalastat)
Amicus Therapeutics
2011 Phase 1 NCT01489995 United States;
A16AB04
FONDAZIONE POLICLINICO UNIVERSITARIO AGOSTINO GEMELLI IRCCS UNIVERSITA' CATTOLICA DEL SACRO CUORE
2018 Phase 4 EUCTR2018-000368-27-IT Italy;
A16AX06
Orphazyme ApS
2015 Phase 1 EUCTR2014-005194-37-ES Germany;Spain;Switzerland;
2015 Phase 1 EUCTR2014-005194-37-DE Germany;Spain;Switzerland;
AAV2/8.TBG.hARSB
FONDAZIONE TELETHON
2018 Phase 1 EUCTR2016-002328-10-NL Italy;Netherlands;Turkey;
2017 Phase 1 EUCTR2016-002328-10-IT Italy;Netherlands;Turkey;
Fondazione Telethon
2017 Phase 1/Phase 2 NCT03173521 Italy;Netherlands;Turkey;
AAV2CUhCLN2 (3x10^12 particle units)
Weill Medical College of Cornell University
2004 Phase 1 NCT00151216 United States;
AAV9-CAG-coh-SGSH
Laboratorios del Dr. Esteve, S.A.
2016 Phase 1;Phase 2 EUCTR2015-000359-26-ES Spain;
AAV9-GLB1
National Human Genome Research Institute (NHGRI)
2019 Phase 1/Phase 2 NCT03952637 United States;
AAVrh.10-hMPSIIIA
SANFILIPPO Therapeutics SAS
- - EUCTR2010-019962-10-FR France;
AAVrh.10CUCLN2
Weill Medical College of Cornell University
2010 Phase 1/Phase 2 NCT01414985 United States;
AAVrh.10CUhCLN2 vector 2.85x10^11 genome copies
Weill Medical College of Cornell University
2010 Phase 1 NCT01161576 United States;
AAVrh.10CUhCLN2 vector 9.0x10^11 genome copies
Weill Medical College of Cornell University
2010 Phase 1 NCT01161576 United States;
AAVrh.10cuARSA
Inserm
2012 - EUCTR2011-004410-42-FR France;
ABO-101
Abeona Therapeutics Europe SL.
2021 Phase 1;Phase 2 EUCTR2019-002936-97-FR France;Germany;United States;
2019 Phase 1;Phase 2 EUCTR2014-001411-39-GB France;Germany;Italy;Netherlands;Spain;United Kingdom;United States;
2019 Phase 1;Phase 2 EUCTR2014-001411-39-DE France;Germany;Spain;United Kingdom;United States;
- Phase 1;Phase 2 EUCTR2019-002936-97-DE France;Germany;Spain;United States;
Abeona Therapeutics, Inc
2020 - NCT04655911 France;Germany;United States;
ABO-102
Abeona Therapeutics Inc
2020 Phase 1;Phase 2 EUCTR2019-002979-34-ES Australia;France;Germany;Spain;United States;
2019 Phase 1;Phase 2 EUCTR2018-000504-42-ES Australia;Spain;United States;
2019 Phase 1;Phase 2 EUCTR2015-003904-21-FR Australia;Brazil;France;Germany;Spain;United Kingdom;United States;
Abeona Therapeutics, Inc
2020 - NCT04360265 Australia;Spain;United States;
2019 Phase 1/Phase 2 NCT04088734 Australia;Spain;United States;
2016 Phase 1/Phase 2 NCT02716246 Australia;France;Germany;Spain;United States;
ACT-434964
IDORSIA PHARMACEUTICALS LTD
2020 Phase 3 EUCTR2018-002210-12-IT Australia;Austria;Belgium;Canada;France;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;United Kingdom;United States;
2020 Phase 3 EUCTR2017-003369-85-IT Australia;Austria;Belgium;Canada;Finland;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;Switzerland;United Kingdom;United States;
Idorsia Pharmaceuticals Japan Ltd
2019 Phase 3 JPRN-JapicCTI-194731 Japan;
Idorsia Pharmaceuticals Ltd
2021 Phase 3 EUCTR2018-002210-12-NO Australia;Austria;Belgium;Canada;France;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;United Kingdom;United States;
2021 Phase 3 EUCTR2018-002210-12-FR Austria;Belgium;Canada;Czech Republic;France;Germany;Netherlands;Poland;United Kingdom;United States;
2020 Phase 3 EUCTR2018-002210-12-BE Australia;Austria;Belgium;Canada;France;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;United Kingdom;United States;
2020 Phase 3 EUCTR2017-003369-85-NO Australia;Austria;Belgium;Canada;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;Switzerland;United Kingdom;United States;
2020 Phase 3 EUCTR2017-003369-85-IE Australia;Austria;Belgium;Canada;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;Switzerland;United Kingdom;United States;
2020 Phase 3 EUCTR2017-003369-85-ES Australia;Austria;Belgium;Canada;Germany;Ireland;Italy;Netherlands;Norway;Poland;Russian Federation;Spain;Switzerland;United Kingdom;United States;
2019 Phase 3 EUCTR2018-002210-12-NL Australia;Austria;Belgium;Canada;France;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;United Kingdom;United States;
2019 Phase 3 EUCTR2018-002210-12-GB Australia;Austria;Belgium;Canada;Germany;Ireland;Netherlands;Poland;United Kingdom;United States;
2019 Phase 3 EUCTR2018-002210-12-DE Australia;Austria;Belgium;Canada;France;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;United Kingdom;United States;
2019 Phase 3 EUCTR2018-002210-12-AT Australia;Austria;Belgium;Canada;France;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;United Kingdom;United States;
2018 Phase 3 EUCTR2017-003369-85-NL Australia;Austria;Belgium;Canada;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;Switzerland;United Kingdom;United States;
2018 Phase 3 EUCTR2017-003369-85-GB Australia;Austria;Belgium;Canada;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;Switzerland;United Kingdom;United States;
2018 Phase 3 EUCTR2017-003369-85-AT Australia;Austria;Belgium;Canada;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;Switzerland;United Kingdom;United States;
ADENO-ASSOCIATED VIRAL (AAV) SEROTYPE 8 (AAV2/8) VECTOR WITH LIVER-SPECIFIC THYROXINE-BINDING GLOBULIN (TBG) PROMOTER, DRIVING THE EXPRESSION OF THE HUMAN ARSB GENE
FONDAZIONE TELETHON
2018 Phase 1 EUCTR2016-002328-10-NL Italy;Netherlands;Turkey;
ADMINISTRATION
Osaka City University HospitalTakashi Hamazaki
2019 Phase 2 JPRN-JMA-IIA00416 Japan;
Tottori University Hospital, Departrment of Child Neurology
2019 Phase 2-3 JPRN-JMA-IIA00421 Japan;
AGALSIDASI ALFA
SANOFI-AVENTIS RECHERCHE E DEVELOPPEMENT
2022 Phase 3 EUCTR2021-002320-20-IT Austria;Canada;China;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Turkey;United Kingdom;United States;
AGALSIDASI BETA
FONDAZIONE POLICLINICO UNIVERSITARIO AGOSTINO GEMELLI IRCCS UNIVERSITA' CATTOLICA DEL SACRO CUORE
2018 Phase 4 EUCTR2018-000368-27-IT Italy;
SANOFI-AVENTIS RECHERCHE E DEVELOPPEMENT
2022 Phase 3 EUCTR2021-002320-20-IT Austria;Canada;China;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Turkey;United Kingdom;United States;
AGT-181
ArmaGen, Inc
2016 Phase 1/Phase 2 NCT03071341 Brazil;
2015 Phase 1/Phase 2 NCT03053089 Brazil;
2015 Phase 1 NCT02597114 -
AGT-181 (HIRMAb-IDUA)
ArmaGen, Inc
2015 Phase 1 NCT02371226 United States;
AGT-182
ArmaGen, Inc
2015 Phase 1 NCT02262338 Germany;Netherlands;Philippines;United States;
ALDURAZYME*INFUS 1FL 5ML 500U
Genzyme Europe BV
2010 - EUCTR2007-007003-33-IT Italy;
ARIMOCLOMOL CITRATE
Orphazyme A/S
2016 Phase 2;Phase 3 EUCTR2015-004438-93-GB Denmark;Germany;Italy;Poland;Spain;Switzerland;United Kingdom;United States;
AT-GTX-501
Amicus Therapeutics, Inc.
2021 Phase 1;Phase 2 EUCTR2021-000706-21-DE Germany;United States;
AT1001
AMICUS THERAPEUTICS, INC
2011 - EUCTR2010-022636-37-IT Argentina;Australia;Austria;Belgium;Brazil;Denmark;Germany;Greece;Italy;Japan;Mexico;Russian Federation;Switzerland;Taiwan;Turkey;United Kingdom;United States;
Amicus Therapeutics
2011 Phase 1 NCT01730469 United States;
2005 Phase 2 EUCTR2005-004384-33-GB United Kingdom;
Amicus Therapeutics UK, Ltd
2020 Phase 3 EUCTR2017-000146-21-IT Australia;Germany;Italy;Spain;United Kingdom;United States;
Amicus Therapeutics, Inc.
2015 Phase 3 EUCTR2014-002701-38-GB Argentina;Australia;Austria;Belgium;Brazil;Canada;Denmark;Egypt;France;Italy;Japan;Spain;Turkey;United Kingdom;United States;
2015 Phase 3 EUCTR2014-002701-38-ES Argentina;Australia;Austria;Belgium;Brazil;Canada;Denmark;Egypt;France;Italy;Japan;Spain;Turkey;United Kingdom;United States;
2015 Phase 3 EUCTR2014-002701-38-DK Argentina;Australia;Austria;Belgium;Brazil;Canada;Denmark;Egypt;France;Italy;Japan;Spain;Turkey;United Kingdom;United States;
2015 Phase 3 EUCTR2014-002701-38-BE Argentina;Australia;Austria;Belgium;Brazil;Canada;Denmark;Egypt;France;Italy;Japan;Spain;Turkey;United Kingdom;United States;
2014 Phase 3 EUCTR2014-002701-38-AT Argentina;Australia;Austria;Belgium;Brazil;Canada;Denmark;Egypt;France;Italy;Japan;Spain;Turkey;United Kingdom;United States;
2014 Phase 3 EUCTR2011-004800-40-AT Argentina;Australia;Austria;Belgium;Brazil;Canada;Denmark;Egypt;France;Italy;Spain;Turkey;United Kingdom;United States;
2013 Phase 3 EUCTR2011-004800-40-BE Argentina;Australia;Austria;Belgium;Brazil;Canada;Denmark;Egypt;France;Italy;Spain;Turkey;United Kingdom;United States;
2012 Phase 3 EUCTR2011-004800-40-GB Argentina;Australia;Austria;Belgium;Brazil;Canada;Denmark;Egypt;France;Italy;Spain;Turkey;United Kingdom;United States;
2012 Phase 3 EUCTR2011-004800-40-DK Argentina;Australia;Austria;Belgium;Brazil;Canada;Denmark;Egypt;France;Italy;Spain;Turkey;United Kingdom;United States;
2012 - EUCTR2010-022709-16-GB Belgium;United Kingdom;
2012 - EUCTR2010-022636-37-DE Argentina;Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Hungary;Israel;Italy;Japan;Mexico;Poland;Russian Federation;Slovakia;Switzerland;Taiwan;Turkey;United Kingdom;United States;
2011 Phase 3 EUCTR2010-022636-37-GR Argentina;Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Hungary;Israel;Italy;Japan;Mexico;Poland;Russian Federation;Slovakia;Switzerland;Taiwan;Turkey;United Kingdom;United States;
2011 Phase 2 EUCTR2010-022709-16-BE Australia;Belgium;Canada;France;Netherlands;Switzerland;United Kingdom;
2011 - EUCTR2010-022636-37-GB Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;United Kingdom;United States;
2011 - EUCTR2010-022636-37-DK Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;United Kingdom;United States;
2011 - EUCTR2010-022636-37-BE Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;United Kingdom;United States;
2011 - EUCTR2010-022636-37-AT Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;United Kingdom;United States;
2010 Phase 3 EUCTR2009-013459-31-ES Belgium;Denmark;France;Germany;Italy;Netherlands;Spain;United Kingdom;
2010 Phase 3 EUCTR2009-013459-31-BE Belgium;Denmark;France;Germany;Italy;Netherlands;Spain;United Kingdom;
2010 - EUCTR2009-013459-31-NL Belgium;Denmark;France;Germany;Italy;Netherlands;Spain;United Kingdom;
2010 - EUCTR2009-013459-31-DK Argentina;Australia;Belgium;Brazil;Canada;Denmark;Egypt;France;Germany;Italy;Netherlands;South Africa;Spain;Turkey;United Kingdom;United States;
2009 Phase 3 EUCTR2009-013459-31-GB Argentina;Australia;Belgium;Brazil;Canada;Denmark;Egypt;France;Germany;Italy;Netherlands;South Africa;Spain;Turkey;United Kingdom;United States;
2009 Phase 3 EUCTR2009-013459-31-FR Belgium;Denmark;France;Germany;Italy;Netherlands;Spain;United Kingdom;
2009 Phase 2 EUCTR2007-001838-13-GB Australia;Brazil;United Kingdom;United States;
2006 Phase 2 EUCTR2006-000181-36-GB United Kingdom;
- - EUCTR2009-013459-31-DE Belgium;Denmark;France;Germany;Italy;Netherlands;Spain;United Kingdom;
Amicus Therapeutics, UK Limited
2020 Phase 3 EUCTR2019-000222-21-GB United Kingdom;United States;
Amicus Therapeutics, UK Ltd
2019 Phase 3 EUCTR2017-000146-21-GB Australia;Germany;Italy;Spain;United Kingdom;United States;
2019 Phase 3 EUCTR2017-000146-21-ES Australia;Germany;Italy;Spain;United Kingdom;United States;
- Phase 3 EUCTR2017-000146-21-DE Australia;Germany;Italy;Spain;United Kingdom;United States;
AT2101
Amicus Therapeutics
2009 Phase 1 NCT00875160 United Kingdom;United States;
Amicus Therapeutics, Inc.
2009 - EUCTR2008-007158-36-GB United Kingdom;
2007 - EUCTR2007-003359-35-GB Germany;United Kingdom;
2007 - EUCTR2007-003359-35-DE Germany;United Kingdom;
AUTOLOGOUS CD34+ CELLS TRANSDUCED WITH A LENTIVIRAL VECTOR CONTAINING THE HUMAN SGSH GENE
University of Manchester
2019 Phase 1;Phase 2 EUCTR2019-002051-42-GB United Kingdom;
AVR-RD-01
AvroBio
2018 Phase 1/Phase 2 NCT03454893 Australia;Canada;United States;
AVR-RD-02
AvroBio
2019 Phase 1/Phase 2 NCT04145037 Australia;Canada;United States;
AX 250
Allievex Corporation
2018 Phase 2 NCT03784287 Colombia;Germany;Spain;Taiwan;Turkey;United Kingdom;United States;
2018 Phase 2 EUCTR2017-003083-13-GB Colombia;Germany;Spain;Taiwan;Turkey;United Kingdom;United States;
2016 Phase 1;Phase 2 EUCTR2015-001985-25-DE Australia;Colombia;Germany;Spain;Taiwan;Turkey;United Kingdom;
2016 Phase 1/Phase 2 NCT02754076 Colombia;Germany;Spain;Taiwan;Turkey;United Kingdom;United States;
2015 Phase 1;Phase 2 EUCTR2015-001985-25-GB Australia;Colombia;Germany;Spain;Taiwan;Turkey;United Kingdom;
- Phase 2 EUCTR2017-003083-13-DE Colombia;Germany;Spain;Taiwan;Turkey;United Kingdom;United States;
AXO-AAV-GM2 High Dose
Sio Gene Therapies
2021 Phase 1 NCT04669535 United States;
AXO-AAV-GM2 Low Dose
Sio Gene Therapies
2021 Phase 1 NCT04669535 United States;
AXO-AAV-GM2 Middle Dose
Sio Gene Therapies
2021 Phase 1 NCT04669535 United States;
AXO-AAV-GM2 Starting Dose
Sio Gene Therapies
2021 Phase 1 NCT04669535 United States;
Abatacept
Emory University
2014 Phase 1 NCT01917708 United States;
Abcertin
ISU Abxis Co., Ltd.
2020 Phase 1 NCT04787887 Australia;
Accupro
Salford Royal NHS Foundation Trust
2009 Phase 4 EUCTR2007-007482-21-GB United Kingdom;
Accuretic
Salford Royal NHS Foundation Trust
2009 Phase 4 EUCTR2007-007482-21-GB United Kingdom;
Adalimumab
Los Angeles Biomedical Research Institute
2015 Phase 1/Phase 2 NCT02437253 United States;
Adalimumab Injection [Humira]
Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
2017 Phase 1/Phase 2 NCT03153319 United States;
Adeno-associated viral vector serotype 9 containing the human N-acetyl-alpha-glucosaminidase gene
Abeona Therapeutics Europe SL.
2021 Phase 1;Phase 2 EUCTR2019-002936-97-FR France;Germany;United States;
- Phase 1;Phase 2 EUCTR2019-002936-97-DE France;Germany;Spain;United States;
Adeno-associated virus (AAV) serotype rh.10 expressing human betagalactosidase (AAVrh.10-CAG-betagal)
Lysogene
- Phase 1;Phase 2 EUCTR2019-004949-32-FR France;United Kingdom;United States;
Adeno-associated virus (AAV) serotype rh.10 expressing human betagalactosidase (AAVrh.10-CAG-ßgal)
Lysogene
- Phase 1;Phase 2 EUCTR2019-004949-32-FR France;United Kingdom;United States;
Adeno-associated virus serotype 2/6 encoding Human Iduronate 2-sulfatase (hIDS) gene donor vector, SB-A6P-HNT Donor Vector
Sangamo Therapeutics, Inc.
2018 Phase 1;Phase 2 EUCTR2018-000192-33-GB United Kingdom;United States;
Adeno-associated virus serotype 2/6 encoding Left side-zinc finger nuclease (ZFN1), SB-A6P-ZLEFT Vector
Sangamo Therapeutics, Inc.
2018 Phase 1;Phase 2 EUCTR2018-000206-28-GB United Kingdom;United States;
2018 Phase 1;Phase 2 EUCTR2018-000192-33-GB United Kingdom;United States;
Adeno-associated virus serotype 2/6 encoding Right side-zinc finger nuclease (ZFN2), SB-A6P-ZRIGHT Vector
Sangamo Therapeutics, Inc.
2018 Phase 1;Phase 2 EUCTR2018-000206-28-GB United Kingdom;United States;
2018 Phase 1;Phase 2 EUCTR2018-000192-33-GB United Kingdom;United States;
Adeno-associated virus serotype 2/6 encoding human alpha-galactosidase A cDNA
Sangamo Therapeutics, Inc.
2019 Phase 1;Phase 2 EUCTR2019-000667-24-GB United Kingdom;United States;
Adeno-associated virus serotype 2/6 encoding human iduronidase (hIDUA) gene donor vector, SB-A6P-HRL Donor Vector
Sangamo Therapeutics, Inc.
2018 Phase 1;Phase 2 EUCTR2018-000206-28-GB United Kingdom;United States;
Adeno-associated virus serotype hu68 containing the human GALC gene
Passage Bio, Inc.
- Phase 1;Phase 2 EUCTR2020-005229-95-NL Brazil;Canada;Israel;Netherlands;United Kingdom;United States;
Adeno-associated virus serotype rh.10 expressing beta-galactosidase
Lysogene
- Phase 1;Phase 2 EUCTR2019-004949-32-FR France;United Kingdom;United States;
Administration Kit
BioMarin Pharmaceutical
2020 - NCT04476862 United States;
Adrabetadex
Mandos LLC
2018 Phase 3 NCT03643562 United States;
2015 Phase 2/Phase 3 NCT04958642 Australia;France;Germany;New Zealand;Singapore;Switzerland;Turkey;United Kingdom;United States;
Afegostat tartrate
Amicus Therapeutics
2009 Phase 2 NCT00813865 Paraguay;United Kingdom;United States;
2008 Phase 2 NCT00446550 Germany;Israel;South Africa;United Kingdom;United States;
2007 Phase 2 NCT00433147 United States;
Amicus Therapeutics, Inc.
2009 - EUCTR2008-007158-36-GB United Kingdom;
Agalsidase
Amicus Therapeutics
2011 Phase 3 NCT01218659 Argentina;Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;Poland;Switzerland;Taiwan;Turkey;United Kingdom;United States;
Agalsidase alfa
AMICUS THERAPEUTICS, INC
2011 - EUCTR2010-022636-37-IT Argentina;Australia;Austria;Belgium;Brazil;Denmark;Germany;Greece;Italy;Japan;Mexico;Russian Federation;Switzerland;Taiwan;Turkey;United Kingdom;United States;
Sanofi
2020 Phase 4 NCT04143958 Czechia;
Shire
2011 Phase 3 NCT01298141 Canada;
2011 Phase 2 NCT01304277 Canada;
2010 - NCT01031173 United States;
2004 Phase 2 NCT00084084 Canada;United States;
TKT Inc
2004 - EUCTR2004-000772-14-CZ Czech Republic;
sanofi-aventis recherche et développement
- Phase 3 EUCTR2021-002320-20-ES Austria;Canada;China;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Turkey;United Kingdom;United States;
Agalsidase alpha
Shire Human Genetic Therapies, Inc. (Shire HGT)
2011 - EUCTR2007-005543-22-SI Australia;Czech Republic;Finland;Paraguay;Slovenia;United Kingdom;United States;
2010 Phase 3;Phase 4 EUCTR2007-005543-22-GB Australia;Czech Republic;Finland;Paraguay;Slovenia;United Kingdom;United States;
2010 Phase 3;Phase 4 EUCTR2007-005543-22-FI Australia;Czech Republic;Finland;Paraguay;Slovenia;United Kingdom;United States;
2008 - EUCTR2007-005543-22-CZ Australia;Czech Republic;Finland;Paraguay;Slovenia;United Kingdom;United States;
Agalsidase beta
AMICUS THERAPEUTICS, INC
2011 - EUCTR2010-022636-37-IT Argentina;Australia;Austria;Belgium;Brazil;Denmark;Germany;Greece;Italy;Japan;Mexico;Russian Federation;Switzerland;Taiwan;Turkey;United Kingdom;United States;
Genzyme Europe BV
2010 - EUCTR2006-001910-33-AT Austria;United Kingdom;
2009 Phase 3 EUCTR2007-005668-28-FR Czech Republic;France;Germany;Netherlands;Portugal;United Kingdom;
2008 - EUCTR2007-005668-28-PT Czech Republic;France;Germany;Netherlands;Portugal;United Kingdom;
2008 - EUCTR2007-005668-28-NL Czech Republic;France;Germany;Netherlands;Portugal;United Kingdom;
2008 - EUCTR2007-005668-28-GB Argentina;Brazil;Canada;Czech Republic;France;Germany;Netherlands;Norway;Poland;Portugal;United Kingdom;United States;
2008 - EUCTR2007-005668-28-DE Czech Republic;France;Germany;Netherlands;Portugal;United Kingdom;
2008 - EUCTR2007-005668-28-CZ Czech Republic;France;Germany;Netherlands;Portugal;United Kingdom;
2007 - EUCTR2006-001910-33-GB Austria;United Kingdom;
Genzyme, a Sanofi Company
2021 Phase 4 NCT05054387 China;
2012 Phase 4 NCT01650779 United States;
2008 Phase 3 NCT00701415 Argentina;Brazil;Canada;Chile;Czech Republic;Netherlands;Norway;Poland;Portugal;United Kingdom;United States;
2006 Phase 4 NCT00230607 Austria;United Kingdom;United States;
2005 Phase 4 NCT00140621 Japan;
2004 Phase 4 NCT00081497 Canada;Czech Republic;Hungary;Poland;United Kingdom;United States;
Protalix
2016 Phase 3 NCT02795676 Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Czechia;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Switzerland;Turkey;United Kingdom;United States;
sanofi-aventis recherche et développement
- Phase 3 EUCTR2021-002320-20-ES Austria;Canada;China;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Turkey;United Kingdom;United States;
Agalsidase beta (GZ419828)
Sanofi
2020 Phase 4 NCT04143958 Czechia;
Agalsidase beta (recombinant form)
Genzyme, a Sanofi Company
2004 - NCT00233870 Japan;
Aldurazyme
Genzyme, a Sanofi Company
2001 Phase 3 NCT00146770 Brazil;Canada;Germany;Italy;Netherlands;United Kingdom;United States;
HUS, Hospital for Children and Adolescents
2007 - EUCTR2006-005216-27-FI Finland;
Aldurazyme (Recombinant Human Alpha-L-Iduronidase)
Genzyme, a Sanofi Company
2005 Phase 3 NCT00258011 Japan;
2004 Phase 4 NCT00144781 Brazil;Canada;
2002 Phase 2 NCT00146757 France;Germany;Netherlands;United Kingdom;
Aldurazyme (laronidase)
Genzyme, a Sanofi Company
2010 Phase 4 NCT00418821 Italy;United States;
University of California, San Francisco
2021 Phase 1 NCT04532047 United States;
Alemtuzumab
Masonic Cancer Center, University of Minnesota
2006 Phase 2 NCT00383448 United States;
Alendronate sodium
Children's Hospital Medical Center, Cincinnati
1998 Phase 2 NCT00004488 Israel;United States;
Alfa-manosidadas humana recombinante
Zymenex A/S
2012 - EUCTR2011-004355-40-ES Belgium;Denmark;Germany;Spain;United Kingdom;
Allogeneic stem cell transplantation
Masonic Cancer Center, University of Minnesota
2009 Phase 2 NCT01043640 United States;
Ambroxol
Exsar Corporation
2012 Phase 1/Phase 2 NCT01463215 United States;
Ioroi Minoru
2021 Phase 1 JPRN-jRCT2051210024 Japan;
Maegaki Yoshihiro
2010 Phase 2 JPRN-jRCTs061180090 Japan;
Narita Aya
2019 Phase 3 JPRN-jRCTs061190017 Japan;
Shaare Zedek Medical Center
2019 Phase 2 NCT03950050 Israel;
Tottori University, Faculty of Medicine, Institute of Neurological Science
2012 Phase 2,3 JPRN-UMIN000009392 Japan;
Anakinra
Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
2019 Phase 2/Phase 3 NCT04018755 United States;
Arimoclomol
Orphazyme
2022 - NCT04316637 United States;
2018 Phase 2 NCT03746587 India;
2016 Phase 2/Phase 3 NCT02612129 Denmark;France;Germany;Italy;Poland;Spain;Switzerland;United Kingdom;United States;
Orphazyme ApS
2016 Phase 2;Phase 3 EUCTR2015-004438-93-IT Denmark;France;Germany;Italy;Poland;Spain;Switzerland;United Kingdom;United States;
2016 Phase 2;Phase 3 EUCTR2015-004438-93-ES Denmark;Spain;Switzerland;United States;
Arimoclomol (HPMC capsule)
Orphazyme A/S
2016 Phase 2;Phase 3 EUCTR2015-004438-93-DE Denmark;France;Germany;Italy;Poland;Spain;Switzerland;United Kingdom;United States;
Arimoclomol (hard gelatine capsule)
Orphazyme A/S
2016 Phase 2;Phase 3 EUCTR2015-004438-93-DE Denmark;France;Germany;Italy;Poland;Spain;Switzerland;United Kingdom;United States;
Arimoclomol citrate
Orphazyme A/S
2016 Phase 2;Phase 3 EUCTR2015-004438-93-DK Denmark;Germany;Italy;Poland;Spain;Switzerland;United Kingdom;United States;
2016 Phase 2;Phase 3 EUCTR2015-004438-93-DE Denmark;France;Germany;Italy;Poland;Spain;Switzerland;United Kingdom;United States;
Arylsulfatase A
Shire Pharmaceuticals Ireland Limited
2006 - EUCTR2006-005341-11-DK Denmark;
Autologous CD34+ cells transduced with a lentiviral vector containing the human SGSH gene
University of Manchester
2020 Phase 1/Phase 2 NCT04201405 United Kingdom;
Autologous CD34+ cells transduced with a lentiviral vector encoding the ARSA cDNA
FONDAZIONE CENTRO S. RAFFAELE DEL MONTE TABOR
2010 - EUCTR2009-017349-77-IT Italy;
Autologous Plasmablasts
Masonic Cancer Center, University of Minnesota
2021 Phase 1/Phase 2 NCT04284254 -
Azathioprine (Aza)
Genzyme, a Sanofi Company
2008 Phase 1/Phase 2 NCT00741338 Brazil;Russian Federation;Ukraine;
B (migalastat)
Amicus Therapeutics
2011 Phase 1 NCT01489995 United States;
BHP001
Torayuki Okuyama
2016 Phase 1-2 JPRN-JMA-IIA00350 Japan;
BM110
BIOMARIN PHARMACEUTICAL INC.
2011 - EUCTR2010-020199-45-IT Argentina;Australia;Brazil;Canada;Colombia;Denmark;Germany;Italy;Japan;Korea, Democratic People's Republic of;Portugal;Qatar;Saudi Arabia;Spain;Switzerland;Taiwan;Turkey;United Kingdom;United States;
BMN 110
BioMarin Pharmaceutical
2013 Phase 3 NCT01966029 Australia;
2012 Phase 2 NCT01697319 Germany;United Kingdom;United States;
2012 Phase 2 NCT01609062 Canada;Germany;United Kingdom;United States;
2011 Phase 2 NCT01515956 Italy;Taiwan;United Kingdom;United States;
2010 Phase 1/Phase 2 NCT01242111 United Kingdom;
2009 Phase 1/Phase 2 NCT00884949 United Kingdom;
2009 - NCT01858103 Puerto Rico;United States;
BMN 110 - Every Other Week
BioMarin Pharmaceutical
2011 Phase 3 NCT01415427 Argentina;Brazil;Canada;Colombia;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Netherlands;Norway;Portugal;Qatar;Saudi Arabia;Spain;Taiwan;Turkey;United Kingdom;United States;
BMN 110 - Weekly
BioMarin Pharmaceutical
2011 Phase 3 NCT01415427 Argentina;Brazil;Canada;Colombia;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Netherlands;Norway;Portugal;Qatar;Saudi Arabia;Spain;Taiwan;Turkey;United Kingdom;United States;
BMN 110 Every Other Week
BioMarin Pharmaceutical
2011 Phase 3 NCT01275066 Argentina;Australia;Brazil;Canada;Colombia;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Portugal;Qatar;Saudi Arabia;Switzerland;Taiwan;Turkey;United Kingdom;United States;
BMN 110 Weekly
BioMarin Pharmaceutical
2011 Phase 3 NCT01275066 Argentina;Australia;Brazil;Canada;Colombia;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Portugal;Qatar;Saudi Arabia;Switzerland;Taiwan;Turkey;United Kingdom;United States;
BMN 190
BioMarin Pharmaceutical
2015 Phase 1/Phase 2 NCT02485899 Germany;Italy;United Kingdom;United States;
2013 Phase 1/Phase 2 NCT01907087 Germany;Italy;United Kingdom;United States;
BioMarin Pharmaceutical Inc
2017 Phase 2 EUCTR2015-000891-85-GB Germany;Italy;United Kingdom;United States;
BioMarin Pharmaceutical Inc.
2016 Phase 2 EUCTR2015-000891-85-DE Germany;Italy;United Kingdom;
2015 Phase 2 EUCTR2014-003480-37-GB Germany;Italy;United Kingdom;United States;
2015 Phase 2 EUCTR2014-003480-37-DE Germany;Italy;United Kingdom;United States;
2015 - EUCTR2014-003480-37-IT Germany;Italy;Japan;United Kingdom;United States;
2014 Phase 1;Phase 2 EUCTR2012-005430-11-IT Germany;Italy;United Kingdom;
2013 Phase 1;Phase 2 EUCTR2012-005430-11-GB Germany;Italy;United Kingdom;United States;
2013 Phase 1;Phase 2 EUCTR2012-005430-11-DE Germany;United Kingdom;
BMN 250
BioMarin Pharmaceutical Inc.
2017 Phase 2 EUCTR2017-003083-13-ES Argentina;Australia;Colombia;Germany;Spain;Taiwan;Turkey;United Kingdom;United States;
2016 Phase 1;Phase 2 EUCTR2015-001985-25-ES Australia;Brazil;Colombia;Germany;Spain;Taiwan;Turkey;United Kingdom;
BMN190 recombinant human tripeptidyl peptidase-1 (rhTPP1)
BioMarin Pharmaceutical
2016 Phase 2 NCT02678689 Germany;Italy;United Kingdom;United States;
BUSILVEX - 6 MG/ML - CONCENTRATO PER SOLUZIONE PER INFUSIONE - USO ENDOVENOSO - FLACONCINO - 10 ML 8 FLACONCINI
OSPEDALE SAN RAFFAELE
2018 Phase 1 EUCTR2017-002430-23-IT Italy;
Beta-mercaptoethylamine Hydrochloride & 2-aminoethanethiol Hydrochloride
Recordati Rare Diseases
2020 Phase 3 EUCTR2018-002984-24-IT Belgium;France;Germany;Italy;United Kingdom;
2020 Phase 3 EUCTR2018-002984-24-DE France;Germany;United Kingdom;
- Phase 3 EUCTR2018-002984-24-BE Belgium;France;Germany;Italy;United Kingdom;
Biological assays
University Hospital, Bordeaux
2016 - NCT03123523 France;
Biopsy
University Hospital, Grenoble
2013 - NCT02841358 France;
Blood draw
Baylor Research Institute
2018 - NCT04002531 United States;
Blood sampling
University Hospital, Grenoble
2013 - NCT02841358 France;
Brain MRI/MRS/fMRI
National Human Genome Research Institute (NHGRI)
2019 Phase 1/Phase 2 NCT03952637 United States;
Brineura
BioMarin Pharmaceutical Inc
2017 Phase 2 EUCTR2015-000891-85-GB Germany;Italy;United Kingdom;United States;
BioMarin Pharmaceutical Inc.
2016 Phase 2 EUCTR2015-000891-85-DE Germany;Italy;United Kingdom;
2015 Phase 2 EUCTR2014-003480-37-GB Germany;Italy;United Kingdom;United States;
2015 Phase 2 EUCTR2014-003480-37-DE Germany;Italy;United Kingdom;United States;
Busulfan
FONDAZIONE CENTRO S. RAFFAELE DEL MONTE TABOR
2010 - EUCTR2009-017349-77-IT Italy;
Masonic Cancer Center, University of Minnesota
2009 Phase 2 NCT01043640 United States;
2008 Phase 2 NCT00668564 United States;
Busulfan, Cyclophosphamide, ATG
Masonic Cancer Center, University of Minnesota
1999 Phase 2 NCT00176917 United States;
Busulfan, Cyclophosphamide, Antithymocyte Globulin
Masonic Cancer Center, University of Minnesota
1995 Phase 2/Phase 3 NCT00176904 United States;
C (migalastat)
Amicus Therapeutics
2011 Phase 1 NCT01489995 United States;
CALD HR-D (High-Risk, Regimen C)
Masonic Cancer Center, University of Minnesota
2014 Phase 2 NCT02171104 United States;
CALD HR-D (High-Risk, Regimen D)
Masonic Cancer Center, University of Minnesota
2014 Phase 2 NCT02171104 United States;
CALD SR-A (Standard-Risk, Regimen A)
Masonic Cancer Center, University of Minnesota
2014 Phase 2 NCT02171104 United States;
CALD SR-B (Standard-Risk, Regimen B)
Masonic Cancer Center, University of Minnesota
2014 Phase 2 NCT02171104 United States;
CEREDASE™
National Institute of Neurological Disorders and Stroke (NINDS)
1993 Phase 2 NCT00001416 United States;
CRYOPRESERVED AUTOLOGOUS CD34+ CELLS TRANSDUCED WITH A LENTIVIRAL VECTOR CONTAINING HUMAN SGSH GENE
University of Manchester
2019 Phase 1;Phase 2 EUCTR2019-002051-42-GB United Kingdom;
CYSTADROPS eye drops, solution
Orphan Europe SARL
2013 - EUCTR2009-012564-13-FR France;
CYSTEAMINE HYDROCHLORIDE
Orphan Europe SARL
2013 - EUCTR2009-012564-13-FR France;
Calcium carbonate
Children's Hospital Medical Center, Cincinnati
1998 Phase 2 NCT00004488 Israel;United States;
Campath-1H
Masonic Cancer Center, University of Minnesota
2012 - NCT01626092 United States;
2009 Phase 2 NCT01043640 United States;
2008 Phase 2 NCT00668564 United States;
Cannabis sativa L., folium cum flore
Albina Nowak, MD
2021 - NCT04820361 -
Capsaicin
Universitaire Ziekenhuizen Leuven
2014 Phase 0 NCT02533076 Belgium;
Cellule CD34+ autologhe trasdotte con IDUA LV codificante per il cDNA dell'alfa-L-iduronidasi
OSPEDALE SAN RAFFAELE
2018 Phase 1 EUCTR2017-002430-23-IT Italy;
Cerdelga
Genzyme Corporation
2019 Phase 3 EUCTR2016-000301-37-NL Argentina;Canada;Egypt;France;Italy;Netherlands;Russian Federation;Spain;Sweden;Turkey;United Kingdom;
2019 Phase 3 EUCTR2016-000301-37-GB Argentina;Canada;Egypt;France;Italy;Netherlands;Russian Federation;Spain;Sweden;Turkey;United Kingdom;
2018 Phase 3 EUCTR2016-000301-37-SE Argentina;Canada;Egypt;France;Italy;Japan;Netherlands;Russian Federation;Spain;Sweden;Taiwan;Turkey;United Kingdom;
2018 Phase 3 EUCTR2016-000301-37-IT Argentina;Canada;Egypt;France;Italy;Netherlands;Russian Federation;Spain;Sweden;Turkey;United Kingdom;
2018 Phase 3 EUCTR2016-000301-37-ES Argentina;Canada;Egypt;France;Italy;Netherlands;Russian Federation;Spain;Sweden;Turkey;United Kingdom;
Cerezyme
Genzyme Corporation
2019 Phase 3 EUCTR2016-000301-37-NL Argentina;Canada;Egypt;France;Italy;Netherlands;Russian Federation;Spain;Sweden;Turkey;United Kingdom;
2019 Phase 3 EUCTR2016-000301-37-GB Argentina;Canada;Egypt;France;Italy;Netherlands;Russian Federation;Spain;Sweden;Turkey;United Kingdom;
2018 Phase 3 EUCTR2016-000301-37-SE Argentina;Canada;Egypt;France;Italy;Japan;Netherlands;Russian Federation;Spain;Sweden;Taiwan;Turkey;United Kingdom;
2018 Phase 3 EUCTR2016-000301-37-IT Argentina;Canada;Egypt;France;Italy;Netherlands;Russian Federation;Spain;Sweden;Turkey;United Kingdom;
2018 Phase 3 EUCTR2016-000301-37-ES Argentina;Canada;Egypt;France;Italy;Netherlands;Russian Federation;Spain;Sweden;Turkey;United Kingdom;
Genzyme Europe B.V.
2010 - EUCTR2008-005223-28-DE Argentina;Australia;Brazil;Canada;Czech Republic;Egypt;France;Germany;Italy;Netherlands;Russian Federation;Spain;Turkey;United Kingdom;United States;
2009 Phase 3 EUCTR2008-005223-28-GB Argentina;Australia;Brazil;Canada;Czech Republic;Egypt;France;Germany;Italy;Netherlands;Russian Federation;Spain;Turkey;United Kingdom;United States;
2009 Phase 3 EUCTR2008-005223-28-FR Czech Republic;France;Germany;Italy;Netherlands;Spain;United Kingdom;
2009 - EUCTR2008-005223-28-NL Czech Republic;France;Germany;Italy;Netherlands;Spain;United Kingdom;
2009 - EUCTR2008-005223-28-CZ Czech Republic;France;Germany;Italy;Netherlands;Spain;United Kingdom;
Genzyme, a Sanofi Company
2001 Phase 4 NCT00364858 Brazil;Canada;Italy;Poland;Spain;United Kingdom;United States;
Shire Human Genetic Therapies Inc
2007 - EUCTR2007-002840-21-ES Italy;Spain;United Kingdom;
Shire Human Genetic Therapies, Inc.
2007 - EUCTR2007-002840-21-GB Italy;Spain;United Kingdom;
Cerezyme (imiglucerase for injection)
Genzyme, a Sanofi Company
1997 Phase 4 NCT00365131 United States;
Cerezyme 400 Units Powder for concentrate for solution for infusion
Sanofi-aventis recherche & développement
- Phase 3 EUCTR2021-005402-10-FR Argentina;Brazil;Canada;China;Egypt;France;Germany;Hungary;Japan;Taiwan;Turkey;United Kingdom;United States;
- Phase 3 EUCTR2021-005402-10-DE Argentina;Brazil;Canada;China;Egypt;France;Germany;Hungary;Japan;Taiwan;Turkey;United Kingdom;United States;
Cerezyme®
ISU Abxis Co., Ltd.
2011 Phase 3 NCT01161914 -
Cerezyme® / Imiglucerase
Sanofi
2021 Phase 4 NCT04656600 China;
Cerliponase alfa
BioMarin Pharmaceutical Inc
2017 Phase 2 EUCTR2015-000891-85-GB Germany;Italy;United Kingdom;United States;
BioMarin Pharmaceutical Inc.
2016 Phase 2 EUCTR2015-000891-85-DE Germany;Italy;United Kingdom;
2015 Phase 2 EUCTR2014-003480-37-GB Germany;Italy;United Kingdom;United States;
2015 Phase 2 EUCTR2014-003480-37-DE Germany;Italy;United Kingdom;United States;
2013 Phase 1;Phase 2 EUCTR2012-005430-11-GB Germany;Italy;United Kingdom;United States;
Cholecalciferol
Children's Hospital Medical Center, Cincinnati
1998 Phase 2 NCT00004488 Israel;United States;
Clofarabine
Masonic Cancer Center, University of Minnesota
2012 - NCT01626092 United States;
2006 Phase 2 NCT00383448 United States;
Contrast sensitivity measurement
Université de Montréal
2014 - NCT02023086 Canada;
Coversyl PLUS
Salford Royal NHS Foundation Trust
2009 Phase 4 EUCTR2007-007482-21-GB United Kingdom;
Cyclophosphamide
Masonic Cancer Center, University of Minnesota
2009 Phase 2 NCT01043640 United States;
2008 Phase 2 NCT00668564 United States;
Cyclosporine A
Masonic Cancer Center, University of Minnesota
2012 - NCT01626092 United States;
2009 Phase 2 NCT01043640 United States;
2006 Phase 2 NCT00383448 United States;
Cyclosporine A (CsA)
Genzyme, a Sanofi Company
2008 Phase 1/Phase 2 NCT00741338 Brazil;Russian Federation;Ukraine;
Cystadane anhydrous
Minna Laine
2017 Phase 2 EUCTR2017-000645-48-FI Finland;
Cystadrops
RECORDATI Rare Diseases SARL
2019 Phase 3 EUCTR2018-002984-24-GB France;United Kingdom;
Recordati Rare Diseases
2020 Phase 3 EUCTR2018-002984-24-IT Belgium;France;Germany;Italy;United Kingdom;
2020 Phase 3 EUCTR2018-002984-24-DE France;Germany;United Kingdom;
- Phase 3 EUCTR2018-002984-24-FR France;United Kingdom;
- Phase 3 EUCTR2018-002984-24-BE Belgium;France;Germany;Italy;United Kingdom;
Cystagon
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
2001 Phase 4 NCT00028262 United States;
Horizon Pharma USA, Inc.
2014 Phase 3 EUCTR2012-002773-64-GB Belgium;France;Germany;Italy;Netherlands;United Kingdom;United States;
2013 Phase 3 EUCTR2012-002773-64-NL Belgium;France;Germany;Italy;Netherlands;United Kingdom;United States;
Raptor Therapeutics Inc.
2014 Phase 3 EUCTR2012-002773-64-BE Belgium;France;Germany;Italy;Netherlands;United Kingdom;United States;
2013 Phase 3 EUCTR2012-002773-64-FR Belgium;France;Italy;Netherlands;United Kingdom;United States;
2010 Phase 3 EUCTR2009-017882-42-FR France;Netherlands;
2010 - EUCTR2009-017882-42-NL France;Netherlands;
Cystagon®
Horizon Pharma USA, Inc.
2013 Phase 3 NCT01733316 Belgium;France;Italy;Netherlands;United Kingdom;United States;
2009 Phase 1/Phase 2 NCT00872729 United States;
Cystagon® (Cysteamine Bitartrate)
Horizon Pharma USA, Inc.
2010 Phase 3 NCT01000961 France;Netherlands;United States;
Cysteamine
Hospices Civils de Lyon
2011 - NCT02012114 France;
National Eye Institute (NEI)
1998 Phase 1 NCT00001736 United States;
1986 Phase 2 NCT00001213 United States;
Cysteamine (mercaptamine) viscous solution
Fundació Hospital Universitari Vall d'Hebron - Institut de Recerca (VHIR)
2014 - EUCTR2013-003228-35-ES Spain;
Cysteamine Bitartrate
Chiesi SA/NV
2020 - NCT04246060 Belgium;
Cysteamine Bitartrate Delayed-release Capsules
Horizon Pharma USA, Inc.
2010 Phase 3 NCT01197378 France;Netherlands;United States;
Cysteamine Bitartrate Delayed-release Capsules (RP103)
Horizon Pharma USA, Inc.
2010 Phase 3 NCT01000961 France;Netherlands;United States;
Cysteamine bitartrate
University of California, San Diego
2011 - NCT01432561 United States;
Cysteamine bitartrate (INN: mercaptamine bitartrate
Raptor Therapeutics Inc.
2010 - EUCTR2009-017882-42-NL France;Netherlands;
Cysteamine bitartrate (INN: mercaptamine bitartrate )
Horizon Pharma USA, Inc.
2014 Phase 3 EUCTR2012-002773-64-GB Belgium;France;Germany;Italy;Netherlands;United Kingdom;United States;
2010 Phase 3 EUCTR2010-018365-34-NL France;Netherlands;United States;
Raptor Therapeutics Inc.
2014 Phase 3 EUCTR2012-002773-64-IT Belgium;France;Germany;Italy;Netherlands;United Kingdom;United States;
2014 Phase 3 EUCTR2012-002773-64-BE Belgium;France;Germany;Italy;Netherlands;United Kingdom;United States;
2013 Phase 3 EUCTR2012-002773-64-FR Belgium;France;Italy;Netherlands;United Kingdom;United States;
2010 Phase 3 EUCTR2009-017882-42-FR France;Netherlands;
- Phase 3 EUCTR2010-018365-34-FR France;Netherlands;
Cysteamine bitartrate (INN: mercaptamine bitartrate)
Horizon Pharma USA, Inc.
2013 Phase 3 EUCTR2012-002773-64-NL Belgium;France;Germany;Italy;Netherlands;United Kingdom;United States;
Cysteamine hydrochloride
FDA Office of Orphan Products Development
1999 - NCT00010426 -
Orphan Europe SARL
2008 - EUCTR2007-006024-35-FR France;
RECORDATI Rare Diseases SARL
2019 Phase 3 EUCTR2018-002984-24-GB France;United Kingdom;
Recordati Rare Diseases
2020 Phase 3 EUCTR2018-002984-24-DE France;Germany;United Kingdom;
- Phase 3 EUCTR2018-002984-24-BE Belgium;France;Germany;Italy;United Kingdom;
D (migalastat)
Amicus Therapeutics
2011 Phase 1 NCT01489995 United States;
DEXA scan
University of Texas Southwestern Medical Center
2006 - NCT00286689 United States;
DNL310
Denali Therapeutics Inc.
2020 Phase 1/Phase 2 NCT04251026 Netherlands;United States;
DNL310 Drug Substance
Denali Therapeutics Inc.
2021 Phase 1;Phase 2 EUCTR2019-004909-27-NL Germany;Italy;Netherlands;United Kingdom;United States;
2020 Phase 1;Phase 2 EUCTR2019-004909-27-GB Germany;Italy;Netherlands;United Kingdom;United States;
DRX005B
National Institute of Neurological Disorders and Stroke (NINDS)
2002 Phase 2 NCT00048906 United States;
DUOC-01
Joanne Kurtzberg, MD
2014 Phase 1 NCT02254863 United States;
Dried blood spot (DBS) sampling
Hospices Civils de Lyon
2016 - NCT02843334 France;
E (migalastat)
Amicus Therapeutics
2011 Phase 1 NCT01489995 United States;
ELIGLUSTAT TARTRATE
Genzyme Corporation
2019 Phase 3 EUCTR2016-000301-37-NL Argentina;Canada;Egypt;France;Italy;Netherlands;Russian Federation;Spain;Sweden;Turkey;United Kingdom;
2019 Phase 3 EUCTR2016-000301-37-GB Argentina;Canada;Egypt;France;Italy;Netherlands;Russian Federation;Spain;Sweden;Turkey;United Kingdom;
2018 Phase 3 EUCTR2016-000301-37-IT Argentina;Canada;Egypt;France;Italy;Netherlands;Russian Federation;Spain;Sweden;Turkey;United Kingdom;
2018 Phase 3 EUCTR2016-000301-37-ES Argentina;Canada;Egypt;France;Italy;Netherlands;Russian Federation;Spain;Sweden;Turkey;United Kingdom;
ELX-02
Eloxx Pharmaceuticals, Inc.
2019 Phase 2 NCT04069260 Canada;
ERT
National Taiwan University Hospital
2015 - NCT02520934 Taiwan;
EU-sourced Cerezyme
ISU Abxis Co., Ltd.
2020 Phase 1 NCT04787887 Australia;
Echocardiogram
Baylor Research Institute
2018 - NCT04002531 United States;
Elaprase
Shire
2022 Phase 4 NCT05058391 India;
2015 Phase 2/Phase 3 NCT02412787 Argentina;Australia;Canada;Colombia;France;Mexico;Spain;United Kingdom;United States;
2010 Phase 1/Phase 2 NCT01506141 Canada;United Kingdom;United States;
Shire Human Genetic Therapies, Inc
2016 Phase 4 EUCTR2014-004804-31-DE Costa Rica;Dominican Republic;Germany;Malaysia;Oman;Philippines;Saudi Arabia;Serbia;Thailand;United States;Vietnam;
Elaprase 2mg/ml
Shire Human Genetic Therapies, Inc. (Shire HGT)
- Phase 4 EUCTR2007-006044-22-PL Poland;
Elaprase for intravenous (IV) infusion
Shire
2015 Phase 4 NCT02455622 Dominican Republic;Germany;Malaysia;Oman;Philippines;Saudi Arabia;Serbia;Thailand;United States;Vietnam;
Elaprase_
Green Cross Corporation
2010 Phase 1/Phase 2 NCT01301898 Korea, Republic of;
Elelyso
Ari Zimran
2020 Phase 4 NCT04002830 India;Israel;Turkey;
Shaare Zedek Medical Center
2017 - NCT04353466 -
Elelyso 60 units
Pfizer
2019 Phase 4 NCT03021941 United States;
Eliglustat
Genzyme Europe B.V.
2011 Phase 3 EUCTR2009-015811-42-PT Argentina;Australia;Austria;Brazil;Canada;China;Croatia;France;Greece;India;Japan;Netherlands;Portugal;Romania;Russian Federation;Serbia;Sweden;United States;
2011 Phase 3 EUCTR2009-015811-42-AT Argentina;Australia;Austria;Brazil;Canada;China;Croatia;France;Greece;India;Japan;Netherlands;Portugal;Romania;Russian Federation;Serbia;Sweden;United States;
2011 - EUCTR2009-015811-42-GR Austria;Greece;Portugal;Sweden;
2010 Phase 3 EUCTR2009-015811-42-SE Argentina;Australia;Austria;Brazil;Canada;China;Croatia;France;Greece;India;Japan;Netherlands;Portugal;Romania;Russian Federation;Serbia;Sweden;United States;
2010 - EUCTR2008-005223-28-DE Argentina;Australia;Brazil;Canada;Czech Republic;Egypt;France;Germany;Italy;Netherlands;Russian Federation;Spain;Turkey;United Kingdom;United States;
2010 - EUCTR2008-005222-37-BG Bulgaria;Canada;Colombia;India;Israel;Jordan;Lebanon;Mexico;Netherlands;Russian Federation;Saudi Arabia;Serbia;Tunisia;United Kingdom;United States;
2009 Phase 3 EUCTR2008-005223-28-GB Argentina;Australia;Brazil;Canada;Czech Republic;Egypt;France;Germany;Italy;Netherlands;Russian Federation;Spain;Turkey;United Kingdom;United States;
2009 Phase 3 EUCTR2008-005222-37-GB Bulgaria;Canada;Colombia;India;Israel;Jordan;Lebanon;Mexico;Netherlands;Russian Federation;Saudi Arabia;Serbia;Tunisia;United Kingdom;United States;
2009 - EUCTR2008-005223-28-NL Czech Republic;France;Germany;Italy;Netherlands;Spain;United Kingdom;
2009 - EUCTR2008-005222-37-NL Bulgaria;Netherlands;United Kingdom;
Genzyme, a Sanofi Company
2015 Phase 1 NCT02536911 United States;
2015 Phase 1 NCT02422654 United States;
National Taiwan University Hospital
2018 - NCT03519646 Taiwan;
Sanofi
2015 Phase 1 NCT02536937 United States;
Eliglustat GZ385660
Genzyme, a Sanofi Company
2015 Phase 3 NCT02536755 Canada;Russian Federation;Tunisia;
Sanofi
2018 Phase 3 NCT03485677 Argentina;Canada;France;Italy;Japan;Russian Federation;Spain;Sweden;Turkey;United Kingdom;
Eliglustat Tartrate – Powder for Oral Suspension
Genzyme Corporation
2018 Phase 3 EUCTR2016-000301-37-SE Argentina;Canada;Egypt;France;Italy;Japan;Netherlands;Russian Federation;Spain;Sweden;Taiwan;Turkey;United Kingdom;
Eliglustat tartrate
Genzyme Europe B.V.
2009 - EUCTR2008-005223-28-CZ Czech Republic;France;Germany;Italy;Netherlands;Spain;United Kingdom;
Genzyme, a Sanofi Company
2010 Phase 3 NCT01074944 Australia;Austria;Brazil;Canada;China;Croatia;France;Greece;India;Japan;Netherlands;Portugal;Romania;Russian Federation;Serbia;Sweden;United States;
2009 Phase 3 NCT00943111 Argentina;Australia;Brazil;Canada;Czech Republic;Egypt;France;Germany;Italy;Netherlands;Russian Federation;Spain;Turkey;United Kingdom;United States;
2009 Phase 3 NCT00891202 Bulgaria;Canada;Chile;Colombia;Georgia;India;Israel;Jordan;Lebanon;Mexico;Netherlands;Russian Federation;Saudi Arabia;Serbia;Tunisia;United Kingdom;United States;
2006 Phase 2 NCT00358150 Argentina;Israel;Italy;Mexico;Russian Federation;United States;
Elosulfase Alfa 1 MG/ML Intravenous Solution [VIMIZIM]
GOIZET
2018 - NCT03204370 France;
Enalapril and other angiotensin converting enzyme inhibitors
University of Alabama at Birmingham
2007 - NCT00446862 Germany;Slovenia;United States;
Enriched Hematopoetic Stem Cell Infusion
Talaris Therapeutics Inc.
2014 - NCT02084121 United States;
2013 - NCT02021266 United States;
Enzyme replacement
Rigshospitalet, Denmark
2001 Phase 4 NCT01997489 Denmark;
Enzyme replacement therapy
Ulla Feldt-Rasmussen
2015 - NCT02969200 Denmark;
Enzyme replacement therapy (Agalsidase alpha (Replagal®))
University Hospital, Rouen
2017 - NCT02956954 France;
Enzyme replacement therapy (ERT)
Idorsia Pharmaceuticals Ltd.
2015 Phase 1 NCT02930655 Germany;
FABRAZYME - 35 MG POLVERE PER CONCENTRATO PER SOLUZIONE PER INFUSIONE ENDOVENOSA 1 FLACONCINO
FONDAZIONE POLICLINICO UNIVERSITARIO AGOSTINO GEMELLI IRCCS UNIVERSITA' CATTOLICA DEL SACRO CUORE
2018 Phase 4 EUCTR2018-000368-27-IT Italy;
PROTALIX LTD
2017 Phase 3 EUCTR2016-000378-38-IT Australia;Belgium;Canada;Czech Republic;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Turkey;United Kingdom;United States;
FBX-101
Forge Biologics, Inc
2021 Phase 1/Phase 2 NCT04693598 United States;
FLT190
Freeline Therapeutics Limited
2019 Phase 1;Phase 2 EUCTR2018-002097-51-IT Denmark;France;Germany;Italy;Norway;United Kingdom;United States;
Freeline Therapeutics Ltd
2021 Phase 1;Phase 2 EUCTR2018-002097-51-AT Austria;Denmark;France;Germany;Italy;Norway;Switzerland;United Kingdom;United States;
2020 Phase 2 EUCTR2019-004645-32-GB Denmark;France;Germany;Italy;Norway;United Kingdom;United States;
2020 Phase 1;Phase 2 EUCTR2018-002097-51-DK Denmark;France;Germany;Italy;Norway;Spain;United Kingdom;United States;
2020 Phase 1;Phase 2 EUCTR2018-002097-51-DE Austria;Denmark;France;Germany;Italy;Norway;Switzerland;United Kingdom;United States;
FLUDARABINA ACCORD - 25 MG/ML CONCENTRATO PER SOLUZIONE INIETTABILE O PER INFUSIONE 5 FLACONCINI IN VETRO DA 2 ML
OSPEDALE SAN RAFFAELE
2018 Phase 1 EUCTR2017-002430-23-IT Italy;
FLUDARABINA FOSFATO
OSPEDALE SAN RAFFAELE
2018 Phase 1 EUCTR2017-002430-23-IT Italy;
Fabrazyme
Amicus Therapeutics, Inc.
2012 - EUCTR2010-022709-16-GB Belgium;United Kingdom;
2012 - EUCTR2010-022636-37-DE Argentina;Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Hungary;Israel;Italy;Japan;Mexico;Poland;Russian Federation;Slovakia;Switzerland;Taiwan;Turkey;United Kingdom;United States;
2011 Phase 3 EUCTR2010-022636-37-GR Argentina;Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Hungary;Israel;Italy;Japan;Mexico;Poland;Russian Federation;Slovakia;Switzerland;Taiwan;Turkey;United Kingdom;United States;
2011 Phase 2 EUCTR2010-022709-16-BE Australia;Belgium;Canada;France;Netherlands;Switzerland;United Kingdom;
2011 - EUCTR2010-022636-37-GB Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;United Kingdom;United States;
2011 - EUCTR2010-022636-37-DK Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;United Kingdom;United States;
2011 - EUCTR2010-022636-37-BE Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;United Kingdom;United States;
2011 - EUCTR2010-022636-37-AT Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;United Kingdom;United States;
Genzyme Europe BV
2010 - EUCTR2006-001910-33-AT Austria;United Kingdom;
2009 Phase 3 EUCTR2007-005668-28-FR Czech Republic;France;Germany;Netherlands;Portugal;United Kingdom;
2008 - EUCTR2007-005668-28-PT Czech Republic;France;Germany;Netherlands;Portugal;United Kingdom;
2008 - EUCTR2007-005668-28-NL Czech Republic;France;Germany;Netherlands;Portugal;United Kingdom;
2008 - EUCTR2007-005668-28-GB Argentina;Brazil;Canada;Czech Republic;France;Germany;Netherlands;Norway;Poland;Portugal;United Kingdom;United States;
2008 - EUCTR2007-005668-28-DE Czech Republic;France;Germany;Netherlands;Portugal;United Kingdom;
2008 - EUCTR2007-005668-28-CZ Czech Republic;France;Germany;Netherlands;Portugal;United Kingdom;
2007 - EUCTR2006-001910-33-GB Austria;United Kingdom;
Protalix Ltd.
2019 Phase 3 EUCTR2016-000378-38-FR Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Denmark;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Switzerland;Turkey;United Kingdom;United States;
2019 Phase 3 EUCTR2016-000378-38-FI Argentina;Australia;Belgium;Brazil;Canada;Czechia;Denmark;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Slovenia;Spain;Sweden;Switzerland;Turkey;United Kingdom;United States;
2017 Phase 3 EUCTR2016-000378-38-SI Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Denmark;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Switzerland;Turkey;United Kingdom;United States;
2017 Phase 3 EUCTR2016-000378-38-NL Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Turkey;United Kingdom;United States;
2016 Phase 3 EUCTR2016-000378-38-HU Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Turkey;United Kingdom;United States;
2016 Phase 3 EUCTR2016-000378-38-GB Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Germany;Hungary;Netherlands;Norway;Paraguay;Slovenia;Spain;Turkey;United Kingdom;United States;
2016 Phase 3 EUCTR2016-000378-38-ES Australia;Belgium;Canada;Czech Republic;Germany;Hungary;Netherlands;Norway;Paraguay;Spain;Turkey;United Kingdom;United States;
2016 Phase 3 EUCTR2016-000378-38-BE Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Turkey;United Kingdom;United States;
SANOFI-AVENTIS RECHERCHE E DEVELOPPEMENT
2022 Phase 3 EUCTR2021-002320-20-IT Austria;Canada;China;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Turkey;United Kingdom;United States;
Sanofi Aventis Groupe (SAG)
2019 Phase 4 EUCTR2019-000064-21-GB Austria;Canada;Czech Republic;Denmark;France;Germany;Italy;Norway;Spain;Turkey;United Kingdom;
- Phase 4 EUCTR2019-000065-20-NO Canada;France;Norway;United States;
- Phase 4 EUCTR2019-000064-21-NO Austria;Canada;Czech Republic;Denmark;France;Germany;Italy;Norway;Spain;Turkey;United Kingdom;
- Phase 4 EUCTR2019-000064-21-CZ Austria;Canada;Czech Republic;Denmark;France;Germany;Italy;Norway;Spain;Turkey;United Kingdom;
sanofi-aventis recherche et développement
- Phase 3 EUCTR2021-002320-20-ES Austria;Canada;China;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Turkey;United Kingdom;United States;
Fabrazyme (agalsidase beta)
Genzyme, a Sanofi Company
2006 Phase 4 NCT00312767 United States;
2003 Phase 2 NCT00196716 Czech Republic;Estonia;Poland;Slovakia;
2002 Phase 2 NCT00837824 United States;
2002 Phase 2 NCT00074958 France;Italy;Poland;United Kingdom;United States;
2001 Phase 4 NCT00074984 Canada;Czech Republic;Hungary;Poland;United Kingdom;United States;
1999 Phase 3 NCT00074971 France;Netherlands;Puerto Rico;United Kingdom;United States;
Fluorescein
Ohio State University
2010 - NCT01165697 United States;
GA-GCB
SHIRE HUMAN GENETIC THERAPIES, INC.
2008 - EUCTR2007-002840-21-IT Italy;Spain;United Kingdom;
2008 - EUCTR2006-006304-11-IT Italy;Spain;United Kingdom;
Shire
2005 Phase 1/Phase 2 NCT00391625 Former Serbia and Montenegro;Israel;Romania;Serbia;
Shire Human Genetic Therapies
- - EUCTR2012-003427-38-Outside-EU/EEA Egypt;India;Tunisia;
Shire Human Genetic Therapies Inc
2007 Phase 2;Phase 3 EUCTR2006-006304-11-GB Germany;Italy;Spain;United Kingdom;
2007 - EUCTR2007-002840-21-ES Italy;Spain;United Kingdom;
2007 - EUCTR2006-006304-11-ES Italy;Spain;United Kingdom;
- Phase 2;Phase 3 EUCTR2006-006304-11-DE Germany;Italy;Spain;United Kingdom;
Shire Human Genetic Therapies, Inc.
2008 Phase 3 EUCTR2008-001965-27-GB Spain;United Kingdom;
2008 - EUCTR2008-001965-27-ES Spain;United Kingdom;
2007 - EUCTR2007-002840-21-GB Italy;Spain;United Kingdom;
GA-GCB (velaglucerase alfa)
Shire
2007 Phase 2/Phase 3 NCT00478647 Israel;Poland;Spain;United Kingdom;United States;
GC1111_
Green Cross Corporation
2010 Phase 1/Phase 2 NCT01301898 Korea, Republic of;
GC1119
Green Cross Corporation
2012 Phase 1 NCT01653444 Korea, Republic of;
GLA gene
Chiayi Christian Hospital
2016 - NCT03596398 -
GLA genetic sequencing.
Chang Gung Memorial Hospital
2018 - NCT05056636 Taiwan;
GLUCOCEREBROSIDASE UMANO GENE-ATTIVATO
SHIRE HUMAN GENETIC THERAPIES, INC
2018 Phase 4 EUCTR2015-001578-17-IT Germany;Israel;Italy;Spain;United Kingdom;United States;
GNR-055
AO GENERIUM
2020 Phase 1 NCT04539340 Russian Federation;
GR181413
GLAXOSMITHKLINE RESEARCH & DEVELOPMENT LTD.
2012 - EUCTR2011-004800-40-IT Australia;Austria;Belgium;Brazil;Denmark;Egypt;Italy;Spain;Turkey;United Kingdom;United States;
GlaxoSmithKline Research & Development Limited
2012 Phase 3 EUCTR2011-004800-40-ES Australia;Austria;Belgium;Brazil;Denmark;Egypt;France;Italy;Spain;Turkey;United Kingdom;United States;
GR181413A
Amicus Therapeutics, Inc.
2014 Phase 3 EUCTR2011-004800-40-AT Argentina;Australia;Austria;Belgium;Brazil;Canada;Denmark;Egypt;France;Italy;Spain;Turkey;United Kingdom;United States;
2013 Phase 3 EUCTR2011-004800-40-BE Argentina;Australia;Austria;Belgium;Brazil;Canada;Denmark;Egypt;France;Italy;Spain;Turkey;United Kingdom;United States;
2012 Phase 3 EUCTR2011-004800-40-GB Argentina;Australia;Austria;Belgium;Brazil;Canada;Denmark;Egypt;France;Italy;Spain;Turkey;United Kingdom;United States;
2012 Phase 3 EUCTR2011-004800-40-DK Argentina;Australia;Austria;Belgium;Brazil;Canada;Denmark;Egypt;France;Italy;Spain;Turkey;United Kingdom;United States;
GLAXOSMITHKLINE RESEARCH & DEVELOPMENT LTD.
2012 - EUCTR2011-004800-40-IT Australia;Austria;Belgium;Brazil;Denmark;Egypt;Italy;Spain;Turkey;United Kingdom;United States;
GlaxoSmithKline Research & Development Limited
2012 Phase 3 EUCTR2011-004800-40-ES Australia;Austria;Belgium;Brazil;Denmark;Egypt;France;Italy;Spain;Turkey;United Kingdom;United States;
GR181413A/AT1001 capsule
Amicus Therapeutics
2011 Phase 1 NCT01853852 Australia;
GR181413A/AT1001 solution
Amicus Therapeutics
2011 Phase 1 NCT01853852 Australia;
GSK2788723
GlaxoSmithKline
2012 - NCT01602601 Japan;
GZ/SAR402671
Genzyme, a Sanofi Company
2015 Phase 2 NCT02489344 France;Poland;Russian Federation;United Kingdom;United States;
2014 Phase 2 NCT02228460 Czech Republic;Czechia;France;Poland;Russian Federation;United Kingdom;United States;
Sanofi-aventis recherche & développement
- Phase 3 EUCTR2021-005402-10-FR Argentina;Brazil;Canada;China;Egypt;France;Germany;Hungary;Japan;Taiwan;Turkey;United Kingdom;United States;
- Phase 3 EUCTR2021-005402-10-DE Argentina;Brazil;Canada;China;Egypt;France;Germany;Hungary;Japan;Taiwan;Turkey;United Kingdom;United States;
GZ385660
Genzyme Corporation
2019 Phase 3 EUCTR2016-000301-37-NL Argentina;Canada;Egypt;France;Italy;Netherlands;Russian Federation;Spain;Sweden;Turkey;United Kingdom;
2019 Phase 3 EUCTR2016-000301-37-GB Argentina;Canada;Egypt;France;Italy;Netherlands;Russian Federation;Spain;Sweden;Turkey;United Kingdom;
2018 Phase 3 EUCTR2016-000301-37-SE Argentina;Canada;Egypt;France;Italy;Japan;Netherlands;Russian Federation;Spain;Sweden;Taiwan;Turkey;United Kingdom;
2018 Phase 3 EUCTR2016-000301-37-IT Argentina;Canada;Egypt;France;Italy;Netherlands;Russian Federation;Spain;Sweden;Turkey;United Kingdom;
2018 Phase 3 EUCTR2016-000301-37-ES Argentina;Canada;Egypt;France;Italy;Netherlands;Russian Federation;Spain;Sweden;Turkey;United Kingdom;
GZ402665
Genzyme Corporation
2015 Phase 2 EUCTR2014-003198-40-IT Brazil;Chile;France;Germany;Italy;United Kingdom;United States;
2015 Phase 2 EUCTR2014-003198-40-GB Brazil;France;Germany;Italy;United Kingdom;United States;
2015 Phase 2 EUCTR2014-003198-40-DE Brazil;France;Germany;Italy;United Kingdom;United States;
- Phase 2 EUCTR2014-003198-40-FR Brazil;Chile;France;Germany;Italy;United Kingdom;United States;
GZ402671
GENZYME CORPORATION
2020 Phase 3 EUCTR2019-002375-34-IT Argentina;Austria;Brazil;Czech Republic;Czechia;France;Germany;Italy;Japan;Portugal;Russian Federation;Spain;Turkey;United Kingdom;United States;
Genzyme Corporation
2021 Phase 3 EUCTR2019-002375-34-AT Argentina;Austria;Brazil;Czech Republic;Czechia;France;Germany;Italy;Japan;Portugal;Russian Federation;Spain;Turkey;United Kingdom;United States;
2021 Phase 2;Phase 3 EUCTR2020-003120-17-HU Argentina;Brazil;Canada;China;Egypt;France;Germany;Hungary;Japan;Sweden;Taiwan;Turkey;United States;
2021 Phase 2;Phase 3 EUCTR2020-003120-17-FR Argentina;Brazil;Canada;China;Egypt;France;Germany;Hungary;Japan;Sweden;Taiwan;Turkey;United States;
2021 Phase 2;Phase 3 EUCTR2020-003120-17-DE Argentina;Brazil;Canada;China;Egypt;France;Germany;Hungary;Japan;Sweden;Taiwan;Turkey;United States;
2020 Phase 3 EUCTR2019-002375-34-PT Argentina;Austria;Brazil;Czech Republic;Czechia;France;Germany;Italy;Japan;Portugal;Russian Federation;Spain;Turkey;United Kingdom;United States;
2020 Phase 3 EUCTR2019-002375-34-GB Argentina;Austria;Brazil;Czech Republic;France;Germany;Italy;Japan;Portugal;Russian Federation;Spain;Turkey;United Kingdom;United States;
2020 Phase 3 EUCTR2019-002375-34-DE Argentina;Austria;Brazil;Czech Republic;France;Germany;Italy;Japan;Portugal;Russian Federation;Spain;Turkey;United Kingdom;United States;
2020 Phase 3 EUCTR2019-002375-34-CZ Argentina;Austria;Brazil;Czech Republic;France;Germany;Italy;Japan;Portugal;Russian Federation;Spain;Turkey;United Kingdom;United States;
2016 Phase 2 EUCTR2014-002550-39-DE Germany;Japan;United Kingdom;United States;
- Phase 3 EUCTR2019-002375-34-FR Argentina;Austria;Brazil;Czech Republic;France;Germany;Italy;Japan;Portugal;Russian Federation;Spain;Turkey;United Kingdom;United States;
- Phase 2;Phase 3 EUCTR2020-003120-17-SE Argentina;Brazil;Canada;China;Egypt;France;Germany;Hungary;Japan;Sweden;Taiwan;Turkey;United States;
GZ402671 / SAR402671
Genzyme Corporation
2014 Phase 2 EUCTR2013-005324-41-GB Czech Republic;France;Poland;Russian Federation;United Kingdom;United States;
2014 Phase 2 EUCTR2013-005324-41-CZ Czech Republic;France;Poland;Russian Federation;United Kingdom;United States;
Gabapentin
University of Minnesota
2012 Phase 2 NCT01588314 United States;
Galafold
Amicus Therapeutics UK, Ltd
2020 Phase 3 EUCTR2017-000146-21-IT Australia;Germany;Italy;Spain;United Kingdom;United States;
Amicus Therapeutics, UK Ltd
2019 Phase 3 EUCTR2017-000146-21-GB Australia;Germany;Italy;Spain;United Kingdom;United States;
2019 Phase 3 EUCTR2017-000146-21-ES Australia;Germany;Italy;Spain;United Kingdom;United States;
- Phase 3 EUCTR2017-000146-21-DE Australia;Germany;Italy;Spain;United Kingdom;United States;
SANOFI-AVENTIS RECHERCHE E DEVELOPPEMENT
2022 Phase 3 EUCTR2021-002320-20-IT Austria;Canada;China;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Turkey;United Kingdom;United States;
Sanofi Aventis Groupe (SAG)
- Phase 4 EUCTR2019-000065-20-NO Canada;France;Norway;United States;
sanofi-aventis recherche et développement
- Phase 3 EUCTR2021-002320-20-ES Austria;Canada;China;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Turkey;United Kingdom;United States;
Galsulfase
BioMarin Pharmaceutical Inc.
2007 Phase 4 EUCTR2005-003512-30-FR France;Portugal;
Gene Activated Human Glucocerebrosidase
SHIRE HUMAN GENETIC THERAPIES, INC.
2008 - EUCTR2007-002840-21-IT Italy;Spain;United Kingdom;
Gene Activated a-Galactosidase A
Shire Human Genetic Therapies, Inc.
2021 Phase 3 EUCTR2018-004689-32-SE Argentina;Australia;Belgium;Bosnia and Herzegovina;Brazil;Canada;Croatia;Finland;Germany;Greece;Hungary;Italy;Latvia;Poland;Portugal;Romania;Slovenia;Spain;Sweden;Ukraine;
2021 Phase 3 EUCTR2018-004689-32-PT Argentina;Australia;Belgium;Bosnia and Herzegovina;Brazil;Canada;Croatia;Finland;Germany;Greece;Hungary;Italy;Latvia;Poland;Portugal;Romania;Slovenia;Spain;Sweden;Ukraine;
2021 Phase 3 EUCTR2018-004689-32-PL Argentina;Australia;Belgium;Bosnia and Herzegovina;Brazil;Canada;Croatia;Finland;Germany;Greece;Hungary;Italy;Latvia;Poland;Portugal;Romania;Slovenia;Spain;Sweden;Ukraine;
2021 Phase 3 EUCTR2018-004689-32-GR Argentina;Australia;Belgium;Bosnia and Herzegovina;Brazil;Canada;Croatia;Finland;Germany;Greece;Hungary;Italy;Latvia;Poland;Portugal;Romania;Slovenia;Spain;Sweden;Ukraine;
2021 Phase 3 EUCTR2018-004689-32-FI Argentina;Australia;Belgium;Bosnia and Herzegovina;Brazil;Canada;Croatia;Finland;Germany;Greece;Hungary;Italy;Latvia;Poland;Portugal;Romania;Slovenia;Spain;Sweden;Ukraine;
Gene activated human glucocerebrosidase
Shire Human Genetic Therapies Inc
2007 Phase 2;Phase 3 EUCTR2006-006304-11-GB Germany;Italy;Spain;United Kingdom;
2007 - EUCTR2006-006304-11-ES Italy;Spain;United Kingdom;
- Phase 2;Phase 3 EUCTR2006-006304-11-DE Germany;Italy;Spain;United Kingdom;
Shire Human Genetic Therapies, Inc.
2008 - EUCTR2008-001965-27-ES Spain;United Kingdom;
Gene activated human glucocerebrosidase, velaglucerase alfa
Shire Human Genetic Therapies, Inc.
2016 Phase 4 EUCTR2015-001578-17-GB Canada;Germany;Israel;Spain;United Kingdom;United States;
2016 Phase 4 EUCTR2015-001578-17-DE Germany;India;Israel;Italy;Spain;Turkey;United Kingdom;United States;
2016 - EUCTR2015-001578-17-ES Canada;Germany;Israel;Italy;Spain;United Kingdom;United States;
2008 Phase 3 EUCTR2008-001965-27-GB Spain;United Kingdom;
Gene-Activated Human Glucocerebrosidase
SHIRE HUMAN GENETIC THERAPIES, INC.
2008 - EUCTR2006-006304-11-IT Italy;Spain;United Kingdom;
Shire Human Genetic Therapies Inc
2007 Phase 2;Phase 3 EUCTR2006-006304-11-GB Germany;Italy;Spain;United Kingdom;
2007 - EUCTR2006-006304-11-ES Italy;Spain;United Kingdom;
- Phase 2;Phase 3 EUCTR2006-006304-11-DE Germany;Italy;Spain;United Kingdom;
Shire Human Genetic Therapies, Inc.
2008 - EUCTR2008-001965-27-ES Spain;United Kingdom;
Gene-Activated Human Glucocerebrosidase 200U/vial
Shire Human Genetic Therapies, Inc.
2008 Phase 3 EUCTR2008-001965-27-GB Spain;United Kingdom;
Gene-Activated Human Glucocerebrosidase 400U/vial
Shire Human Genetic Therapies
- - EUCTR2012-003427-38-Outside-EU/EEA Egypt;India;Tunisia;
Shire Human Genetic Therapies, Inc.
2008 Phase 3 EUCTR2008-001965-27-GB Spain;United Kingdom;
Genetic HMI-203
Homology Medicines, Inc
2022 Phase 1 NCT05238324 United States;
Genistein aglycone
Manchester University NHS Foundation Trust
2014 Phase 3 EUCTR2013-001479-18-GB United Kingdom;
Genz-112638
Genzyme Europe B.V.
2011 Phase 3 EUCTR2009-015811-42-PT Argentina;Australia;Austria;Brazil;Canada;China;Croatia;France;Greece;India;Japan;Netherlands;Portugal;Romania;Russian Federation;Serbia;Sweden;United States;
2011 Phase 3 EUCTR2009-015811-42-AT Argentina;Australia;Austria;Brazil;Canada;China;Croatia;France;Greece;India;Japan;Netherlands;Portugal;Romania;Russian Federation;Serbia;Sweden;United States;
2011 - EUCTR2009-015811-42-GR Austria;Greece;Portugal;Sweden;
2010 Phase 3 EUCTR2009-015811-42-SE Argentina;Australia;Austria;Brazil;Canada;China;Croatia;France;Greece;India;Japan;Netherlands;Portugal;Romania;Russian Federation;Serbia;Sweden;United States;
2010 - EUCTR2008-005223-28-ES Czech Republic;France;Germany;Italy;Netherlands;Spain;United Kingdom;
2010 - EUCTR2008-005223-28-DE Argentina;Australia;Brazil;Canada;Czech Republic;Egypt;France;Germany;Italy;Netherlands;Russian Federation;Spain;Turkey;United Kingdom;United States;
2010 - EUCTR2008-005222-37-BG Bulgaria;Canada;Colombia;India;Israel;Jordan;Lebanon;Mexico;Netherlands;Russian Federation;Saudi Arabia;Serbia;Tunisia;United Kingdom;United States;
2009 Phase 3 EUCTR2008-005223-28-GB Argentina;Australia;Brazil;Canada;Czech Republic;Egypt;France;Germany;Italy;Netherlands;Russian Federation;Spain;Turkey;United Kingdom;United States;
2009 Phase 3 EUCTR2008-005223-28-FR Czech Republic;France;Germany;Italy;Netherlands;Spain;United Kingdom;
2009 Phase 3 EUCTR2008-005222-37-GB Bulgaria;Canada;Colombia;India;Israel;Jordan;Lebanon;Mexico;Netherlands;Russian Federation;Saudi Arabia;Serbia;Tunisia;United Kingdom;United States;
2009 - EUCTR2008-005223-28-NL Czech Republic;France;Germany;Italy;Netherlands;Spain;United Kingdom;
2009 - EUCTR2008-005223-28-CZ Czech Republic;France;Germany;Italy;Netherlands;Spain;United Kingdom;
2009 - EUCTR2008-005222-37-NL Bulgaria;Netherlands;United Kingdom;
Genzyme Europe BV
2010 - EUCTR2008-005223-28-IT Czech Republic;France;Germany;Italy;Netherlands;Spain;United Kingdom;
2006 - EUCTR2005-004732-42-IT Italy;
Genz-682452-AA
Genzyme Corporation
2014 Phase 2 EUCTR2013-005324-41-GB Czech Republic;France;Poland;Russian Federation;United Kingdom;United States;
2014 Phase 2 EUCTR2013-005324-41-CZ Czech Republic;France;Poland;Russian Federation;United Kingdom;United States;
Genz-682452-AU
GENZYME CORPORATION
2020 Phase 3 EUCTR2019-002375-34-IT Argentina;Austria;Brazil;Czech Republic;Czechia;France;Germany;Italy;Japan;Portugal;Russian Federation;Spain;Turkey;United Kingdom;United States;
Genzyme Corporation
2021 Phase 3 EUCTR2019-002375-34-AT Argentina;Austria;Brazil;Czech Republic;Czechia;France;Germany;Italy;Japan;Portugal;Russian Federation;Spain;Turkey;United Kingdom;United States;
2021 Phase 2;Phase 3 EUCTR2020-003120-17-HU Argentina;Brazil;Canada;China;Egypt;France;Germany;Hungary;Japan;Sweden;Taiwan;Turkey;United States;
2021 Phase 2;Phase 3 EUCTR2020-003120-17-FR Argentina;Brazil;Canada;China;Egypt;France;Germany;Hungary;Japan;Sweden;Taiwan;Turkey;United States;
2021 Phase 2;Phase 3 EUCTR2020-003120-17-DE Argentina;Brazil;Canada;China;Egypt;France;Germany;Hungary;Japan;Sweden;Taiwan;Turkey;United States;
2020 Phase 3 EUCTR2019-002375-34-PT Argentina;Austria;Brazil;Czech Republic;Czechia;France;Germany;Italy;Japan;Portugal;Russian Federation;Spain;Turkey;United Kingdom;United States;
2020 Phase 3 EUCTR2019-002375-34-GB Argentina;Austria;Brazil;Czech Republic;France;Germany;Italy;Japan;Portugal;Russian Federation;Spain;Turkey;United Kingdom;United States;
2020 Phase 3 EUCTR2019-002375-34-DE Argentina;Austria;Brazil;Czech Republic;France;Germany;Italy;Japan;Portugal;Russian Federation;Spain;Turkey;United Kingdom;United States;
2020 Phase 3 EUCTR2019-002375-34-CZ Argentina;Austria;Brazil;Czech Republic;France;Germany;Italy;Japan;Portugal;Russian Federation;Spain;Turkey;United Kingdom;United States;
2016 Phase 2 EUCTR2014-002550-39-DE Germany;Japan;United Kingdom;United States;
2015 Phase 2 EUCTR2014-004995-49-PL Czech Republic;France;Poland;Russian Federation;United Kingdom;United States;
2015 Phase 2 EUCTR2014-004995-49-GB Czech Republic;France;Poland;Russian Federation;United Kingdom;United States;
2015 Phase 2 EUCTR2014-004995-49-FR Czech Republic;France;Poland;Russian Federation;United Kingdom;United States;
- Phase 3 EUCTR2019-002375-34-FR Argentina;Austria;Brazil;Czech Republic;France;Germany;Italy;Japan;Portugal;Russian Federation;Spain;Turkey;United Kingdom;United States;
- Phase 2;Phase 3 EUCTR2020-003120-17-SE Argentina;Brazil;Canada;China;Egypt;France;Germany;Hungary;Japan;Sweden;Taiwan;Turkey;United States;
Glucocerebrosidase
National Center for Research Resources (NCRR)
1999 Phase 2 NCT00004293 United States;
HGT-1110
Shire
2013 Phase 1/Phase 2 NCT01887938 Argentina;Australia;Brazil;Czech Republic;Czechia;Denmark;France;Germany;Japan;
Shire Human Genetics Therapies Inc
2012 Phase 1;Phase 2 EUCTR2011-002044-28-DE Australia;Brazil;Denmark;France;Germany;Japan;
2011 Phase 1;Phase 2 EUCTR2011-002044-28-DK Australia;Brazil;Denmark;France;Germany;Japan;
- Phase 1;Phase 2 EUCTR2012-003775-20-FR Argentina;Australia;Brazil;Czech Republic;Denmark;France;Germany;United Kingdom;
HGT-1111
Shire
2008 Phase 2 NCT00681811 Denmark;
Shire Human Genetic Therapies Inc
2010 Phase 2 EUCTR2008-000084-41-BE Belgium;Denmark;France;Italy;
2009 Phase 2 EUCTR2008-000084-41-IT Belgium;Denmark;France;Italy;
2009 - EUCTR2008-000084-41-FR Belgium;Denmark;France;Italy;
2008 Phase 2 EUCTR2008-000084-41-DK Belgium;Denmark;France;Italy;
HGT-1410
SHIRE HUMAN GENETIC THERAPIES, INC
2015 Phase 2 EUCTR2014-003960-20-IT France;Germany;Italy;Netherlands;Spain;United Kingdom;United States;
Shire
2015 Phase 2 NCT02350816 France;Germany;Italy;Netherlands;Spain;United Kingdom;United States;
Shire Human Genetic Therapies, Inc
2014 Phase 2 EUCTR2013-003450-24-NL Argentina;France;Germany;Italy;Netherlands;Spain;United Kingdom;United States;
2014 Phase 2 EUCTR2013-003450-24-IT France;Germany;Italy;Netherlands;Spain;United Kingdom;
2014 Phase 2 EUCTR2013-003450-24-GB Argentina;Brazil;France;Germany;Italy;Netherlands;Spain;United Kingdom;United States;
2014 Phase 2 EUCTR2013-003450-24-ES Argentina;France;Germany;Italy;Netherlands;Spain;United Kingdom;United States;
2014 Phase 2 EUCTR2013-003450-24-DE Argentina;Brazil;France;Germany;Italy;Netherlands;Spain;United Kingdom;United States;
2011 Phase 1;Phase 2 EUCTR2010-021348-16-NL Netherlands;United Kingdom;
2011 Phase 1;Phase 2 EUCTR2010-021348-16-GB Netherlands;United Kingdom;
- Phase 2 EUCTR2013-003450-24-FR Argentina;Brazil;France;Germany;Italy;Netherlands;Spain;United Kingdom;United States;
Shire Human Genetic Therapies, Inc.
2015 Phase 2 EUCTR2014-003960-20-NL France;Germany;Italy;Netherlands;Spain;United Kingdom;United States;
2015 Phase 2 EUCTR2014-003960-20-GB France;Germany;Italy;Netherlands;Spain;United Kingdom;United States;
2015 Phase 2 EUCTR2014-003960-20-DE France;Germany;Italy;Netherlands;Spain;United Kingdom;United States;
Shire Human Genetic Therapies, Inc. (Shire HGT)
2010 Phase 1;Phase 2 EUCTR2009-015984-15-GB United Kingdom;
Shire human Genetic Therapies, Inc.
2015 Phase 2 EUCTR2014-003960-20-ES Argentina;France;Germany;Italy;Netherlands;Spain;United Kingdom;United States;
- Phase 2 EUCTR2014-003960-20-FR Argentina;France;Germany;Italy;Netherlands;Spain;United Kingdom;United States;
HGT-2310
Shire HGT Inc
2013 Phase 2;Phase 3 EUCTR2013-002885-38-GB Argentina;Australia;Canada;Colombia;France;Germany;Mexico;Spain;United Kingdom;United States;
2013 Phase 2;Phase 3 EUCTR2013-002885-38-ES Argentina;Mexico;Spain;United Kingdom;United States;
- Phase 2;Phase 3 EUCTR2013-002885-38-FR Argentina;Australia;France;Germany;Mexico;Spain;United Kingdom;United States;
Shire Human Genetic Therapies, Inc.
2015 Phase 2;Phase 3 EUCTR2014-004143-13-GB Argentina;Australia;Canada;Colombia;France;Mexico;Spain;United Kingdom;United States;
2015 - EUCTR2014-004143-13-ES Argentina;Canada;Colombia;Mexico;Spain;United Kingdom;United States;
- Phase 2;Phase 3 EUCTR2014-004143-13-FR Argentina;Australia;Canada;Colombia;France;Mexico;Spain;United Kingdom;United States;
HYDROXYPROPYLBETADEX
Cyclo Therapeutics, Inc.
2022 Phase 3 EUCTR2020-003136-25-PL Australia;France;Germany;Israel;Italy;Poland;United Kingdom;United States;
2022 Phase 3 EUCTR2020-003136-25-DE Australia;France;Germany;Israel;Italy;Poland;United Kingdom;United States;
2021 Phase 3 EUCTR2020-003136-25-FR Australia;France;Israel;United Kingdom;United States;
- Phase 3 EUCTR2020-003136-25-IT Australia;France;Germany;Israel;Italy;Poland;Spain;United Kingdom;United States;
Hematopoetic Stem Cell Transplantation
University of Pittsburgh
2012 - NCT03333200 United States;
Hematopoietic Stem Cell Transplantation
Masonic Cancer Center, University of Minnesota
2006 Phase 2 NCT00383448 United States;
Hematopoietic stem cell infusion
Talaris Therapeutics Inc.
2011 Phase 1/Phase 2 NCT01372228 United States;
Hematopoietic stem cell transplantation
Masonic Cancer Center, University of Minnesota
2012 - NCT01626092 United States;
Heparán N sulfatasa humana recombinante (rhHNS)
Shire Human Genetic Therapies, Inc
2014 Phase 2 EUCTR2013-003450-24-ES Argentina;France;Germany;Italy;Netherlands;Spain;United Kingdom;United States;
HuCNS-SC
StemCells, Inc.
2010 Phase 1 NCT01238315 United States;
Human Glucocerebrosidase (prGCD)
Protalix
2005 Phase 1 NCT00258778 Israel;
Human Placental Derived Stem Cell
New York Medical College
2013 Phase 1 NCT01586455 United States;
Hunterase
Green Cross Corporation
2012 Phase 3 NCT01645189 Korea, Republic of;
Hydroxypropyl-beta-cyclodextrin
CTD Holdings, Inc.
2016 Phase 1;Phase 2 EUCTR2015-005761-23-GB Italy;Sweden;United Kingdom;
Cyclo Therapeutics, Inc.
2021 Phase 3 NCT04860960 United States;
2019 Phase 1 NCT03893071 United States;
2017 Phase 1;Phase 2 EUCTR2015-005761-23-SE Italy;Sweden;United Kingdom;
2017 Phase 1/Phase 2 NCT02912793 Israel;Sweden;United Kingdom;
2017 Phase 1 NCT02939547 United States;
Hydroxypropyl-ß-cyclodextrin
Cyclo Therapeutics, Inc.
2019 Phase 1 NCT03893071 United States;
Hydroxyurea
Masonic Cancer Center, University of Minnesota
2006 Phase 2 NCT00383448 United States;
I2S
TKT INC
2005 - EUCTR2004-002743-27-IT Germany;Italy;Spain;Sweden;United Kingdom;
I2S-IT
Shire HGT Inc
- - EUCTR2011-000212-25-Outside-EU/EEA Canada;United States;
Shire HGT, Inc
2011 Phase 1;Phase 2 EUCTR2010-020048-36-GB United Kingdom;United States;
IB1001
IntraBio Inc
2019 Phase 2 NCT03759665 Germany;Spain;United Kingdom;United States;
2019 Phase 2 NCT03759639 Germany;Slovakia;Spain;United Kingdom;United States;
IntraBio Ltd
2022 Phase 3 EUCTR2021-005356-10-SK Australia;Czechia;Germany;Netherlands;Slovakia;Switzerland;United Kingdom;United States;
2022 Phase 3 EUCTR2021-005356-10-DE Australia;Czechia;Germany;Netherlands;Slovakia;Spain;Switzerland;United Kingdom;United States;
2019 Phase 2 EUCTR2018-004406-25-GB Germany;Spain;United Kingdom;United States;
2019 Phase 2 EUCTR2018-004406-25-ES Germany;Spain;United Kingdom;United States;
2019 Phase 2 EUCTR2018-004406-25-DE Germany;Spain;United Kingdom;United States;
2019 Phase 2 EUCTR2018-004331-71-SK Germany;Slovakia;Spain;United Kingdom;United States;
2019 Phase 2 EUCTR2018-004331-71-GB Germany;Slovakia;Spain;United Kingdom;United States;
2019 Phase 2 EUCTR2018-004331-71-ES Germany;Slovakia;Spain;United Kingdom;United States;
2019 Phase 2 EUCTR2018-004331-71-DE Germany;Slovakia;Spain;United Kingdom;United States;
- Phase 3 EUCTR2021-005356-10-NL Australia;Czechia;Germany;Netherlands;Slovakia;Spain;Switzerland;United Kingdom;United States;
IDURONATE-2-SULFATASE FUSED TO A FC POLYPEPTIDE THAT BINDS TO THE HUMAN TRANSFERRIN RECEPTOR
Denali Therapeutics Inc.
2021 Phase 1;Phase 2 EUCTR2019-004909-27-NL Germany;Italy;Netherlands;United Kingdom;United States;
2020 Phase 1;Phase 2 EUCTR2019-004909-27-GB Germany;Italy;Netherlands;United Kingdom;United States;
IMD Preparative Regimen
Masonic Cancer Center, University of Minnesota
2014 Phase 2 NCT02171104 United States;
INN Not available
Genzyme Europe B.V.
2010 - EUCTR2008-005223-28-ES Czech Republic;France;Germany;Italy;Netherlands;Spain;United Kingdom;
IRT Laronidase
Masonic Cancer Center, University of Minnesota
2008 Phase 1 NCT00638547 United States;
ISU302
ISU Abxis Co., Ltd.
2011 Phase 3 NCT02770625 -
2011 Phase 3 NCT01161914 -
2010 Phase 1 NCT01881633 -
IV migalastat HCl
Amicus Therapeutics
2014 Phase 1 NCT02082327 Netherlands;
IVA 336
Inventiva S.A.
2019 Phase 2 EUCTR2017-002158-35-PT France;Germany;Portugal;United Kingdom;
2018 Phase 2 EUCTR2017-002158-35-DE France;Germany;Portugal;United Kingdom;
2017 Phase 2 EUCTR2017-002158-35-GB France;Germany;Portugal;United Kingdom;
- Phase 2 EUCTR2017-002158-35-FR France;Germany;Portugal;United Kingdom;
Iduronate-2-sulfatase enzyme replacement therapy
Shire
2003 Phase 2/Phase 3 NCT00069641 Brazil;Germany;United Kingdom;United States;
Idursulfase
GlaxoSmithKline
2012 - NCT01602601 Japan;
Shire
2008 - NCT00882921 Brazil;United Kingdom;United States;
2007 Phase 4 NCT00607386 Brazil;Poland;Taiwan;
2004 Phase 2/Phase 3 NCT00630747 Brazil;Canada;France;Germany;Italy;Romania;Spain;Sweden;United Kingdom;United States;
TKT INC
2005 - EUCTR2004-002743-27-IT Germany;Italy;Spain;Sweden;United Kingdom;
Idursulfase (I2S)
Shire Human Genetic Therapies INC.
2005 - EUCTR2004-002743-27-SE Germany;Italy;Spain;Sweden;United Kingdom;
2004 - EUCTR2004-002743-27-DE Germany;Italy;Spain;Sweden;United Kingdom;
Shire Human Genetic Therapies, Inc. (Shire HGT)
- Phase 4 EUCTR2007-006044-22-PL Poland;
TKT Inc.
2005 Phase 3 EUCTR2004-002743-27-GB Germany;Italy;Spain;Sweden;United Kingdom;
2005 - EUCTR2004-002743-27-ES Germany;Italy;Spain;Sweden;United Kingdom;
Idursulfase (I2S)-IT
Shire HGT, Inc
2011 Phase 1;Phase 2 EUCTR2010-020048-36-GB United Kingdom;United States;
Idursulfase IT (1 mg)
Shire
2009 Phase 1/Phase 2 NCT00920647 United Kingdom;United States;
Idursulfase IT (10 mg)
Shire
2009 Phase 1/Phase 2 NCT00920647 United Kingdom;United States;
Idursulfase IT (30 mg)
Shire
2009 Phase 1/Phase 2 NCT00920647 United Kingdom;United States;
Idursulfase beta
Green Cross Corporation
2016 Phase 2 NCT02663024 -
Idursulfase(12S)-IT
Shire HGT Inc
2012 Phase 1;Phase 2 EUCTR2011-000212-25-GB Canada;United Kingdom;United States;
Idursulfase(I2S)-IT
Shire HGT Inc
- - EUCTR2011-000212-25-Outside-EU/EEA Canada;United States;
Idursulfase-IT
Shire
2015 Phase 2/Phase 3 NCT02412787 Argentina;Australia;Canada;Colombia;France;Mexico;Spain;United Kingdom;United States;
2014 Phase 2/Phase 3 NCT02055118 Argentina;Australia;Canada;Colombia;France;Mexico;Spain;United Kingdom;United States;
2010 Phase 1/Phase 2 NCT01506141 Canada;United Kingdom;United States;
Shire HGT Inc
2013 Phase 2;Phase 3 EUCTR2013-002885-38-GB Argentina;Australia;Canada;Colombia;France;Germany;Mexico;Spain;United Kingdom;United States;
2013 Phase 2;Phase 3 EUCTR2013-002885-38-ES Argentina;Mexico;Spain;United Kingdom;United States;
2012 Phase 1;Phase 2 EUCTR2011-000212-25-GB Canada;United Kingdom;United States;
- Phase 2;Phase 3 EUCTR2013-002885-38-FR Argentina;Australia;France;Germany;Mexico;Spain;United Kingdom;United States;
- - EUCTR2011-000212-25-Outside-EU/EEA Canada;United States;
Shire HGT, Inc
2011 Phase 1;Phase 2 EUCTR2010-020048-36-GB United Kingdom;United States;
Shire Human Genetic Therapies, Inc.
2015 Phase 2;Phase 3 EUCTR2014-004143-13-GB Argentina;Australia;Canada;Colombia;France;Mexico;Spain;United Kingdom;United States;
2015 - EUCTR2014-004143-13-ES Argentina;Canada;Colombia;Mexico;Spain;United Kingdom;United States;
- Phase 2;Phase 3 EUCTR2014-004143-13-FR Argentina;Australia;Canada;Colombia;France;Mexico;Spain;United Kingdom;United States;
Imiglucerase
Genzyme Europe BV
2010 - EUCTR2008-005223-28-IT Czech Republic;France;Germany;Italy;Netherlands;Spain;United Kingdom;
Genzyme, a Sanofi Company
2022 Phase 3 NCT05222906 -
2017 Phase 2 NCT02843035 Germany;Japan;United Kingdom;United States;
2009 Phase 3 NCT00943111 Argentina;Australia;Brazil;Canada;Czech Republic;Egypt;France;Germany;Italy;Netherlands;Russian Federation;Spain;Turkey;United Kingdom;United States;
ISTITUTO GIANNINA GASLINI
2009 - EUCTR2007-005516-61-IT Italy;
SHIRE HUMAN GENETIC THERAPIES, INC.
2008 - EUCTR2007-002840-21-IT Italy;Spain;United Kingdom;
Sanofi-aventis recherche & développement
- Phase 3 EUCTR2021-005402-10-FR Argentina;Brazil;Canada;China;Egypt;France;Germany;Hungary;Japan;Taiwan;Turkey;United Kingdom;United States;
- Phase 3 EUCTR2021-005402-10-DE Argentina;Brazil;Canada;China;Egypt;France;Germany;Hungary;Japan;Taiwan;Turkey;United Kingdom;United States;
Shire
2008 Phase 3 NCT00553631 Argentina;India;Israel;Paraguay;Russian Federation;Spain;Tunisia;United Kingdom;United States;
University Research Foundation for Lysosomal Storage Diseases, Inc.
2010 - NCT01136304 United States;
Imiglucerase GZ437843
Sanofi
2018 Phase 3 NCT03485677 Argentina;Canada;France;Italy;Japan;Russian Federation;Spain;Sweden;Turkey;United Kingdom;
Imiglucerasi
SHIRE HUMAN GENETIC THERAPIES, INC.
2008 - EUCTR2007-002840-21-IT Italy;Spain;United Kingdom;
Imiglucérase (drug) pharmacokinetics
University Hospital, Clermont-Ferrand
2012 Phase 2 NCT01951989 France;
Inactive Reminder Capsules
Amicus Therapeutics, Inc.
2015 Phase 3 EUCTR2014-002701-38-GB Argentina;Australia;Austria;Belgium;Brazil;Canada;Denmark;Egypt;France;Italy;Japan;Spain;Turkey;United Kingdom;United States;
2015 Phase 3 EUCTR2014-002701-38-DK Argentina;Australia;Austria;Belgium;Brazil;Canada;Denmark;Egypt;France;Italy;Japan;Spain;Turkey;United Kingdom;United States;
2015 Phase 3 EUCTR2014-002701-38-BE Argentina;Australia;Austria;Belgium;Brazil;Canada;Denmark;Egypt;France;Italy;Japan;Spain;Turkey;United Kingdom;United States;
2014 Phase 3 EUCTR2014-002701-38-AT Argentina;Australia;Austria;Belgium;Brazil;Canada;Denmark;Egypt;France;Italy;Japan;Spain;Turkey;United Kingdom;United States;
2012 - EUCTR2010-022636-37-DE Argentina;Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Hungary;Israel;Italy;Japan;Mexico;Poland;Russian Federation;Slovakia;Switzerland;Taiwan;Turkey;United Kingdom;United States;
2011 Phase 3 EUCTR2010-022636-37-GR Argentina;Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Hungary;Israel;Italy;Japan;Mexico;Poland;Russian Federation;Slovakia;Switzerland;Taiwan;Turkey;United Kingdom;United States;
2011 - EUCTR2010-022636-37-GB Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;United Kingdom;United States;
2011 - EUCTR2010-022636-37-DK Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;United Kingdom;United States;
2011 - EUCTR2010-022636-37-BE Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;United Kingdom;United States;
2011 - EUCTR2010-022636-37-AT Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;United Kingdom;United States;
Inactive Reminder Product
Amicus Therapeutics, Inc.
2010 - EUCTR2009-013459-31-DK Argentina;Australia;Belgium;Brazil;Canada;Denmark;Egypt;France;Germany;Italy;Netherlands;South Africa;Spain;Turkey;United Kingdom;United States;
2009 Phase 3 EUCTR2009-013459-31-GB Argentina;Australia;Belgium;Brazil;Canada;Denmark;Egypt;France;Germany;Italy;Netherlands;South Africa;Spain;Turkey;United Kingdom;United States;
Intrathecal recombinant human alpha iduronidase
agnes chen
2014 - NCT02232477 United States;
Intraventricular Access Device
BioMarin Pharmaceutical
2015 Phase 1/Phase 2 NCT02485899 Germany;Italy;United Kingdom;United States;
Intraventricular access device
BioMarin Pharmaceutical
2016 Phase 2 NCT02678689 Germany;Italy;United Kingdom;United States;
Iohexol
Genzyme, a Sanofi Company
2013 - NCT01839526 Argentina;Austria;Belgium;Brazil;Canada;Finland;France;Hungary;Norway;Poland;Spain;Taiwan;United Kingdom;United States;
2012 - EUCTR2012-001966-14-ES Canada;Netherlands;Norway;Spain;United Kingdom;United States;
Isofagamine tartrate
Amicus Therapeutics, Inc.
2007 - EUCTR2007-003359-35-GB Germany;United Kingdom;
2007 - EUCTR2007-003359-35-DE Germany;United Kingdom;
JR-141
JCR Pharmaceuticals Co., Ltd.
2019 Phase 2/Phase 3 NCT04348136 Japan;
2019 Phase 2 NCT03708965 Brazil;
2018 Phase 2/Phase 3 NCT03568175 Japan;
2018 Phase 2 NCT03359213 Brazil;
2017 Phase 1/Phase 2 NCT03128593 Japan;
- Phase 3 EUCTR2020-003200-14-FR Brazil;France;Germany;United Kingdom;United States;
- Phase 3 EUCTR2020-003200-14-DE Brazil;France;Germany;United Kingdom;United States;
JR-141 or Idursulfase
JCR Pharmaceuticals Co., Ltd.
2022 Phase 3 NCT04573023 United States;
JR-171
JCR Pharmaceuticals Co., Ltd.
2021 Phase 1/Phase 2 NCT04453085 Brazil;Japan;
2020 Phase 1/Phase 2 NCT04227600 Brazil;Japan;United States;
Kanuma
Alexion Pharmaceuticals, Inc.
2013 Phase 3 EUCTR2011-002750-31-DE Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Japan;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;United States;
LYS-GM101
LYSOGENE
2021 Phase 1/Phase 2 NCT04273269 France;United Kingdom;United States;
Lysogene
- Phase 1;Phase 2 EUCTR2019-004949-32-FR France;United Kingdom;United States;
LYS-SAF302
Lysogene SA
2019 Phase 2;Phase 3 EUCTR2018-000195-15-NL France;Germany;Netherlands;United Kingdom;United States;
2019 Phase 2;Phase 3 EUCTR2018-000195-15-GB France;Germany;Netherlands;United Kingdom;United States;
2019 Phase 2;Phase 3 EUCTR2018-000195-15-DE France;Germany;Netherlands;United Kingdom;United States;
2018 Phase 2;Phase 3 EUCTR2018-000195-15-FR France;Germany;Netherlands;United Kingdom;United States;
Lamazym
Chiesi Farmaceutici S.p.A.
2013 Phase 3 NCT01908725 Denmark;
2013 Phase 3 NCT01908712 France;
Zymenex A/S
2015 Phase 3 NCT02478840 Denmark;
2013 Phase 3 EUCTR2012-000979-17-ES Belgium;Denmark;Germany;Spain;Sweden;
2013 - EUCTR2012-000979-17-SE Belgium;Denmark;Germany;Spain;Sweden;
2013 - EUCTR2012-000979-17-BE Belgium;Denmark;Germany;Spain;Sweden;
2012 Phase 3 NCT01681953 Denmark;France;Germany;Poland;United Kingdom;
2012 Phase 2 NCT01681940 Belgium;Denmark;Spain;United Kingdom;
2012 Phase 2 EUCTR2011-004355-40-DK Belgium;Denmark;Germany;Spain;United Kingdom;
2012 - EUCTR2012-000979-17-DK Belgium;Denmark;Germany;Spain;Sweden;
2012 - EUCTR2012-000979-17-DE Belgium;Denmark;Germany;Spain;Sweden;
2012 - EUCTR2011-004355-40-GB Belgium;Denmark;Germany;Spain;United Kingdom;
2012 - EUCTR2011-004355-40-ES Belgium;Denmark;Germany;Spain;United Kingdom;
2012 - EUCTR2011-004355-40-DE Belgium;Denmark;Germany;Spain;United Kingdom;
2012 - EUCTR2011-004355-40-BE Belgium;Denmark;Germany;Spain;United Kingdom;
2011 Phase 2 NCT01285700 Denmark;
2010 Phase 1 NCT01268358 -
2010 - EUCTR2010-022085-26-DK Denmark;
2010 - EUCTR2010-022084-36-DK Denmark;
Laronidase
Genzyme Europe BV
2010 - EUCTR2007-007003-33-IT Italy;
Genzyme, a Sanofi Company
2021 Phase 4 NCT05134571 China;
2008 Phase 1/Phase 2 NCT00741338 Brazil;Russian Federation;Ukraine;
2004 Phase 4 NCT00144768 United States;
Masonic Cancer Center, University of Minnesota
2012 Phase 1 NCT01173016 United States;
2012 - NCT01572636 United States;
Patricia I. Dickson, M.D.
2009 - NCT00852358 United States;
2008 Phase 1 NCT00786968 Finland;United States;
2005 Phase 1 NCT00215527 Finland;United States;
Laronidase ERT
Masonic Cancer Center, University of Minnesota
2004 Phase 2 NCT00176891 United States;
Ledipasvir/Sofosbuvir
Mansoura University Children Hospital
2018 Phase 4 NCT03721627 Egypt;
Lentivirus Alpha-gal A transduced stem cells
University Health Network, Toronto
2016 Phase 1 NCT02800070 Canada;
Lentivirus-mediated delivery of ARSA to the CNS.
Shenzhen Geno-Immune Medical Institute
2018 - NCT03725670 China;
Leucovorin
The Hospital for Sick Children
2009 Phase 1/Phase 2 NCT01102686 Canada;
Lipasa ácida lisosómica, esterasa, colesterol (gen humano LIPA), lipasa ácida lisosómica (gen humano LIPA)
Synageva BioPharma Corp.
2014 Phase 2 EUCTR2011-004287-30-ES Australia;Belgium;Brazil;Canada;Croatia;Denmark;Germany;Italy;Japan;Mexico;Netherlands;Poland;Russian Federation;Spain;Turkey;United Kingdom;United States;
Lisinopril
Salford Royal NHS Foundation Trust
2009 Phase 4 EUCTR2007-007482-21-GB United Kingdom;
Lopace
Salford Royal NHS Foundation Trust
2009 Phase 4 EUCTR2007-007482-21-GB United Kingdom;
Losartan
Hospital de Clinicas de Porto Alegre
2018 Phase 2 NCT03632213 Brazil;
Losartan and other angiotensin receptor blockers
University of Alabama at Birmingham
2007 - NCT00446862 Germany;Slovenia;United States;
Lucerastat
IDORSIA PHARMACEUTICALS LTD
2020 Phase 3 EUCTR2018-002210-12-IT Australia;Austria;Belgium;Canada;France;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;United Kingdom;United States;
2020 Phase 3 EUCTR2017-003369-85-IT Australia;Austria;Belgium;Canada;Finland;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;Switzerland;United Kingdom;United States;
Idorsia Pharmaceuticals Japan Ltd
2019 Phase 3 JPRN-JapicCTI-194975 Japan;
2019 Phase 3 JPRN-JapicCTI-194731 Japan;
Idorsia Pharmaceuticals Ltd
2021 Phase 3 EUCTR2018-002210-12-NO Australia;Austria;Belgium;Canada;France;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;United Kingdom;United States;
2021 Phase 3 EUCTR2018-002210-12-FR Austria;Belgium;Canada;Czech Republic;France;Germany;Netherlands;Poland;United Kingdom;United States;
2020 Phase 3 EUCTR2018-002210-12-BE Australia;Austria;Belgium;Canada;France;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;United Kingdom;United States;
2020 Phase 3 EUCTR2017-003369-85-NO Australia;Austria;Belgium;Canada;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;Switzerland;United Kingdom;United States;
2020 Phase 3 EUCTR2017-003369-85-IE Australia;Austria;Belgium;Canada;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;Switzerland;United Kingdom;United States;
2020 Phase 3 EUCTR2017-003369-85-ES Australia;Austria;Belgium;Canada;Germany;Ireland;Italy;Netherlands;Norway;Poland;Russian Federation;Spain;Switzerland;United Kingdom;United States;
2019 Phase 3 EUCTR2018-002210-12-NL Australia;Austria;Belgium;Canada;France;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;United Kingdom;United States;
2019 Phase 3 EUCTR2018-002210-12-GB Australia;Austria;Belgium;Canada;Germany;Ireland;Netherlands;Poland;United Kingdom;United States;
2019 Phase 3 EUCTR2018-002210-12-DE Australia;Austria;Belgium;Canada;France;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;United Kingdom;United States;
2019 Phase 3 EUCTR2018-002210-12-AT Australia;Austria;Belgium;Canada;France;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;United Kingdom;United States;
2018 Phase 3 EUCTR2017-003369-85-NL Australia;Austria;Belgium;Canada;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;Switzerland;United Kingdom;United States;
2018 Phase 3 EUCTR2017-003369-85-GB Australia;Austria;Belgium;Canada;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;Switzerland;United Kingdom;United States;
2018 Phase 3 EUCTR2017-003369-85-AT Australia;Austria;Belgium;Canada;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;Switzerland;United Kingdom;United States;
Idorsia Pharmaceuticals Ltd.
2018 Phase 3 NCT03737214 Australia;Austria;Belgium;Canada;Czechia;France;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;Switzerland;United Kingdom;United States;
2018 Phase 3 NCT03425539 Australia;Austria;Belgium;Canada;Czechia;France;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;Switzerland;United Kingdom;United States;
2015 Phase 1 NCT02930655 Germany;
Lumbar puncture
National Human Genome Research Institute (NHGRI)
2019 Phase 1/Phase 2 NCT03952637 United States;
Lysodase
National Institute of Mental Health (NIMH)
1993 Phase 1 NCT00001410 United States;
Lysosomal acid lipase, Esterase cholesterol (human gene LIPA), Lysosomal acid lipase (human gene LIPA)
Synageva BioPharma Corp.
2014 Phase 2 EUCTR2014-000533-22-IT Finland;Italy;United Kingdom;United States;
Lysosomal acid lipase, Esterase, cholesterol (human gene LIPA), Lysosomal acid lipase (human gene LIPA)
Alexion Pharmaceuitcals Inc
2014 Phase 2 EUCTR2011-004287-30-HR Australia;Belgium;Brazil;Canada;Croatia;Denmark;Germany;Italy;Japan;Mexico;Netherlands;Russian Federation;Spain;Turkey;United Kingdom;United States;
2014 Phase 2 EUCTR2011-004287-30-DE Australia;Belgium;Brazil;Canada;Croatia;Denmark;Germany;Italy;Japan;Mexico;Netherlands;Russian Federation;Spain;Turkey;United Kingdom;United States;
Alexion Pharmaceuticals Inc
2015 Phase 2 EUCTR2014-000533-22-FI Finland;Italy;United Kingdom;United States;
2015 Phase 2 EUCTR2011-004287-30-NL Australia;Belgium;Brazil;Canada;Croatia;Denmark;Germany;Italy;Mexico;Netherlands;Russian Federation;Spain;Turkey;United Kingdom;United States;
2015 Phase 2 EUCTR2011-004287-30-GB Australia;Belgium;Brazil;Canada;Croatia;Denmark;Germany;Italy;Japan;Mexico;Netherlands;Poland;Russian Federation;Spain;Turkey;United Kingdom;United States;
2014 Phase 2 EUCTR2014-000533-22-GB Finland;Italy;United Kingdom;United States;
Alexion Pharmaceuticals inc
2014 Phase 2 EUCTR2011-004287-30-DK Australia;Belgium;Brazil;Canada;Croatia;Denmark;Germany;Italy;Japan;Mexico;Netherlands;Poland;Russian Federation;Spain;Turkey;United Kingdom;United States;
Alexion Pharmaceuticals, Inc.
2015 Phase 3 EUCTR2011-002750-31-CZ Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Japan;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;United States;
2013 Phase 3 EUCTR2011-002750-31-GB Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Japan;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;United States;
2013 Phase 3 EUCTR2011-002750-31-DE Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Japan;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;United States;
- Phase 3 EUCTR2011-002750-31-PL Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Japan;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;United States;
Synageva BioPharma Corp.
2014 Phase 2 EUCTR2011-004287-30-IT Australia;Belgium;Brazil;Canada;Croatia;Denmark;Germany;Italy;Japan;Mexico;Netherlands;Poland;Russian Federation;Spain;Turkey;United Kingdom;United States;
2014 Phase 2 EUCTR2011-004287-30-BE Australia;Belgium;Brazil;Canada;Croatia;Denmark;Germany;Italy;Japan;Mexico;Netherlands;Russian Federation;Spain;Turkey;United Kingdom;United States;
Synageva BioPharma Corporation
2013 Phase 3 EUCTR2011-002750-31-IT Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;
2013 Phase 3 EUCTR2011-002750-31-HR Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;
2013 Phase 3 EUCTR2011-002750-31-GR Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;
2013 Phase 3 EUCTR2011-002750-31-ES Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;
- Phase 3 EUCTR2011-002750-31-FR Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;
MABTHERA - 2 FIALE 100 MG 10 ML
OSPEDALE SAN RAFFAELE
2018 Phase 1 EUCTR2017-002430-23-IT Italy;
MERCAPTAMINE BITARTRATE
HOSPICES CIVILS DE LYON
2010 - EUCTR2010-020098-18-FR France;
MERCAPTAMINE HYDROCHLORIDE
Fundació Hospital Universitari Vall d'Hebron - Institut de Recerca (VHIR)
2014 - EUCTR2013-003228-35-ES Spain;
MIGALASTAT HYDROCHLORIDE
Amicus Therapeutics UK, Ltd
2020 Phase 3 EUCTR2017-000146-21-IT Australia;Germany;Italy;Spain;United Kingdom;United States;
Amicus Therapeutics, UK Limited
2020 Phase 3 EUCTR2019-000222-21-GB United Kingdom;United States;
Amicus Therapeutics, UK Ltd
2019 Phase 3 EUCTR2017-000146-21-GB Australia;Germany;Italy;Spain;United Kingdom;United States;
2019 Phase 3 EUCTR2017-000146-21-ES Australia;Germany;Italy;Spain;United Kingdom;United States;
- Phase 3 EUCTR2017-000146-21-DE Australia;Germany;Italy;Spain;United Kingdom;United States;
MIGLUSTAT (ZAVESCA)
INSTITUTO ARAGONÉS DE CIENCIAS DE LA SALUD
2008 - EUCTR2007-007164-20-ES Spain;
MOZOBIL - 20 MG/ML - SOLUZIONE INIETTABILE - USO SOTTOCUTANEO - F
OSPEDALE SAN RAFFAELE
2018 Phase 1 EUCTR2017-002430-23-IT Italy;
MPS
Mackay Memorial Hospital
2021 - NCT04958070 Taiwan;
National MPS Society
2011 - NCT01675674 United States;
MRI with contrast agent injection
University Hospital, Bordeaux
2016 - NCT03123523 France;
MRI without contrast agent injection
University Hospital, Bordeaux
2016 - NCT03123523 France;
MYELOSTIM - 34 1 FLACONCINO LIOFILIZZATO + SIRINGA PRERIEMPITA SOLVENTE 1 ML
OSPEDALE SAN RAFFAELE
2018 Phase 1 EUCTR2017-002430-23-IT Italy;
Magnetic Resonance Imaging
University Hospital, Rouen
2017 - NCT02956954 France;
Mechanical stimulation with Von Frey filaments
Universitaire Ziekenhuizen Leuven
2014 Phase 0 NCT02533076 Belgium;
Medication to suppress the immune system
StemCells, Inc.
2006 Phase 1 NCT00337636 United States;
Melphalan
Masonic Cancer Center, University of Minnesota
2012 - NCT01626092 United States;
2006 Phase 2 NCT00383448 United States;
Mepsevii
Ultragenyx Pharmaceutical Inc
2018 - NCT03775174 -
Mercaptamina
Recordati Rare Diseases
2020 Phase 3 EUCTR2018-002984-24-IT Belgium;France;Germany;Italy;United Kingdom;
Mercaptamine
Recordati Rare Diseases
2020 Phase 3 NCT04125927 France;
Mercaptamine hydrochloride
Orphan Europe SARL
2013 - EUCTR2009-012564-13-FR France;
RECORDATI Rare Diseases SARL
2019 Phase 3 EUCTR2018-002984-24-GB France;United Kingdom;
Mercaptamine, mercamine, 2-aminoethanethiol
Orphan Europe SARL
2008 - EUCTR2007-006024-35-FR France;
Metazym
Shire Pharmaceutical Ireland Limited
2008 - EUCTR2007-006345-40-DK Denmark;
Shire Pharmaceuticals Ireland Limited
2008 Phase 2 EUCTR2007-007165-20-DK Denmark;
2006 - EUCTR2006-005341-11-DK Denmark;
Methotrexate
Central Manchester University Hospitals NHS Foundation Trust
2015 Phase 3 EUCTR2015-003031-35-GB United Kingdom;
Methylprednisolone
National Human Genome Research Institute (NHGRI)
2019 Phase 1/Phase 2 NCT03952637 United States;
Prevail Therapeutics
2021 Phase 1/Phase 2 NCT04411654 United Kingdom;United States;
Migalastat
Amicus Therapeutics
2020 - NCT04252066 United States;
Manchester University NHS Foundation Trust
2019 - NCT03949920 United Kingdom;
SANOFI-AVENTIS RECHERCHE E DEVELOPPEMENT
2022 Phase 3 EUCTR2021-002320-20-IT Austria;Canada;China;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Turkey;United Kingdom;United States;
sanofi-aventis recherche et développement
- Phase 3 EUCTR2021-002320-20-ES Austria;Canada;China;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Turkey;United Kingdom;United States;
Migalastat HCl
Amicus Therapeutics
2022 Phase 3 NCT04020055 Belgium;France;Italy;Spain;United Kingdom;United States;
2019 Phase 3 NCT04049760 Spain;United Kingdom;United States;
2018 Phase 3 NCT03500094 Spain;United Kingdom;United States;
2014 Phase 3 NCT02194985 Argentina;Australia;Austria;Belgium;Brazil;Canada;Denmark;Egypt;France;Italy;Japan;Spain;Turkey;United Kingdom;United States;
2011 Phase 2 NCT01196871 Australia;Belgium;Canada;France;Netherlands;United Kingdom;United States;
2011 - NCT01476163 Australia;United States;
2007 Phase 2 NCT00526071 Australia;Brazil;France;United Kingdom;United States;
2006 Phase 2 NCT00304512 Australia;Brazil;Canada;France;United Kingdom;United States;
2006 Phase 2 NCT00283959 Australia;Brazil;
2006 Phase 2 NCT00283933 Canada;France;United Kingdom;
2006 Phase 2 NCT00214500 United States;
Migalastat Hydrochloride
Amicus Therapeutics, Inc.
2014 Phase 3 EUCTR2011-004800-40-AT Argentina;Australia;Austria;Belgium;Brazil;Canada;Denmark;Egypt;France;Italy;Spain;Turkey;United Kingdom;United States;
2013 Phase 3 EUCTR2011-004800-40-BE Argentina;Australia;Austria;Belgium;Brazil;Canada;Denmark;Egypt;France;Italy;Spain;Turkey;United Kingdom;United States;
2012 Phase 3 EUCTR2011-004800-40-GB Argentina;Australia;Austria;Belgium;Brazil;Canada;Denmark;Egypt;France;Italy;Spain;Turkey;United Kingdom;United States;
2012 Phase 3 EUCTR2011-004800-40-DK Argentina;Australia;Austria;Belgium;Brazil;Canada;Denmark;Egypt;France;Italy;Spain;Turkey;United Kingdom;United States;
GLAXOSMITHKLINE RESEARCH & DEVELOPMENT LTD.
2012 - EUCTR2011-004800-40-IT Australia;Austria;Belgium;Brazil;Denmark;Egypt;Italy;Spain;Turkey;United Kingdom;United States;
GlaxoSmithKline Research & Development Limited
2012 Phase 3 EUCTR2011-004800-40-ES Australia;Austria;Belgium;Brazil;Denmark;Egypt;France;Italy;Spain;Turkey;United Kingdom;United States;
Migalastat hydrochloride
AMICUS THERAPEUTICS, INC
2010 - EUCTR2009-013459-31-IT Belgium;Denmark;France;Germany;Italy;Netherlands;Spain;United Kingdom;
Amicus Therapeutics
2011 Phase 3 NCT01458119 Argentina;Australia;Austria;Belgium;Brazil;Canada;Denmark;Egypt;France;Italy;Japan;Spain;Turkey;United Kingdom;United States;
2011 Phase 3 NCT01218659 Argentina;Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;Poland;Switzerland;Taiwan;Turkey;United Kingdom;United States;
2009 Phase 3 NCT00925301 Argentina;Australia;Belgium;Brazil;Canada;Chile;Denmark;Egypt;France;Germany;Israel;Italy;Netherlands;Poland;South Africa;Spain;Turkey;United Kingdom;United States;
2005 Phase 2 EUCTR2005-004384-33-GB United Kingdom;
Amicus Therapeutics, Inc.
2012 - EUCTR2010-022709-16-GB Belgium;United Kingdom;
2012 - EUCTR2010-022636-37-DE Argentina;Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Hungary;Israel;Italy;Japan;Mexico;Poland;Russian Federation;Slovakia;Switzerland;Taiwan;Turkey;United Kingdom;United States;
2011 Phase 3 EUCTR2010-022636-37-GR Argentina;Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Hungary;Israel;Italy;Japan;Mexico;Poland;Russian Federation;Slovakia;Switzerland;Taiwan;Turkey;United Kingdom;United States;
2011 Phase 2 EUCTR2010-022709-16-BE Australia;Belgium;Canada;France;Netherlands;Switzerland;United Kingdom;
2011 - EUCTR2010-022636-37-GB Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;United Kingdom;United States;
2011 - EUCTR2010-022636-37-DK Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;United Kingdom;United States;
2011 - EUCTR2010-022636-37-BE Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;United Kingdom;United States;
2011 - EUCTR2010-022636-37-AT Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;United Kingdom;United States;
2010 Phase 3 EUCTR2009-013459-31-ES Belgium;Denmark;France;Germany;Italy;Netherlands;Spain;United Kingdom;
2010 Phase 3 EUCTR2009-013459-31-BE Belgium;Denmark;France;Germany;Italy;Netherlands;Spain;United Kingdom;
2010 - EUCTR2009-013459-31-NL Belgium;Denmark;France;Germany;Italy;Netherlands;Spain;United Kingdom;
2010 - EUCTR2009-013459-31-DK Argentina;Australia;Belgium;Brazil;Canada;Denmark;Egypt;France;Germany;Italy;Netherlands;South Africa;Spain;Turkey;United Kingdom;United States;
2009 Phase 3 EUCTR2009-013459-31-GB Argentina;Australia;Belgium;Brazil;Canada;Denmark;Egypt;France;Germany;Italy;Netherlands;South Africa;Spain;Turkey;United Kingdom;United States;
2009 Phase 3 EUCTR2009-013459-31-FR Belgium;Denmark;France;Germany;Italy;Netherlands;Spain;United Kingdom;
2009 Phase 2 EUCTR2007-001838-13-GB Australia;Brazil;United Kingdom;United States;
2006 Phase 2 EUCTR2006-000181-36-GB United Kingdom;
- - EUCTR2009-013459-31-DE Belgium;Denmark;France;Germany;Italy;Netherlands;Spain;United Kingdom;
Migalastat hydrocloride
AMICUS THERAPEUTICS, INC
2011 - EUCTR2010-022636-37-IT Argentina;Australia;Austria;Belgium;Brazil;Denmark;Germany;Greece;Italy;Japan;Mexico;Russian Federation;Switzerland;Taiwan;Turkey;United Kingdom;United States;
Miglustat
Actelion
2020 Phase 4 NCT03910621 China;
2006 Phase 3 NCT00319046 Australia;Brazil;Canada;Czech Republic;Czechia;France;Germany;Hungary;Italy;Netherlands;Spain;Taiwan;United Kingdom;United States;
2002 Phase 2 NCT00517153 United States;
Actelion Pharmaceuticals Ltd
2007 - EUCTR2005-001651-37-HU Czech Republic;Germany;Hungary;Italy;Spain;United Kingdom;
2006 - EUCTR2005-001651-37-ES Czech Republic;Germany;Hungary;Italy;Spain;United Kingdom;
2006 - EUCTR2005-001651-37-DE Czech Republic;Germany;Hungary;Italy;Spain;United Kingdom;
2006 - EUCTR2005-001651-37-CZ Czech Republic;Germany;Hungary;Italy;Spain;United Kingdom;
2005 Phase 4 EUCTR2005-001651-37-GB Czech Republic;Germany;Hungary;Italy;Spain;United Kingdom;
Actelion Registration Ltd
2006 - EUCTR2005-001651-37-IT Czech Republic;Germany;Hungary;Italy;Spain;United Kingdom;
Dipartimento di Pediatria Universita` di Napoli Federico II
2006 - EUCTR2006-005842-35-IT Italy;
ISTITUTO GIANNINA GASLINI
2009 - EUCTR2007-005516-61-IT Italy;
National Taiwan University Hospital
2015 - NCT02520934 Taiwan;
2008 Phase 3 NCT01760564 Taiwan;
Tehran University of Medical Sciences
2019 Phase 3 NCT03822013 Iran, Islamic Republic of;
The Hospital for Sick Children
2004 Phase 2 NCT00418847 Canada;
University of Minnesota
2015 Phase 4 NCT02030015 United States;
Moss-aGal (recombinant human alpha-galactosidase A produced in moss)
Greenovation Biotech GmbH
2016 Phase 1 NCT02995993 Germany;
Mycophenolate mofetil
Masonic Cancer Center, University of Minnesota
2012 - NCT01626092 United States;
University of Rochester
2011 Phase 2 NCT01399047 United States;
Mycophenylate mofetil
Masonic Cancer Center, University of Minnesota
2006 Phase 2 NCT00383448 United States;
N Acetyl L leucine
IntraBio Ltd
2022 Phase 3 EUCTR2021-005356-10-SK Australia;Czechia;Germany;Netherlands;Slovakia;Switzerland;United Kingdom;United States;
2019 Phase 2 EUCTR2018-004406-25-ES Germany;Spain;United Kingdom;United States;
N acetyl cysteine
University of Sao Paulo
2011 Phase 4 NCT01614431 -
N-Acetyl Cysteine
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
2009 Phase 1/Phase 2 NCT00975689 United States;
N-Acetyl-L Leucine
IntraBio Ltd
- Phase 3 EUCTR2021-005356-10-NL Australia;Czechia;Germany;Netherlands;Slovakia;Spain;Switzerland;United Kingdom;United States;
N-Acetyl-L-Leucine
IntraBio Inc
2022 Phase 3 NCT05163288 -
IntraBio Ltd
2019 Phase 2 EUCTR2018-004406-25-ES Germany;Spain;United Kingdom;United States;
2019 Phase 2 EUCTR2018-004406-25-DE Germany;Spain;United Kingdom;United States;
2019 Phase 2 EUCTR2018-004331-71-SK Germany;Slovakia;Spain;United Kingdom;United States;
N-acetyl-L-leucine
IntraBio Ltd
2022 Phase 3 EUCTR2021-005356-10-SK Australia;Czechia;Germany;Netherlands;Slovakia;Switzerland;United Kingdom;United States;
- Phase 3 EUCTR2021-005356-10-NL Australia;Czechia;Germany;Netherlands;Slovakia;Spain;Switzerland;United Kingdom;United States;
N-acetylcysteine
University of Minnesota
2015 Phase 2 NCT02583672 United States;
2011 Phase 1 NCT01427517 United States;
N-acetylgalactosamine 4-sulfatase
BioMarin Pharmaceutical
2004 Phase 3 NCT00104234 United States;
2003 Phase 3 NCT00067470 United States;
2002 Phase 2 NCT00048711 United States;
2000 Phase 1 NCT00048620 United States;
Naglazyme
BioMarin Pharmaceutical
2006 Phase 4 NCT00299000 France;Portugal;United States;
BioMarin Pharmaceutical Inc.
2007 Phase 4 EUCTR2005-003512-30-FR France;Portugal;
Naglazyme (galsulfase)
BioMarin Pharmaceutical Inc
2007 - EUCTR2005-003512-30-PT France;Portugal;
Naglazyme®
Masonic Cancer Center, University of Minnesota
2016 - NCT02156674 United States;
Non applicable
Inserm
2012 - EUCTR2011-004410-42-FR France;
OGT 918
National Institute of Neurological Disorders and Stroke (NINDS)
2002 Phase 2 NCT00041535 United Kingdom;United States;
OGT918
Actelion Pharmaceuticals Ltd
2007 - EUCTR2005-001651-37-HU Czech Republic;Germany;Hungary;Italy;Spain;United Kingdom;
2006 - EUCTR2005-001651-37-ES Czech Republic;Germany;Hungary;Italy;Spain;United Kingdom;
2006 - EUCTR2005-001651-37-DE Czech Republic;Germany;Hungary;Italy;Spain;United Kingdom;
2006 - EUCTR2005-001651-37-CZ Czech Republic;Germany;Hungary;Italy;Spain;United Kingdom;
2005 Phase 4 EUCTR2005-001651-37-GB Czech Republic;Germany;Hungary;Italy;Spain;United Kingdom;
National Eye Institute (NEI)
2002 Phase 1 NCT00316498 United Kingdom;United States;
OGT923
IDORSIA PHARMACEUTICALS LTD
2020 Phase 3 EUCTR2018-002210-12-IT Australia;Austria;Belgium;Canada;France;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;United Kingdom;United States;
2020 Phase 3 EUCTR2017-003369-85-IT Australia;Austria;Belgium;Canada;Finland;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;Switzerland;United Kingdom;United States;
Idorsia Pharmaceuticals Ltd
2021 Phase 3 EUCTR2018-002210-12-NO Australia;Austria;Belgium;Canada;France;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;United Kingdom;United States;
2021 Phase 3 EUCTR2018-002210-12-FR Austria;Belgium;Canada;Czech Republic;France;Germany;Netherlands;Poland;United Kingdom;United States;
2020 Phase 3 EUCTR2018-002210-12-BE Australia;Austria;Belgium;Canada;France;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;United Kingdom;United States;
2020 Phase 3 EUCTR2017-003369-85-NO Australia;Austria;Belgium;Canada;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;Switzerland;United Kingdom;United States;
2020 Phase 3 EUCTR2017-003369-85-IE Australia;Austria;Belgium;Canada;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;Switzerland;United Kingdom;United States;
2020 Phase 3 EUCTR2017-003369-85-ES Australia;Austria;Belgium;Canada;Germany;Ireland;Italy;Netherlands;Norway;Poland;Russian Federation;Spain;Switzerland;United Kingdom;United States;
2019 Phase 3 EUCTR2018-002210-12-NL Australia;Austria;Belgium;Canada;France;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;United Kingdom;United States;
2019 Phase 3 EUCTR2018-002210-12-GB Australia;Austria;Belgium;Canada;Germany;Ireland;Netherlands;Poland;United Kingdom;United States;
2019 Phase 3 EUCTR2018-002210-12-DE Australia;Austria;Belgium;Canada;France;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;United Kingdom;United States;
2019 Phase 3 EUCTR2018-002210-12-AT Australia;Austria;Belgium;Canada;France;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;United Kingdom;United States;
2018 Phase 3 EUCTR2017-003369-85-NL Australia;Austria;Belgium;Canada;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;Switzerland;United Kingdom;United States;
2018 Phase 3 EUCTR2017-003369-85-GB Australia;Austria;Belgium;Canada;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;Switzerland;United Kingdom;United States;
2018 Phase 3 EUCTR2017-003369-85-AT Australia;Austria;Belgium;Canada;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;Switzerland;United Kingdom;United States;
OSOME
Université de Montréal
2014 - NCT02023086 Canada;
OTL-200 Dispersion for Infusion
Orchard Therapeutics (Europe) Limited
2019 Phase 3 EUCTR2019-002636-82-IT Italy;
Orchard Therapeutics (Europe) Ltd
2017 Phase 3 EUCTR2017-001730-26-IT Italy;
Odiparcil
Inventiva Pharma
2017 Phase 2 NCT03370653 France;Germany;Portugal;United Kingdom;
Inventiva S.A.
2019 Phase 2 EUCTR2017-002158-35-PT France;Germany;Portugal;United Kingdom;
2018 Phase 2 EUCTR2017-002158-35-DE France;Germany;Portugal;United Kingdom;
2017 Phase 2 EUCTR2017-002158-35-GB France;Germany;Portugal;United Kingdom;
- Phase 2 EUCTR2017-002158-35-FR France;Germany;Portugal;United Kingdom;
Olenasufligene relduparvovec
Lysogene SA
2019 Phase 2;Phase 3 EUCTR2018-000195-15-NL France;Germany;Netherlands;United Kingdom;United States;
2018 Phase 2;Phase 3 EUCTR2018-000195-15-FR France;Germany;Netherlands;United Kingdom;United States;
Olipudase alfa (rhASM)
Genzyme Corporation
2015 Phase 2 EUCTR2014-003198-40-GB Brazil;France;Germany;Italy;United Kingdom;United States;
2015 Phase 2 EUCTR2014-003198-40-DE Brazil;France;Germany;Italy;United Kingdom;United States;
Ophthalmology exam
National Human Genome Research Institute (NHGRI)
2019 Phase 1/Phase 2 NCT03952637 United States;
Oral migalastat HCl
Amicus Therapeutics
2014 Phase 1 NCT02082327 Netherlands;
Osteopetrosis Haploidentical Only Preparative Regimen
Masonic Cancer Center, University of Minnesota
2014 Phase 2 NCT02171104 United States;
Osteopetrosis Only Preparative Regimen
Masonic Cancer Center, University of Minnesota
2014 Phase 2 NCT02171104 United States;
Oxygen flow at the optic nerve head measurement
Université de Montréal
2014 - NCT02023086 Canada;
PBGM01
Passage Bio, Inc.
2021 Phase 1/Phase 2 NCT04713475 Brazil;Canada;Turkey;United Kingdom;United States;
PBKR03
Passage Bio, Inc.
2022 Phase 1/Phase 2 NCT04771416 Brazil;Canada;Israel;Netherlands;United Kingdom;United States;
- Phase 1;Phase 2 EUCTR2020-005229-95-NL Brazil;Canada;Israel;Netherlands;United Kingdom;United States;
PENTOSAN POLYSULFATE SODIUM
Multiplex Pharma Holdings LLC
2014 - EUCTR2014-000350-11-DE Germany;
PLX-200
Polaryx Therapeutics, Inc.
2021 Phase 3 NCT04637282 -
PR001
Prevail Therapeutics
2021 Phase 1/Phase 2 NCT04411654 United Kingdom;United States;
PRX-102
PROTALIX LTD
2020 Phase 3 EUCTR2018-001148-67-IT Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Finland;Hungary;Italy;Netherlands;Norway;Slovenia;Spain;Switzerland;Turkey;United Kingdom;United States;
2019 Phase 3 EUCTR2018-001947-30-IT Australia;Belgium;Canada;Czechia;Denmark;Finland;France;Italy;Netherlands;Norway;Spain;Sweden;Taiwan;Turkey;United Kingdom;United States;
2018 Phase 3 EUCTR2017-001528-23-IT Austria;Belgium;Canada;Czech Republic;Czechia;Denmark;Germany;Italy;Netherlands;Norway;Spain;Taiwan;Turkey;United Kingdom;United States;
2017 Phase 3 EUCTR2016-000378-38-IT Australia;Belgium;Canada;Czech Republic;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Turkey;United Kingdom;United States;
Protalix
2017 Phase 3 NCT02921620 -
2013 Phase 1/Phase 2 NCT01769001 Australia;Paraguay;Spain;United Kingdom;United States;
2012 Phase 1/Phase 2 NCT01678898 Australia;Paraguay;Serbia;Spain;United Kingdom;United States;
Protalix Ltd.
2021 Phase 3 EUCTR2018-001148-67-FI Australia;Canada;Czech Republic;Czechia;Finland;France;Hungary;Italy;Netherlands;Norway;Slovenia;Spain;United Kingdom;United States;
2020 Phase 3 EUCTR2018-001148-67-SI Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Hungary;Italy;Netherlands;Norway;Slovenia;Spain;Switzerland;Turkey;United Kingdom;United States;
2019 Phase 3 EUCTR2018-001947-30-GB Australia;Belgium;Canada;Czech Republic;Denmark;Italy;Netherlands;Norway;Spain;Taiwan;Turkey;United Kingdom;United States;
2019 Phase 3 EUCTR2018-001947-30-DK Belgium;Czechia;Denmark;Italy;Norway;United Kingdom;United States;
2019 Phase 3 EUCTR2018-001947-30-BE Belgium;Czechia;Denmark;Italy;Norway;United Kingdom;United States;
2019 Phase 3 EUCTR2018-001148-67-NL Australia;Canada;Czech Republic;Czechia;Finland;France;Hungary;Italy;Netherlands;Norway;Slovenia;Spain;United Kingdom;United States;
2019 Phase 3 EUCTR2018-001148-67-HU Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Hungary;Italy;Netherlands;Norway;Slovenia;Spain;Switzerland;Turkey;United Kingdom;United States;
2019 Phase 3 EUCTR2018-001148-67-ES Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Denmark;Germany;Hungary;Italy;Netherlands;Norway;Slovenia;Spain;Switzerland;Turkey;United Kingdom;United States;
2019 Phase 3 EUCTR2016-000378-38-FR Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Denmark;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Switzerland;Turkey;United Kingdom;United States;
2019 Phase 3 EUCTR2016-000378-38-FI Argentina;Australia;Belgium;Brazil;Canada;Czechia;Denmark;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Slovenia;Spain;Sweden;Switzerland;Turkey;United Kingdom;United States;
2018 Phase 3 EUCTR2018-001148-67-GB Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Denmark;Germany;Hungary;Italy;Netherlands;Norway;Slovenia;Spain;Switzerland;Turkey;United Kingdom;United States;
2018 Phase 3 EUCTR2018-001148-67-CZ Australia;Canada;Czech Republic;Czechia;Finland;France;Hungary;Italy;Netherlands;Norway;Slovenia;Spain;United Kingdom;United States;
2018 Phase 3 EUCTR2017-001528-23-DK Austria;Belgium;Canada;Czech Republic;Denmark;Germany;Netherlands;Norway;Spain;Taiwan;Turkey;United Kingdom;United States;
2018 Phase 3 EUCTR2017-001528-23-CZ Austria;Belgium;Canada;Czech Republic;Denmark;Germany;Netherlands;Norway;Spain;Taiwan;Turkey;United Kingdom;United States;
2018 Phase 3 EUCTR2016-001318-11-NL Australia;Canada;Czech Republic;Germany;Netherlands;Norway;Slovenia;Spain;United Kingdom;
2017 Phase 3 EUCTR2017-001528-23-GB Belgium;Canada;Czech Republic;Denmark;Germany;Netherlands;Spain;Turkey;United Kingdom;United States;
2017 Phase 3 EUCTR2017-001528-23-BE Belgium;Canada;Czech Republic;Denmark;Germany;Netherlands;Spain;Turkey;United Kingdom;United States;
2017 Phase 3 EUCTR2016-001318-11-SI Australia;Canada;Czech Republic;Germany;Netherlands;Norway;Slovenia;Spain;United Kingdom;
2017 Phase 3 EUCTR2016-001318-11-GB Australia;Canada;Czech Republic;Germany;Netherlands;Norway;Slovenia;Spain;United Kingdom;
2017 Phase 3 EUCTR2016-001318-11-ES Australia;Canada;Germany;Netherlands;Norway;Spain;United Kingdom;
2017 Phase 3 EUCTR2016-001318-11-DE Australia;Canada;Czech Republic;Germany;Netherlands;Norway;Slovenia;Spain;United Kingdom;
2017 Phase 3 EUCTR2016-001318-11-CZ Australia;Canada;Czech Republic;Germany;Netherlands;Norway;Slovenia;Spain;United Kingdom;
2017 Phase 3 EUCTR2016-000378-38-SI Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Denmark;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Switzerland;Turkey;United Kingdom;United States;
2017 Phase 3 EUCTR2016-000378-38-NL Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Turkey;United Kingdom;United States;
2016 Phase 3 EUCTR2016-000378-38-HU Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Turkey;United Kingdom;United States;
2016 Phase 3 EUCTR2016-000378-38-GB Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Germany;Hungary;Netherlands;Norway;Paraguay;Slovenia;Spain;Turkey;United Kingdom;United States;
2016 Phase 3 EUCTR2016-000378-38-ES Australia;Belgium;Canada;Czech Republic;Germany;Hungary;Netherlands;Norway;Paraguay;Spain;Turkey;United Kingdom;United States;
2016 Phase 3 EUCTR2016-000378-38-BE Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Turkey;United Kingdom;United States;
2015 Phase 1;Phase 2 EUCTR2014-005544-18-GB Paraguay;Spain;United Kingdom;United States;
2013 Phase 1;Phase 2 EUCTR2013-002554-78-GB Argentina;Australia;Chile;Israel;Paraguay;Spain;United Kingdom;United States;
2013 - EUCTR2012-004786-40-GB Argentina;Australia;Chile;Israel;Paraguay;Spain;United Kingdom;United States;
PRX-102 (pegunigalsidase alfa)
Protalix
2017 Phase 3 NCT03018730 Australia;Canada;Czech Republic;Czechia;Germany;Netherlands;Norway;Slovenia;Spain;United Kingdom;
2016 Phase 3 NCT02795676 Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Czechia;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Switzerland;Turkey;United Kingdom;United States;
2013 Phase 1/Phase 2 NCT01981720 Paraguay;Spain;United Kingdom;United States;
PRX-112
Protalix
2014 Phase 2 NCT02107846 Israel;
2013 Phase 1 NCT01747980 Israel;
PRX102
Protalix Ltd.
2015 - EUCTR2014-005544-18-ES Paraguay;Spain;United Kingdom;United States;
2013 Phase 1;Phase 2 EUCTR2013-002554-78-ES Australia;Israel;Paraguay;Serbia;Spain;United Kingdom;United States;
2013 - EUCTR2012-004786-40-ES Argentina;Australia;Israel;Paraguay;Serbia;Spain;United Kingdom;United States;
PTC124
PTC Therapeutics Inc.
2015 Phase 2 EUCTR2015-003105-41-GB United Kingdom;
2015 Phase 2 EUCTR2014-002596-28-GB Germany;United Kingdom;
Pabinafusp alfa
JCR Pharmaceuticals Co., Ltd.
- Phase 3 EUCTR2020-003200-14-FR Brazil;France;Germany;United Kingdom;United States;
- Phase 3 EUCTR2020-003200-14-DE Brazil;France;Germany;United Kingdom;United States;
Yamamoto Tatsuyoshi
2021 Phase 4 JPRN-jRCT2031210126 Japan;
Paricalcitol
Federico II University
2012 - NCT02090608 Italy;
Parts A/B: Adrabetadex
Mandos LLC
2015 Phase 2/Phase 3 NCT02534844 Australia;France;Germany;New Zealand;Singapore;Spain;Turkey;United Kingdom;United States;
Pegunigalsidase alfa
PROTALIX LTD
2018 Phase 3 EUCTR2017-001528-23-IT Austria;Belgium;Canada;Czech Republic;Czechia;Denmark;Germany;Italy;Netherlands;Norway;Spain;Taiwan;Turkey;United Kingdom;United States;
2017 Phase 3 EUCTR2016-000378-38-IT Australia;Belgium;Canada;Czech Republic;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Turkey;United Kingdom;United States;
Protalix
2018 Phase 3 NCT03614234 Belgium;Czechia;Denmark;Italy;Norway;United Kingdom;United States;
2018 Phase 3 NCT03566017 Australia;Canada;Czechia;Finland;France;Hungary;Italy;Netherlands;Norway;Slovenia;Spain;United Kingdom;United States;
2017 Phase 3 NCT03180840 Belgium;Canada;Czechia;Denmark;Italy;Netherlands;Norway;Spain;Taiwan;Turkey;United Kingdom;United States;
Protalix Ltd.
2019 Phase 3 EUCTR2016-000378-38-FR Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Denmark;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Switzerland;Turkey;United Kingdom;United States;
2019 Phase 3 EUCTR2016-000378-38-FI Argentina;Australia;Belgium;Brazil;Canada;Czechia;Denmark;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Slovenia;Spain;Sweden;Switzerland;Turkey;United Kingdom;United States;
2018 Phase 3 EUCTR2017-001528-23-DK Austria;Belgium;Canada;Czech Republic;Denmark;Germany;Netherlands;Norway;Spain;Taiwan;Turkey;United Kingdom;United States;
2018 Phase 3 EUCTR2017-001528-23-CZ Austria;Belgium;Canada;Czech Republic;Denmark;Germany;Netherlands;Norway;Spain;Taiwan;Turkey;United Kingdom;United States;
2018 Phase 3 EUCTR2016-001318-11-NL Australia;Canada;Czech Republic;Germany;Netherlands;Norway;Slovenia;Spain;United Kingdom;
2017 Phase 3 EUCTR2017-001528-23-GB Belgium;Canada;Czech Republic;Denmark;Germany;Netherlands;Spain;Turkey;United Kingdom;United States;
2017 Phase 3 EUCTR2017-001528-23-BE Belgium;Canada;Czech Republic;Denmark;Germany;Netherlands;Spain;Turkey;United Kingdom;United States;
2017 Phase 3 EUCTR2016-001318-11-SI Australia;Canada;Czech Republic;Germany;Netherlands;Norway;Slovenia;Spain;United Kingdom;
2017 Phase 3 EUCTR2016-001318-11-GB Australia;Canada;Czech Republic;Germany;Netherlands;Norway;Slovenia;Spain;United Kingdom;
2017 Phase 3 EUCTR2016-001318-11-ES Australia;Canada;Germany;Netherlands;Norway;Spain;United Kingdom;
2017 Phase 3 EUCTR2016-001318-11-DE Australia;Canada;Czech Republic;Germany;Netherlands;Norway;Slovenia;Spain;United Kingdom;
2017 Phase 3 EUCTR2016-001318-11-CZ Australia;Canada;Czech Republic;Germany;Netherlands;Norway;Slovenia;Spain;United Kingdom;
2017 Phase 3 EUCTR2016-000378-38-SI Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Denmark;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Switzerland;Turkey;United Kingdom;United States;
2017 Phase 3 EUCTR2016-000378-38-NL Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Turkey;United Kingdom;United States;
2016 Phase 3 EUCTR2016-000378-38-HU Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Turkey;United Kingdom;United States;
2016 Phase 3 EUCTR2016-000378-38-GB Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Germany;Hungary;Netherlands;Norway;Paraguay;Slovenia;Spain;Turkey;United Kingdom;United States;
2016 Phase 3 EUCTR2016-000378-38-ES Australia;Belgium;Canada;Czech Republic;Germany;Hungary;Netherlands;Norway;Paraguay;Spain;Turkey;United Kingdom;United States;
2016 Phase 3 EUCTR2016-000378-38-BE Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Turkey;United Kingdom;United States;
Pegunigalsidase alpha
PROTALIX LTD
2017 Phase 3 EUCTR2016-000378-38-IT Australia;Belgium;Canada;Czech Republic;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Turkey;United Kingdom;United States;
Protalix Ltd.
2019 Phase 3 EUCTR2016-000378-38-FR Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Denmark;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Switzerland;Turkey;United Kingdom;United States;
2019 Phase 3 EUCTR2016-000378-38-FI Argentina;Australia;Belgium;Brazil;Canada;Czechia;Denmark;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Slovenia;Spain;Sweden;Switzerland;Turkey;United Kingdom;United States;
2018 Phase 3 EUCTR2016-001318-11-NL Australia;Canada;Czech Republic;Germany;Netherlands;Norway;Slovenia;Spain;United Kingdom;
2017 Phase 3 EUCTR2016-001318-11-SI Australia;Canada;Czech Republic;Germany;Netherlands;Norway;Slovenia;Spain;United Kingdom;
2017 Phase 3 EUCTR2016-001318-11-GB Australia;Canada;Czech Republic;Germany;Netherlands;Norway;Slovenia;Spain;United Kingdom;
2017 Phase 3 EUCTR2016-001318-11-ES Australia;Canada;Germany;Netherlands;Norway;Spain;United Kingdom;
2017 Phase 3 EUCTR2016-001318-11-DE Australia;Canada;Czech Republic;Germany;Netherlands;Norway;Slovenia;Spain;United Kingdom;
2017 Phase 3 EUCTR2016-001318-11-CZ Australia;Canada;Czech Republic;Germany;Netherlands;Norway;Slovenia;Spain;United Kingdom;
2017 Phase 3 EUCTR2016-000378-38-SI Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Denmark;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Switzerland;Turkey;United Kingdom;United States;
2017 Phase 3 EUCTR2016-000378-38-NL Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Turkey;United Kingdom;United States;
2016 Phase 3 EUCTR2016-000378-38-HU Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Turkey;United Kingdom;United States;
2016 Phase 3 EUCTR2016-000378-38-GB Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Germany;Hungary;Netherlands;Norway;Paraguay;Slovenia;Spain;Turkey;United Kingdom;United States;
2016 Phase 3 EUCTR2016-000378-38-ES Australia;Belgium;Canada;Czech Republic;Germany;Hungary;Netherlands;Norway;Paraguay;Spain;Turkey;United Kingdom;United States;
2016 Phase 3 EUCTR2016-000378-38-BE Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Turkey;United Kingdom;United States;
Pending
Ultragenyx Pharmaceutical Inc.
2016 Phase 3 EUCTR2015-001875-32-PT Brazil;Mexico;Portugal;United States;
- - EUCTR2015-001875-32-Outside-EU/EEA United States;
- - EUCTR2015-000104-26-Outside-EU/EEA United States;
Pentosan Polysulfat SP 54® injection solution
Multiplex Pharma Holdings LLC
2014 - EUCTR2014-000350-11-DE Germany;
Pentosan Polysulphate SP54®
Multiplex Pharma Holdings LLC
2014 - EUCTR2014-000350-11-DE Germany;
Perdix
Salford Royal NHS Foundation Trust
2009 Phase 4 EUCTR2007-007482-21-GB United Kingdom;
Plant Cell Recombinant Glucocerebrosidase
PROTALIX BIOTHERAPEUTICS
2009 - EUCTR2008-005826-35-IT Italy;Spain;United Kingdom;
2009 - EUCTR2008-005825-12-IT Germany;Italy;Spain;United Kingdom;
2008 - EUCTR2007-000498-42-IT Italy;Spain;United Kingdom;
Plant cell expressed recombinant glucocerebrosidase (prGCD)
Pfizer
2007 Phase 3 NCT00376168 Canada;Chile;Israel;Italy;South Africa;Spain;United Kingdom;United States;
2006 - NCT00962260 Israel;United States;
Plasma Lyso-GB3
Chang Gung Memorial Hospital
2018 - NCT05056636 Taiwan;
PrGCD
PROTALIX BIOTHERAPEUTICS
2009 - EUCTR2008-005826-35-IT Italy;Spain;United Kingdom;
2009 - EUCTR2008-005825-12-IT Germany;Italy;Spain;United Kingdom;
2008 - EUCTR2007-000498-42-IT Italy;Spain;United Kingdom;
Protalix Biotherapeutics
2012 - EUCTR2011-003685-32-ES Canada;Chile;Israel;Italy;Mexico;Serbia;South Africa;Spain;
2011 - EUCTR2011-003676-37-ES Israel;South Africa;Spain;
2009 - EUCTR2008-005826-35-ES Italy;Spain;United Kingdom;
2009 - EUCTR2008-005825-12-ES Germany;Italy;Spain;United Kingdom;
2008 Phase 3 EUCTR2007-000498-42-GB Italy;Spain;United Kingdom;
2008 - EUCTR2007-000498-42-ES Italy;Spain;United Kingdom;
- - EUCTR2008-005826-35-Outside-EU/EEA Australia;Canada;Chile;Israel;South Africa;Spain;United Kingdom;United States;
- - EUCTR2008-005825-12-Outside-EU/EEA Canada;Israel;United Kingdom;United States;
- - EUCTR2007-000498-42-Outside-EU/EEA Israel;United States;
Prednisone
National Human Genome Research Institute (NHGRI)
2019 Phase 1/Phase 2 NCT03952637 United States;
Prevail Therapeutics
2021 Phase 1/Phase 2 NCT04411654 United Kingdom;United States;
Prevenar13
Maastricht University
2016 Phase 4 EUCTR2015-004846-25-BE Belgium;
Pyrimethamine
Exsar Corporation
2008 Phase 1 NCT00679744 Canada;United States;
The Hospital for Sick Children
2009 Phase 1/Phase 2 NCT01102686 Canada;
Quantitative chemical shift imaging (QCSI)
Shaare Zedek Medical Center
2017 - NCT04353466 -
RAAV 2/6 hIDS DONOR
Sangamo Therapeutics, Inc.
2018 Phase 1;Phase 2 EUCTR2018-000192-33-GB United Kingdom;United States;
RAAV2/5-hNAGLU
UniQure Biopharma B.V.
2013 Phase 1/Phase 2 NCT03300453 France;
RAAV2/5-hNaGlu
Institut Pasteur
- Phase 1;Phase 2 EUCTR2012-000856-33-FR France;
RAAV2/6 Left ZFN Vector
Sangamo Therapeutics, Inc.
2018 Phase 1;Phase 2 EUCTR2018-000206-28-GB United Kingdom;United States;
2018 Phase 1;Phase 2 EUCTR2018-000192-33-GB United Kingdom;United States;
RAAV2/6 Right ZFN Vector
Sangamo Therapeutics, Inc.
2018 Phase 1;Phase 2 EUCTR2018-000206-28-GB United Kingdom;United States;
2018 Phase 1;Phase 2 EUCTR2018-000192-33-GB United Kingdom;United States;
RAAV9.CMV.hNAGLU
Abeona Therapeutics Europe SL.
2021 Phase 1;Phase 2 EUCTR2019-002936-97-FR France;Germany;United States;
2019 Phase 1;Phase 2 EUCTR2014-001411-39-GB France;Germany;Italy;Netherlands;Spain;United Kingdom;United States;
2019 Phase 1;Phase 2 EUCTR2014-001411-39-DE France;Germany;Spain;United Kingdom;United States;
- Phase 1;Phase 2 EUCTR2019-002936-97-DE France;Germany;Spain;United States;
Abeona Therapeutics Inc
2018 Phase 1;Phase 2 EUCTR2014-001411-39-ES France;Germany;Italy;Netherlands;Spain;United Kingdom;United States;
RECOMBINANT HUMAN ACID SPHINGOMYELINASE (rhASM)
Genzyme Corporation
2015 Phase 2 EUCTR2014-003198-40-IT Brazil;Chile;France;Germany;Italy;United Kingdom;United States;
2015 Phase 2 EUCTR2014-003198-40-GB Brazil;France;Germany;Italy;United Kingdom;United States;
2015 Phase 2 EUCTR2014-003198-40-DE Brazil;France;Germany;Italy;United Kingdom;United States;
- Phase 2 EUCTR2014-003198-40-FR Brazil;Chile;France;Germany;Italy;United Kingdom;United States;
RECOMBINANT HUMAN ARYLSULFATASE A
Shire Human Genetic Therapies, Inc.
2021 Phase 2 EUCTR2018-003291-12-GR Argentina;Belgium;Brazil;Canada;France;Germany;Greece;Israel;Italy;Japan;Netherlands;Spain;United Kingdom;United States;
2020 Phase 2 EUCTR2018-003291-12-DE Belgium;Brazil;Canada;France;Germany;Israel;Japan;Mexico;Netherlands;Spain;United Kingdom;United States;
2019 Phase 2 EUCTR2018-003291-12-NL Argentina;Belgium;Brazil;Canada;France;Germany;Greece;Israel;Italy;Japan;Netherlands;Spain;United Kingdom;United States;
2019 Phase 2 EUCTR2018-003291-12-GB Belgium;Brazil;Canada;France;Germany;Israel;Japan;Mexico;Netherlands;Spain;United Kingdom;United States;
2019 Phase 2 EUCTR2018-003291-12-FR Belgium;Brazil;Canada;Denmark;France;Germany;Israel;Japan;Mexico;Netherlands;Spain;United Kingdom;United States;
2019 Phase 2 EUCTR2018-003291-12-ES Belgium;Brazil;Canada;Denmark;France;Germany;Israel;Japan;Mexico;Netherlands;Spain;United Kingdom;United States;
2019 Phase 2 EUCTR2018-003291-12-BE Belgium;Brazil;Canada;France;Germany;Israel;Japan;Mexico;Netherlands;Spain;United Kingdom;United States;
RECOMBINANT HUMAN BETA GLUCURONIDASE
Ultragenyx Pharmaceutical Inc.
2016 Phase 3 EUCTR2015-001875-32-PT Brazil;Mexico;Portugal;United States;
2016 Phase 2 EUCTR2015-000104-26-PT Portugal;Spain;United States;
2016 Phase 2 EUCTR2015-000104-26-ES Portugal;Spain;United States;
2014 Phase 1;Phase 2 EUCTR2013-001152-35-ES Spain;United Kingdom;
2013 Phase 1;Phase 2 EUCTR2013-001152-35-GB Spain;United Kingdom;
- - EUCTR2015-001875-32-Outside-EU/EEA United States;
- - EUCTR2015-000104-26-Outside-EU/EEA United States;
- - EUCTR2014-005638-71-Outside-EU/EEA United States;
RECOMBINANT HUMAN TRIPEPTIDYL PEPTIDASE-1 (RHTPP1)
BioMarin Pharmaceutical Inc.
2015 Phase 2 EUCTR2014-003480-37-GB Germany;Italy;United Kingdom;United States;
2015 Phase 2 EUCTR2014-003480-37-DE Germany;Italy;United Kingdom;United States;
2015 - EUCTR2014-003480-37-IT Germany;Italy;Japan;United Kingdom;United States;
REPLAGAL ®
Shire Human Genetic Therapies, Inc.
2021 Phase 3 EUCTR2018-004689-32-SE Argentina;Australia;Belgium;Bosnia and Herzegovina;Brazil;Canada;Croatia;Finland;Germany;Greece;Hungary;Italy;Latvia;Poland;Portugal;Romania;Slovenia;Spain;Sweden;Ukraine;
2021 Phase 3 EUCTR2018-004689-32-PT Argentina;Australia;Belgium;Bosnia and Herzegovina;Brazil;Canada;Croatia;Finland;Germany;Greece;Hungary;Italy;Latvia;Poland;Portugal;Romania;Slovenia;Spain;Sweden;Ukraine;
2021 Phase 3 EUCTR2018-004689-32-PL Argentina;Australia;Belgium;Bosnia and Herzegovina;Brazil;Canada;Croatia;Finland;Germany;Greece;Hungary;Italy;Latvia;Poland;Portugal;Romania;Slovenia;Spain;Sweden;Ukraine;
2021 Phase 3 EUCTR2018-004689-32-GR Argentina;Australia;Belgium;Bosnia and Herzegovina;Brazil;Canada;Croatia;Finland;Germany;Greece;Hungary;Italy;Latvia;Poland;Portugal;Romania;Slovenia;Spain;Sweden;Ukraine;
2021 Phase 3 EUCTR2018-004689-32-FI Argentina;Australia;Belgium;Bosnia and Herzegovina;Brazil;Canada;Croatia;Finland;Germany;Greece;Hungary;Italy;Latvia;Poland;Portugal;Romania;Slovenia;Spain;Sweden;Ukraine;
RHGUS
Ultragenyx Pharmaceutical Inc.
2016 Phase 2 EUCTR2015-000104-26-PT Portugal;Spain;United States;
2016 Phase 2 EUCTR2015-000104-26-ES Portugal;Spain;United States;
2014 Phase 1;Phase 2 EUCTR2013-001152-35-ES Spain;United Kingdom;
2013 Phase 1;Phase 2 EUCTR2013-001152-35-GB Spain;United Kingdom;
- - EUCTR2015-001875-32-Outside-EU/EEA United States;
- - EUCTR2015-000104-26-Outside-EU/EEA United States;
- - EUCTR2014-005638-71-Outside-EU/EEA United States;
RHNAGLU-IGF2
Allievex Corporation
2018 Phase 2 EUCTR2017-003083-13-GB Colombia;Germany;Spain;Taiwan;Turkey;United Kingdom;United States;
2016 Phase 1;Phase 2 EUCTR2015-001985-25-DE Australia;Colombia;Germany;Spain;Taiwan;Turkey;United Kingdom;
2015 Phase 1;Phase 2 EUCTR2015-001985-25-GB Australia;Colombia;Germany;Spain;Taiwan;Turkey;United Kingdom;
- Phase 2 EUCTR2017-003083-13-DE Colombia;Germany;Spain;Taiwan;Turkey;United Kingdom;United States;
BioMarin Pharmaceutical Inc.
2017 Phase 2 EUCTR2017-003083-13-ES Argentina;Australia;Colombia;Germany;Spain;Taiwan;Turkey;United Kingdom;United States;
2016 Phase 1;Phase 2 EUCTR2015-001985-25-ES Australia;Brazil;Colombia;Germany;Spain;Taiwan;Turkey;United Kingdom;
RP 103
HOSPICES CIVILS DE LYON
2010 - EUCTR2010-020098-18-FR France;
RP103
Horizon Pharma USA, Inc.
2014 Phase 3 EUCTR2012-002773-64-GB Belgium;France;Germany;Italy;Netherlands;United Kingdom;United States;
2013 Phase 3 NCT01733316 Belgium;France;Italy;Netherlands;United Kingdom;United States;
2013 Phase 3 EUCTR2012-002773-64-NL Belgium;France;Germany;Italy;Netherlands;United Kingdom;United States;
2012 Phase 3 NCT01744782 Brazil;United States;
2010 Phase 3 EUCTR2010-018365-34-NL France;Netherlands;United States;
2009 Phase 1/Phase 2 NCT00872729 United States;
Raptor Therapeutics Inc.
2014 Phase 3 EUCTR2012-002773-64-IT Belgium;France;Germany;Italy;Netherlands;United Kingdom;United States;
2014 Phase 3 EUCTR2012-002773-64-BE Belgium;France;Germany;Italy;Netherlands;United Kingdom;United States;
2013 Phase 3 EUCTR2012-002773-64-FR Belgium;France;Italy;Netherlands;United Kingdom;United States;
2010 Phase 3 EUCTR2009-017882-42-FR France;Netherlands;
2010 - EUCTR2009-017882-42-NL France;Netherlands;
- Phase 3 EUCTR2010-018365-34-FR France;Netherlands;
RVX000222
Resverlogix Corp
2019 Phase 1/Phase 2 NCT03228940 Canada;
Rebisufligene etisparvovec
Abeona Therapeutics Inc
2020 Phase 1;Phase 2 EUCTR2019-002979-34-ES Australia;France;Germany;Spain;United States;
2019 Phase 1;Phase 2 EUCTR2015-003904-21-FR Australia;Brazil;France;Germany;Spain;United Kingdom;United States;
Recombinant Adeno-associated virus 2/6 vector encoding the cDNA for human alpha galactosidase A
Sangamo Therapeutics, Inc.
2019 Phase 1;Phase 2 EUCTR2019-000667-24-GB United Kingdom;United States;
Recombinant Human Alpha-N-Acetylglucosaminidase (rhNAGLU)
Alexion Pharmaceuticals, Inc
2015 Phase 2 EUCTR2015-001983-20-GB United Kingdom;
Alexion Pharmaceuticals, Inc.
2016 Phase 2 EUCTR2015-000753-20-NL Netherlands;
2016 Phase 1 EUCTR2013-003400-39-ES Spain;United Kingdom;United States;
2014 Phase 1;Phase 2 EUCTR2013-003400-39-GB Spain;United Kingdom;United States;
Recombinant Human Arylsulfatase A (rhASA)
SHIRE HUMAN GENETIC THERAPIES, INC
2020 Phase 2 EUCTR2018-003291-12-IT Belgium;Brazil;Canada;France;Germany;Israel;Italy;Japan;Netherlands;Spain;United Kingdom;United States;
Shire Human Genetics Therapies Inc
2017 Phase 1;Phase 2 EUCTR2012-003775-20-IT Australia;Brazil;Czech Republic;Denmark;France;Germany;Italy;Japan;United Kingdom;
2016 Phase 1;Phase 2 EUCTR2012-003775-20-GB Australia;Brazil;Czech Republic;Denmark;France;Germany;Italy;Japan;United Kingdom;
2014 Phase 1;Phase 2 EUCTR2012-003775-20-CZ Australia;Brazil;Czech Republic;Denmark;France;Germany;Italy;Japan;United Kingdom;
2013 Phase 1;Phase 2 EUCTR2012-003775-20-DK Australia;Brazil;Czech Republic;Czechia;Denmark;France;Germany;Italy;Japan;United Kingdom;
2013 Phase 1;Phase 2 EUCTR2012-003775-20-DE Australia;Brazil;Czech Republic;Denmark;France;Germany;Italy;Japan;United Kingdom;
- Phase 1;Phase 2 EUCTR2012-003775-20-FR Argentina;Australia;Brazil;Czech Republic;Denmark;France;Germany;United Kingdom;
Recombinant Human Arylsulfatase A (rhASA,
Shire Human Genetics Therapies Inc
2012 Phase 1;Phase 2 EUCTR2011-002044-28-DE Australia;Brazil;Denmark;France;Germany;Japan;
2011 Phase 1;Phase 2 EUCTR2011-002044-28-DK Australia;Brazil;Denmark;France;Germany;Japan;
Recombinant Human Glucocerebrosidase (prGCD)
Protalix Biotherapeutics
2009 - EUCTR2008-005825-12-DE Germany;Italy;Spain;United Kingdom;
Recombinant alpha-galactosidase A
Assistance Publique - Hôpitaux de Paris
2005 Phase 4 NCT00487630 France;
Recombinant human Arylsulfatase A (rhASA)
Shire
2007 Phase 1/Phase 2 NCT00633139 Denmark;
Recombinant human Arysulfatase B, recombinant human N-acetylgalactosamine 4-sulfatase
BioMarin Pharmaceutical Inc.
2007 Phase 4 EUCTR2005-003512-30-FR France;Portugal;
Recombinant human N-acetylgalactosamine 4-sulfatase
BioMarin Pharmaceutical Inc.
- Phase 3 EUCTR2004-000642-21-IE Ireland;
Recombinant human N-acetylgalactosamine 6-sulfatase, BMN 110
BIOMARIN PHARMACEUTICAL INC.
2011 - EUCTR2010-020199-45-IT Argentina;Australia;Brazil;Canada;Colombia;Denmark;Germany;Italy;Japan;Korea, Democratic People's Republic of;Portugal;Qatar;Saudi Arabia;Spain;Switzerland;Taiwan;Turkey;United Kingdom;United States;
BioMarin Pharmaceutical Inc.
2012 Phase 3 EUCTR2010-020199-45-DE Argentina;Australia;Brazil;Canada;Colombia;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Portugal;Qatar;Saudi Arabia;Spain;Switzerland;Taiwan;Turkey;United Kingdom;United States;
2012 - EUCTR2010-020199-45-PT Argentina;Australia;Brazil;Canada;Colombia;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Portugal;Qatar;Saudi Arabia;Spain;Switzerland;Taiwan;Turkey;United Kingdom;United States;
Recombinant human N-acetylgalactosamine 6-sulfatase, BMN 110 drug substance
BioMarin Pharmaceutical Inc.
2009 - EUCTR2008-007365-23-GB United Kingdom;
Recombinant human N-acetylgalactosamine-6-sulfatase (rhGALNS)
BioMarin Pharmaceutical Inc.
2013 Phase 3 EUCTR2010-020199-45-ES Argentina;Australia;Brazil;Canada;Colombia;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Netherlands;Norway;Portugal;Qatar;Saudi Arabia;Spain;Taiwan;United Kingdom;United States;
2012 Phase 3 EUCTR2010-020199-45-DK Argentina;Australia;Brazil;Canada;Colombia;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Netherlands;Norway;Portugal;Qatar;Saudi Arabia;Spain;Taiwan;United Kingdom;United States;
2012 - EUCTR2011-005703-33-GB Germany;Italy;United Kingdom;United States;
2012 - EUCTR2011-005703-33-DE Germany;Italy;United Kingdom;United States;
2011 EUCTR2010-020199-45-GB Argentina;Australia;Brazil;Canada;Colombia;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Portugal;Qatar;Saudi Arabia;Spain;Switzerland;Taiwan;United Kingdom;United States;
Recombinant human N-acetylgalactosamine-6-sulfatase, BMN 110
BioMarin Pharmaceutical Inc.
2013 Phase 3 EUCTR2010-020199-45-ES Argentina;Australia;Brazil;Canada;Colombia;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Netherlands;Norway;Portugal;Qatar;Saudi Arabia;Spain;Taiwan;United Kingdom;United States;
2012 Phase 3 EUCTR2010-020199-45-DK Argentina;Australia;Brazil;Canada;Colombia;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Netherlands;Norway;Portugal;Qatar;Saudi Arabia;Spain;Taiwan;United Kingdom;United States;
Recombinant human N-acetylgalactosamine-6-sulfatase, BMN 110 drug substance
BIOMARIN PHARMACEUTICAL INC.
2012 Phase 2 EUCTR2011-003197-84-IT Italy;United Kingdom;United States;
2011 - EUCTR2010-020198-18-IT Denmark;Germany;Italy;Norway;Portugal;United Kingdom;
BioMarin Pharmaceutical Inc
2012 Phase 3 EUCTR2010-020198-18-NO Argentina;Australia;Brazil;Canada;Colombia;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Portugal;Qatar;Saudi Arabia;Switzerland;Taiwan;United Kingdom;United States;
2011 - EUCTR2010-020198-18-PT Argentina;Australia;Brazil;Canada;Colombia;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Portugal;Qatar;Saudi Arabia;Switzerland;Taiwan;United Kingdom;United States;
2011 - EUCTR2010-020198-18-DK Argentina;Australia;Brazil;Canada;Colombia;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Portugal;Qatar;Saudi Arabia;Switzerland;Taiwan;United Kingdom;United States;
2011 - EUCTR2010-020198-18-DE Argentina;Australia;Brazil;Canada;Colombia;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Portugal;Qatar;Saudi Arabia;Switzerland;Taiwan;United Kingdom;United States;
2010 - EUCTR2010-020198-18-GB Argentina;Australia;Brazil;Canada;Colombia;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Portugal;Qatar;Saudi Arabia;Spain;Switzerland;Taiwan;Turkey;United Kingdom;United States;
- Phase 3 EUCTR2010-020198-18-NL Denmark;France;Germany;Italy;Netherlands;Norway;Portugal;United Kingdom;
- Phase 3 EUCTR2010-020198-18-FR Denmark;France;Germany;Italy;Netherlands;Norway;Portugal;United Kingdom;
BioMarin Pharmaceutical Inc.
2012 - EUCTR2011-005703-33-GB Germany;Italy;United Kingdom;United States;
2012 - EUCTR2011-005703-33-DE Germany;Italy;United Kingdom;United States;
2012 - EUCTR2011-005682-20-GB Canada;Germany;United Kingdom;United States;
2012 - EUCTR2011-005682-20-DE Canada;Germany;United Kingdom;United States;
2011 Phase 2 EUCTR2011-003197-84-GB Italy;United Kingdom;United States;
2011 EUCTR2010-020199-45-GB Argentina;Australia;Brazil;Canada;Colombia;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Portugal;Qatar;Saudi Arabia;Spain;Switzerland;Taiwan;United Kingdom;United States;
2010 EUCTR2010-021048-16-GB United Kingdom;
Recombinant human Nacetylgalactosamine-
BioMarin Pharmaceutical Inc.
2012 Phase 3 EUCTR2010-020199-45-DE Argentina;Australia;Brazil;Canada;Colombia;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Portugal;Qatar;Saudi Arabia;Spain;Switzerland;Taiwan;Turkey;United Kingdom;United States;
2012 - EUCTR2010-020199-45-PT Argentina;Australia;Brazil;Canada;Colombia;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Portugal;Qatar;Saudi Arabia;Spain;Switzerland;Taiwan;Turkey;United Kingdom;United States;
Recombinant human alpha-galactosidase
Genzyme Europe BV
2008 - EUCTR2007-005668-28-GB Argentina;Brazil;Canada;Czech Republic;France;Germany;Netherlands;Norway;Poland;Portugal;United Kingdom;United States;
Recombinant human alpha-galactosidase abbreviated as r-h-alpha-GAL
Genzyme Europe BV
2009 Phase 3 EUCTR2007-005668-28-FR Czech Republic;France;Germany;Netherlands;Portugal;United Kingdom;
2008 - EUCTR2007-005668-28-PT Czech Republic;France;Germany;Netherlands;Portugal;United Kingdom;
2008 - EUCTR2007-005668-28-NL Czech Republic;France;Germany;Netherlands;Portugal;United Kingdom;
2008 - EUCTR2007-005668-28-DE Czech Republic;France;Germany;Netherlands;Portugal;United Kingdom;
2008 - EUCTR2007-005668-28-CZ Czech Republic;France;Germany;Netherlands;Portugal;United Kingdom;
Recombinant human alpha-mannosidase
CHIESI FARMACEUTICI S.P.A.
2018 Phase 2 EUCTR2016-001988-36-IT Austria;Denmark;France;Germany;Italy;
Chiesi Farmaceutici S.p.A.
2017 Phase 2 EUCTR2016-001988-36-FR Austria;Denmark;France;Germany;Italy;
2017 Phase 2 EUCTR2016-001988-36-DE Austria;Denmark;Germany;
2017 Phase 2 EUCTR2016-001988-36-AT Austria;Denmark;Germany;
2016 Phase 2 EUCTR2016-001988-36-DK Austria;Denmark;Germany;Italy;
2013 Phase 3 EUCTR2013-000321-31-DK Denmark;Poland;
- Phase 3 EUCTR2013-000321-31-PL Denmark;Poland;
Zymenex A/S
2013 Phase 3 EUCTR2012-000979-17-ES Belgium;Denmark;Germany;Spain;Sweden;
2013 - EUCTR2012-000979-17-SE Belgium;Denmark;Germany;Spain;Sweden;
2013 - EUCTR2012-000979-17-BE Belgium;Denmark;Germany;Spain;Sweden;
2012 Phase 2 EUCTR2011-004355-40-DK Belgium;Denmark;Germany;Spain;United Kingdom;
2012 - EUCTR2012-000979-17-DK Belgium;Denmark;Germany;Spain;Sweden;
2012 - EUCTR2012-000979-17-DE Belgium;Denmark;Germany;Spain;Sweden;
2012 - EUCTR2011-004355-40-GB Belgium;Denmark;Germany;Spain;United Kingdom;
2012 - EUCTR2011-004355-40-DE Belgium;Denmark;Germany;Spain;United Kingdom;
2012 - EUCTR2011-004355-40-BE Belgium;Denmark;Germany;Spain;United Kingdom;
2010 - EUCTR2010-022085-26-DK Denmark;
2010 - EUCTR2010-022084-36-DK Denmark;
Recombinant human arylsulfatase A
Shire
2012 Phase 1/Phase 2 NCT01510028 Argentina;Australia;Brazil;Denmark;France;Germany;Japan;
Shire Human Genetic Therapies Inc
2010 Phase 2 EUCTR2008-000084-41-BE Belgium;Denmark;France;Italy;
2009 - EUCTR2008-000084-41-FR Belgium;Denmark;France;Italy;
2008 Phase 2 EUCTR2008-000084-41-DK Belgium;Denmark;France;Italy;
Shire Pharmaceutical Ireland Limited
2008 - EUCTR2007-006345-40-DK Denmark;
Shire Pharmaceuticals Ireland Limited
2008 Phase 2 EUCTR2007-007165-20-DK Denmark;
Recombinant human beta-glucuronidase
Hamazaki Takashi
2020 Phase 3 JPRN-jRCT2051190085 Japan;
Ultragenyx Pharmaceutical Inc.
2016 Phase 2 EUCTR2015-000104-26-PT Portugal;Spain;United States;
2016 Phase 2 EUCTR2015-000104-26-ES Portugal;Spain;United States;
2014 Phase 1;Phase 2 EUCTR2013-001152-35-ES Spain;United Kingdom;
2013 Phase 1;Phase 2 EUCTR2013-001152-35-GB Spain;United Kingdom;
- - EUCTR2015-001875-32-Outside-EU/EEA United States;
- - EUCTR2014-005638-71-Outside-EU/EEA United States;
Recombinant human beta-glucuronidase (rhGUS)
Ultragenyx Pharmaceutical Inc.
2016 Phase 3 EUCTR2015-001875-32-PT Brazil;Mexico;Portugal;United States;
Recombinant human betaglucuronidase
Ultragenyx Pharmaceutical Inc.
- - EUCTR2015-000104-26-Outside-EU/EEA United States;
Recombinant human derived macrophage-targeted beta-Glucocerebrosidase
Genzyme Europe B.V.
2010 - EUCTR2008-005223-28-DE Argentina;Australia;Brazil;Canada;Czech Republic;Egypt;France;Germany;Italy;Netherlands;Russian Federation;Spain;Turkey;United Kingdom;United States;
2009 Phase 3 EUCTR2008-005223-28-GB Argentina;Australia;Brazil;Canada;Czech Republic;Egypt;France;Germany;Italy;Netherlands;Russian Federation;Spain;Turkey;United Kingdom;United States;
2009 Phase 3 EUCTR2008-005223-28-FR Czech Republic;France;Germany;Italy;Netherlands;Spain;United Kingdom;
2009 - EUCTR2008-005223-28-NL Czech Republic;France;Germany;Italy;Netherlands;Spain;United Kingdom;
Recombinant human derived macrophage-targeted beta-glucocerebrosidase
Genzyme Europe B.V.
2009 - EUCTR2008-005223-28-CZ Czech Republic;France;Germany;Italy;Netherlands;Spain;United Kingdom;
Recombinant human derived macrophage-targeted ß-Glucocerebrosidase
Genzyme Europe B.V.
2010 - EUCTR2008-005223-28-DE Argentina;Australia;Brazil;Canada;Czech Republic;Egypt;France;Germany;Italy;Netherlands;Russian Federation;Spain;Turkey;United Kingdom;United States;
2009 Phase 3 EUCTR2008-005223-28-GB Argentina;Australia;Brazil;Canada;Czech Republic;Egypt;France;Germany;Italy;Netherlands;Russian Federation;Spain;Turkey;United Kingdom;United States;
2009 Phase 3 EUCTR2008-005223-28-FR Czech Republic;France;Germany;Italy;Netherlands;Spain;United Kingdom;
2009 - EUCTR2008-005223-28-NL Czech Republic;France;Germany;Italy;Netherlands;Spain;United Kingdom;
Recombinant human derived macrophage-targeted ß-glucocerebrosidase
Genzyme Europe B.V.
2009 - EUCTR2008-005223-28-CZ Czech Republic;France;Germany;Italy;Netherlands;Spain;United Kingdom;
Recombinant human glucerebrosidase
Protalix Biotherapeutics
2012 - EUCTR2011-003685-32-ES Canada;Chile;Israel;Italy;Mexico;Serbia;South Africa;Spain;
2011 - EUCTR2011-003676-37-ES Israel;South Africa;Spain;
Recombinant human glucocerebrosidase
Protalix Biotherapeutics
2009 - EUCTR2008-005826-35-ES Italy;Spain;United Kingdom;
2009 - EUCTR2008-005825-12-ES Germany;Italy;Spain;United Kingdom;
2008 Phase 3 EUCTR2007-000498-42-GB Italy;Spain;United Kingdom;
2008 - EUCTR2007-000498-42-ES Italy;Spain;United Kingdom;
Recombinant human glucocerebrosidase (prGCD)
Protalix Biotherapeutics
2009 Phase 3 EUCTR2008-005826-35-GB Italy;Spain;United Kingdom;
2009 Phase 3 EUCTR2008-005825-12-GB Germany;Italy;Spain;United Kingdom;
Recombinant human heparan N-sulfatase
SHIRE HUMAN GENETIC THERAPIES, INC
2015 Phase 2 EUCTR2014-003960-20-IT France;Germany;Italy;Netherlands;Spain;United Kingdom;United States;
Recombinant human heparan N-sulfatase (rhHNS)
Shire
2010 Phase 1/Phase 2 NCT01155778 Netherlands;United Kingdom;
Shire Human Genetic Therapies, Inc
2014 Phase 2 EUCTR2013-003450-24-NL Argentina;France;Germany;Italy;Netherlands;Spain;United Kingdom;United States;
2014 Phase 2 EUCTR2013-003450-24-IT France;Germany;Italy;Netherlands;Spain;United Kingdom;
2014 Phase 2 EUCTR2013-003450-24-GB Argentina;Brazil;France;Germany;Italy;Netherlands;Spain;United Kingdom;United States;
2014 Phase 2 EUCTR2013-003450-24-DE Argentina;Brazil;France;Germany;Italy;Netherlands;Spain;United Kingdom;United States;
2011 Phase 1;Phase 2 EUCTR2010-021348-16-NL Netherlands;United Kingdom;
2011 Phase 1;Phase 2 EUCTR2010-021348-16-GB Netherlands;United Kingdom;
- Phase 2 EUCTR2013-003450-24-FR Argentina;Brazil;France;Germany;Italy;Netherlands;Spain;United Kingdom;United States;
Shire Human Genetic Therapies, Inc.
2015 Phase 2 EUCTR2014-003960-20-NL France;Germany;Italy;Netherlands;Spain;United Kingdom;United States;
2015 Phase 2 EUCTR2014-003960-20-GB France;Germany;Italy;Netherlands;Spain;United Kingdom;United States;
2015 Phase 2 EUCTR2014-003960-20-DE France;Germany;Italy;Netherlands;Spain;United Kingdom;United States;
Shire Human Genetic Therapies, Inc. (Shire HGT)
2010 Phase 1;Phase 2 EUCTR2009-015984-15-GB United Kingdom;
Shire human Genetic Therapies, Inc.
2015 Phase 2 EUCTR2014-003960-20-ES Argentina;France;Germany;Italy;Netherlands;Spain;United Kingdom;United States;
- Phase 2 EUCTR2014-003960-20-FR Argentina;France;Germany;Italy;Netherlands;Spain;United Kingdom;United States;
Recombinant human heparan N-sulfatase [rhHNS]
Shire
2014 Phase 2 NCT02060526 Argentina;France;Germany;Italy;Netherlands;Spain;United Kingdom;United States;
Recombinant human heparan Nsulfatase (rhHNS)
SHIRE HUMAN GENETIC THERAPIES, INC
2015 Phase 2 EUCTR2014-003960-20-IT France;Germany;Italy;Netherlands;Spain;United Kingdom;United States;
Recombinant human lysosomal acid lipase (rhLAL)
Alexion Pharmaceuticals Inc.
2013 Phase 2;Phase 3 EUCTR2011-000032-28-IE Egypt;France;Germany;Ireland;Italy;Saudi Arabia;Taiwan;Turkey;United Kingdom;United States;
SYNAGEVA BIOPHARMA CORP.
2012 - EUCTR2011-000032-28-IT Germany;Ireland;Italy;Taiwan;United Kingdom;United States;
Synageva Biopharma Corp.
2013 - EUCTR2011-000032-28-DE France;Germany;Italy;Taiwan;United Kingdom;United States;
2011 Phase 1;Phase 2;Phase 3 EUCTR2011-000032-28-GB France;Germany;Ireland;Italy;Taiwan;United Kingdom;United States;
2011 - EUCTR2011-002880-42-GB France;Germany;United Kingdom;United States;
2011 - EUCTR2010-024068-16-GB Czech Republic;United Kingdom;
2011 - EUCTR2010-024068-16-CZ Czech Republic;United Kingdom;United States;
- Phase 2 EUCTR2011-002880-42-FR France;Germany;United Kingdom;United States;
- Phase 1;Phase 2 EUCTR2011-000032-28-FR France;Germany;Ireland;Italy;United Kingdom;United States;
Recombinant human lysosomal acid lipase (rhLAL), esterase, cholesterol (human gene LIPA)
Synageva Biopharma Corp.
2011 Phase 2 EUCTR2011-001513-13-GB Czech Republic;France;United Kingdom;United States;
Recombinant human lysosomal acid lipase (rhLAL), esterase, cholesterol (human gene LIPA), lysosomal acid lipase (human gene LIPA)
Synageva Biopharma Corp.
2011 Phase 2 EUCTR2011-001513-13-CZ Czech Republic;France;United Kingdom;United States;
Recombinant human lysosomal alpha-mannosidase
Zymenex A/S
2015 - EUCTR2014-003950-15-DK Denmark;
2013 Phase 3 EUCTR2013-000336-97-FR Denmark;France;
Recombinant human tripeptidyl peptidase-1
BioMarin Pharmaceutical Inc
2017 Phase 2 EUCTR2015-000891-85-GB Germany;Italy;United Kingdom;United States;
BioMarin Pharmaceutical Inc.
2016 Phase 2 EUCTR2015-000891-85-DE Germany;Italy;United Kingdom;
2014 Phase 1;Phase 2 EUCTR2012-005430-11-IT Germany;Italy;United Kingdom;
2013 Phase 1;Phase 2 EUCTR2012-005430-11-GB Germany;Italy;United Kingdom;United States;
2013 Phase 1;Phase 2 EUCTR2012-005430-11-DE Germany;United Kingdom;
Recombinant self-complementary adeno-associated viral vector serotype 9 containing the human CLN6 gene
Amicus Therapeutics, Inc.
2021 Phase 1;Phase 2 EUCTR2021-000706-21-DE Germany;United States;
Reduced Intensity Conditioning
Duke University
2008 Phase 1 NCT00744692 United States;
Reminder Product
Amicus Therapeutics, Inc.
2010 Phase 3 EUCTR2009-013459-31-BE Belgium;Denmark;France;Germany;Italy;Netherlands;Spain;United Kingdom;
2010 - EUCTR2009-013459-31-NL Belgium;Denmark;France;Germany;Italy;Netherlands;Spain;United Kingdom;
- - EUCTR2009-013459-31-DE Belgium;Denmark;France;Germany;Italy;Netherlands;Spain;United Kingdom;
Reminder capsules
Amicus Therapeutics, Inc.
2009 Phase 2 EUCTR2007-001838-13-GB Australia;Brazil;United Kingdom;United States;
Replagal
Amicus Therapeutics, Inc.
2012 - EUCTR2010-022709-16-GB Belgium;United Kingdom;
2012 - EUCTR2010-022636-37-DE Argentina;Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Hungary;Israel;Italy;Japan;Mexico;Poland;Russian Federation;Slovakia;Switzerland;Taiwan;Turkey;United Kingdom;United States;
2011 Phase 3 EUCTR2010-022636-37-GR Argentina;Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Hungary;Israel;Italy;Japan;Mexico;Poland;Russian Federation;Slovakia;Switzerland;Taiwan;Turkey;United Kingdom;United States;
2011 Phase 2 EUCTR2010-022709-16-BE Australia;Belgium;Canada;France;Netherlands;Switzerland;United Kingdom;
2011 - EUCTR2010-022636-37-GB Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;United Kingdom;United States;
2011 - EUCTR2010-022636-37-DK Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;United Kingdom;United States;
2011 - EUCTR2010-022636-37-BE Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;United Kingdom;United States;
2011 - EUCTR2010-022636-37-AT Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;United Kingdom;United States;
Baylor Research Institute
2003 Phase 2 NCT00068107 United States;
National Institute of Neurological Disorders and Stroke (NINDS)
2004 Phase 2 NCT00075244 United States;
2003 Phase 2 NCT00071877 United States;
SANOFI-AVENTIS RECHERCHE E DEVELOPPEMENT
2022 Phase 3 EUCTR2021-002320-20-IT Austria;Canada;China;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Turkey;United Kingdom;United States;
Sanofi Aventis Groupe (SAG)
2019 Phase 4 EUCTR2019-000064-21-GB Austria;Canada;Czech Republic;Denmark;France;Germany;Italy;Norway;Spain;Turkey;United Kingdom;
- Phase 4 EUCTR2019-000064-21-NO Austria;Canada;Czech Republic;Denmark;France;Germany;Italy;Norway;Spain;Turkey;United Kingdom;
- Phase 4 EUCTR2019-000064-21-CZ Austria;Canada;Czech Republic;Denmark;France;Germany;Italy;Norway;Spain;Turkey;United Kingdom;
Shire
2022 Phase 4 NCT05067868 India;
2010 Phase 3 NCT01124643 Australia;Czech Republic;Czechia;Finland;Paraguay;Poland;Slovenia;United Kingdom;United States;
2008 Phase 3 NCT00864851 Australia;Czech Republic;Czechia;Finland;Paraguay;Poland;Slovenia;United Kingdom;United States;
Shire Human Genetic Therapies (HGT), Inc.
2012 Phase 3;Phase 4 EUCTR2009-015985-75-SI Australia;Czech Republic;Finland;Paraguay;Poland;Slovenia;United Kingdom;United States;
2011 Phase 3;Phase 4 EUCTR2009-015985-75-GB Australia;Czech Republic;Finland;Paraguay;Poland;Slovenia;United Kingdom;United States;
2011 - EUCTR2009-015985-75-FI Australia;Czech Republic;Finland;Paraguay;Poland;Slovenia;United Kingdom;United States;
2010 - EUCTR2009-015985-75-PL Australia;Czech Republic;Finland;Paraguay;Poland;Slovenia;United Kingdom;United States;
2010 - EUCTR2009-015985-75-CZ Australia;Czech Republic;Finland;Paraguay;Poland;Slovenia;United Kingdom;United States;
Shire Human Genetic Therapies, Inc. (Shire HGT)
2011 - EUCTR2007-005543-22-SI Australia;Czech Republic;Finland;Paraguay;Slovenia;United Kingdom;United States;
2010 Phase 3;Phase 4 EUCTR2007-005543-22-GB Australia;Czech Republic;Finland;Paraguay;Slovenia;United Kingdom;United States;
2010 Phase 3;Phase 4 EUCTR2007-005543-22-FI Australia;Czech Republic;Finland;Paraguay;Slovenia;United Kingdom;United States;
2008 - EUCTR2007-005543-22-CZ Australia;Czech Republic;Finland;Paraguay;Slovenia;United Kingdom;United States;
TKT Inc
2004 - EUCTR2004-000772-14-CZ Czech Republic;
sanofi-aventis recherche et développement
- Phase 3 EUCTR2021-002320-20-ES Austria;Canada;China;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Turkey;United Kingdom;United States;
Replagal (Agalsidase Alfa)
National Institute of Neurological Disorders and Stroke (NINDS)
2004 Phase 4 NCT00097890 United States;
Replagal (agalsidase alfa)
Shire
2011 Phase 2 NCT01363492 United States;
Replagal agalsidase alfa
National Institute of Neurological Disorders and Stroke (NINDS)
2003 Phase 1 NCT00357786 United States;
Rh Growth Hormone
National Human Genome Research Institute (NHGRI)
2014 Phase 1/Phase 2 NCT02124070 United States;
RhARSA
Assistance Publique - Hôpitaux de Paris
2008 Phase 2 NCT01303146 France;
RhASA
Shire Human Genetic Therapies Inc
2010 Phase 2 EUCTR2008-000084-41-BE Belgium;Denmark;France;Italy;
2009 - EUCTR2008-000084-41-FR Belgium;Denmark;France;Italy;
2008 Phase 2 EUCTR2008-000084-41-DK Belgium;Denmark;France;Italy;
Shire Pharmaceutical Ireland Limited
2008 - EUCTR2007-006345-40-DK Denmark;
Shire Pharmaceuticals Ireland Limited
2008 Phase 2 EUCTR2007-007165-20-DK Denmark;
2006 - EUCTR2006-005341-11-DK Denmark;
RhASA - Dose Level 1
Shire
2007 Phase 1 NCT00418561 Denmark;
RhASA - Dose Level 2
Shire
2007 Phase 1 NCT00418561 Denmark;
RhASA - Dose Level 3
Shire
2007 Phase 1 NCT00418561 Denmark;
RhASM
Genzyme, a Sanofi Company
2006 Phase 1 NCT00410566 United States;
RhHNS-
Shire
2011 Phase 1/Phase 2 NCT01299727 Netherlands;United Kingdom;
RhIDU (recombinant human-Alpha-L-Iduronidase)
Genzyme, a Sanofi Company
2000 Phase 3 NCT00912925 Canada;Germany;United States;
RhNAGLU radiolabelled
Alexion Pharmaceuticals, Inc.
2016 Phase 2 EUCTR2015-000753-20-NL Netherlands;
Rimiducid
Bellicum Pharmaceuticals
2017 - NCT03639844 United States;
Rituximab
National Human Genome Research Institute (NHGRI)
2019 Phase 1/Phase 2 NCT03952637 United States;
Rivogenlecleucel
Bellicum Pharmaceuticals
2017 - NCT03639844 United States;
SAF-301
SANFILIPPO Therapeutics SAS
- - EUCTR2010-019962-10-FR France;
SAR402671, GZ402671 o GZ/SAR402671
SANOFI-AVENTIS RECHERCHE E DEVELOPPEMENT
2022 Phase 3 EUCTR2021-002320-20-IT Austria;Canada;China;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Turkey;United Kingdom;United States;
2021 Phase 3 EUCTR2021-002350-90-IT Argentina;Austria;Brazil;Bulgaria;Canada;China;Denmark;Finland;France;Germany;Greece;Italy;Mexico;Netherlands;Norway;Poland;Romania;Russian Federation;Turkey;Ukraine;United Kingdom;
SAR402671, GZ402671 or GZ / SAR402671
Sanofi-aventis recherche & développement
- Phase 3 EUCTR2021-005402-10-FR Argentina;Brazil;Canada;China;Egypt;France;Germany;Hungary;Japan;Taiwan;Turkey;United Kingdom;United States;
- Phase 3 EUCTR2021-005402-10-DE Argentina;Brazil;Canada;China;Egypt;France;Germany;Hungary;Japan;Taiwan;Turkey;United Kingdom;United States;
SAR402671, GZ402671 or GZ/SAR402671
Genzyme Corporation
2015 Phase 2 EUCTR2014-004995-49-PL Czech Republic;France;Poland;Russian Federation;United Kingdom;United States;
2015 Phase 2 EUCTR2014-004995-49-GB Czech Republic;France;Poland;Russian Federation;United Kingdom;United States;
2015 Phase 2 EUCTR2014-004995-49-FR Czech Republic;France;Poland;Russian Federation;United Kingdom;United States;
Sanofi-Aventis Recherche & Développement
2021 Phase 3 EUCTR2021-002350-90-GR Argentina;Austria;Brazil;Bulgaria;Canada;China;Denmark;Finland;France;Germany;Greece;Italy;Mexico;Netherlands;Norway;Poland;Romania;Russian Federation;Turkey;Ukraine;United Kingdom;
sanofi-aventis recherche et développement
- Phase 3 EUCTR2021-002320-20-ES Austria;Canada;China;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Turkey;United Kingdom;United States;
SB-318
Sangamo Therapeutics
2020 - NCT04628871 United States;
2017 Phase 1/Phase 2 NCT02702115 United States;
SB-47171
Sangamo Therapeutics, Inc.
2018 Phase 1;Phase 2 EUCTR2018-000206-28-GB United Kingdom;United States;
2018 Phase 1;Phase 2 EUCTR2018-000192-33-GB United Kingdom;United States;
SB-47898
Sangamo Therapeutics, Inc.
2018 Phase 1;Phase 2 EUCTR2018-000206-28-GB United Kingdom;United States;
2018 Phase 1;Phase 2 EUCTR2018-000192-33-GB United Kingdom;United States;
SB-913
Sangamo Therapeutics
2020 - NCT04628871 United States;
2017 Phase 1/Phase 2 NCT03041324 United States;
SB-FIX
Sangamo Therapeutics
2020 - NCT04628871 United States;
SB-IDUA
Sangamo Therapeutics, Inc.
2018 Phase 1;Phase 2 EUCTR2018-000206-28-GB United Kingdom;United States;
SBC-102
Alexion Pharmaceuitcals Inc
2014 Phase 2 EUCTR2011-004287-30-HR Australia;Belgium;Brazil;Canada;Croatia;Denmark;Germany;Italy;Japan;Mexico;Netherlands;Russian Federation;Spain;Turkey;United Kingdom;United States;
2014 Phase 2 EUCTR2011-004287-30-DE Australia;Belgium;Brazil;Canada;Croatia;Denmark;Germany;Italy;Japan;Mexico;Netherlands;Russian Federation;Spain;Turkey;United Kingdom;United States;
Alexion Pharmaceuticals
2011 Phase 2/Phase 3 NCT01473875 France;United Kingdom;
Alexion Pharmaceuticals Inc
2015 Phase 2 EUCTR2014-000533-22-FI Finland;Italy;United Kingdom;United States;
2015 Phase 2 EUCTR2011-004287-30-NL Australia;Belgium;Brazil;Canada;Croatia;Denmark;Germany;Italy;Mexico;Netherlands;Russian Federation;Spain;Turkey;United Kingdom;United States;
2015 Phase 2 EUCTR2011-004287-30-GB Australia;Belgium;Brazil;Canada;Croatia;Denmark;Germany;Italy;Japan;Mexico;Netherlands;Poland;Russian Federation;Spain;Turkey;United Kingdom;United States;
2014 Phase 2 EUCTR2014-000533-22-GB Finland;Italy;United Kingdom;United States;
Alexion Pharmaceuticals inc
2014 Phase 2 EUCTR2011-004287-30-DK Australia;Belgium;Brazil;Canada;Croatia;Denmark;Germany;Italy;Japan;Mexico;Netherlands;Poland;Russian Federation;Spain;Turkey;United Kingdom;United States;
Alexion Pharmaceuticals, Inc.
2015 Phase 3 EUCTR2011-002750-31-CZ Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Japan;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;United States;
2013 Phase 3 EUCTR2011-002750-31-GB Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Japan;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;United States;
2013 Phase 3 EUCTR2011-002750-31-DE Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Japan;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;United States;
- Phase 3 EUCTR2011-002750-31-PL Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Japan;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;United States;
SYNAGEVA BIOPHARMA CORP.
2012 - EUCTR2011-000032-28-IT Germany;Ireland;Italy;Taiwan;United Kingdom;United States;
Synageva BioPharma Corp.
2014 Phase 2 EUCTR2014-000533-22-IT Finland;Italy;United Kingdom;United States;
2014 Phase 2 EUCTR2011-004287-30-IT Australia;Belgium;Brazil;Canada;Croatia;Denmark;Germany;Italy;Japan;Mexico;Netherlands;Poland;Russian Federation;Spain;Turkey;United Kingdom;United States;
2014 Phase 2 EUCTR2011-004287-30-ES Australia;Belgium;Brazil;Canada;Croatia;Denmark;Germany;Italy;Japan;Mexico;Netherlands;Poland;Russian Federation;Spain;Turkey;United Kingdom;United States;
2014 Phase 2 EUCTR2011-004287-30-BE Australia;Belgium;Brazil;Canada;Croatia;Denmark;Germany;Italy;Japan;Mexico;Netherlands;Russian Federation;Spain;Turkey;United Kingdom;United States;
Synageva BioPharma Corporation
2013 Phase 3 EUCTR2011-002750-31-IT Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;
2013 Phase 3 EUCTR2011-002750-31-HR Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;
2013 Phase 3 EUCTR2011-002750-31-GR Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;
2013 Phase 3 EUCTR2011-002750-31-ES Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;
- Phase 3 EUCTR2011-002750-31-FR Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;
Synageva Biopharma Corp.
2011 Phase 2 EUCTR2011-001513-13-GB Czech Republic;France;United Kingdom;United States;
2011 Phase 2 EUCTR2011-001513-13-CZ Czech Republic;France;United Kingdom;United States;
2011 Phase 1;Phase 2;Phase 3 EUCTR2011-000032-28-GB France;Germany;Ireland;Italy;Taiwan;United Kingdom;United States;
2011 - EUCTR2011-002880-42-GB France;Germany;United Kingdom;United States;
2011 - EUCTR2010-024068-16-GB Czech Republic;United Kingdom;
2011 - EUCTR2010-024068-16-CZ Czech Republic;United Kingdom;United States;
- Phase 2 EUCTR2011-002880-42-FR France;Germany;United Kingdom;United States;
- Phase 1;Phase 2 EUCTR2011-000032-28-FR France;Germany;Ireland;Italy;United Kingdom;United States;
SBC-102, recombinant human lysosomal acid lipase
Alexion Pharmaceuticals Inc.
2013 Phase 2;Phase 3 EUCTR2011-000032-28-IE Egypt;France;Germany;Ireland;Italy;Saudi Arabia;Taiwan;Turkey;United Kingdom;United States;
Synageva Biopharma Corp.
2013 - EUCTR2011-000032-28-DE France;Germany;Italy;Taiwan;United Kingdom;United States;
SBC-103
Alexion Pharmaceuticals
2015 Phase 1/Phase 2 NCT02618512 United Kingdom;
2015 Phase 1/Phase 2 NCT02324049 Spain;United Kingdom;United States;
Alexion Pharmaceuticals, Inc
2015 Phase 2 EUCTR2015-001983-20-GB United Kingdom;
Alexion Pharmaceuticals, Inc.
2016 Phase 1 EUCTR2013-003400-39-ES Spain;United Kingdom;United States;
2014 Phase 1;Phase 2 EUCTR2013-003400-39-GB Spain;United Kingdom;United States;
SHP611
SHIRE HUMAN GENETIC THERAPIES, INC
2020 Phase 2 EUCTR2018-003291-12-IT Belgium;Brazil;Canada;France;Germany;Israel;Italy;Japan;Netherlands;Spain;United Kingdom;United States;
Shire
2019 Phase 2 NCT03771898 Belgium;Brazil;Canada;Denmark;France;Germany;Israel;Japan;Mexico;Netherlands;Spain;United Kingdom;United States;
Shire Human Genetic Therapies, Inc.
2021 Phase 2 EUCTR2018-003291-12-GR Argentina;Belgium;Brazil;Canada;France;Germany;Greece;Israel;Italy;Japan;Netherlands;Spain;United Kingdom;United States;
2020 Phase 2 EUCTR2018-003291-12-DE Belgium;Brazil;Canada;France;Germany;Israel;Japan;Mexico;Netherlands;Spain;United Kingdom;United States;
2019 Phase 2 EUCTR2018-003291-12-NL Argentina;Belgium;Brazil;Canada;France;Germany;Greece;Israel;Italy;Japan;Netherlands;Spain;United Kingdom;United States;
2019 Phase 2 EUCTR2018-003291-12-GB Belgium;Brazil;Canada;France;Germany;Israel;Japan;Mexico;Netherlands;Spain;United Kingdom;United States;
2019 Phase 2 EUCTR2018-003291-12-FR Belgium;Brazil;Canada;Denmark;France;Germany;Israel;Japan;Mexico;Netherlands;Spain;United Kingdom;United States;
2019 Phase 2 EUCTR2018-003291-12-ES Belgium;Brazil;Canada;Denmark;France;Germany;Israel;Japan;Mexico;Netherlands;Spain;United Kingdom;United States;
2019 Phase 2 EUCTR2018-003291-12-BE Belgium;Brazil;Canada;France;Germany;Israel;Japan;Mexico;Netherlands;Spain;United Kingdom;United States;
Shire Human Genetics Therapies Inc
2017 Phase 1;Phase 2 EUCTR2012-003775-20-IT Australia;Brazil;Czech Republic;Denmark;France;Germany;Italy;Japan;United Kingdom;
2016 Phase 1;Phase 2 EUCTR2012-003775-20-GB Australia;Brazil;Czech Republic;Denmark;France;Germany;Italy;Japan;United Kingdom;
2014 Phase 1;Phase 2 EUCTR2012-003775-20-CZ Australia;Brazil;Czech Republic;Denmark;France;Germany;Italy;Japan;United Kingdom;
2013 Phase 1;Phase 2 EUCTR2012-003775-20-DK Australia;Brazil;Czech Republic;Czechia;Denmark;France;Germany;Italy;Japan;United Kingdom;
2013 Phase 1;Phase 2 EUCTR2012-003775-20-DE Australia;Brazil;Czech Republic;Denmark;France;Germany;Italy;Japan;United Kingdom;
SHP675
Shire Human Genetic Therapies, Inc.
2021 Phase 3 EUCTR2018-004689-32-SE Argentina;Australia;Belgium;Bosnia and Herzegovina;Brazil;Canada;Croatia;Finland;Germany;Greece;Hungary;Italy;Latvia;Poland;Portugal;Romania;Slovenia;Spain;Sweden;Ukraine;
2021 Phase 3 EUCTR2018-004689-32-PT Argentina;Australia;Belgium;Bosnia and Herzegovina;Brazil;Canada;Croatia;Finland;Germany;Greece;Hungary;Italy;Latvia;Poland;Portugal;Romania;Slovenia;Spain;Sweden;Ukraine;
2021 Phase 3 EUCTR2018-004689-32-PL Argentina;Australia;Belgium;Bosnia and Herzegovina;Brazil;Canada;Croatia;Finland;Germany;Greece;Hungary;Italy;Latvia;Poland;Portugal;Romania;Slovenia;Spain;Sweden;Ukraine;
2021 Phase 3 EUCTR2018-004689-32-GR Argentina;Australia;Belgium;Bosnia and Herzegovina;Brazil;Canada;Croatia;Finland;Germany;Greece;Hungary;Italy;Latvia;Poland;Portugal;Romania;Slovenia;Spain;Sweden;Ukraine;
2021 Phase 3 EUCTR2018-004689-32-FI Argentina;Australia;Belgium;Bosnia and Herzegovina;Brazil;Canada;Croatia;Finland;Germany;Greece;Hungary;Italy;Latvia;Poland;Portugal;Romania;Slovenia;Spain;Sweden;Ukraine;
SOBI003
Swedish Orphan Biovitrum
2019 Phase 1/Phase 2 NCT03811028 Turkey;United States;
2018 Phase 1/Phase 2 NCT03423186 Germany;Turkey;United States;
Swedish Orphan Biovitrum AB (publ)
2019 Phase 1 EUCTR2017-002806-10-NL Germany;Netherlands;Turkey;United States;
- Phase 1 EUCTR2017-002806-10-DE Germany;Netherlands;Turkey;United States;
ST-920
Sangamo Therapeutics
2021 - NCT05039866 United States;
2019 Phase 1/Phase 2 NCT04046224 United Kingdom;United States;
Sangamo Therapeutics, Inc.
2019 Phase 1;Phase 2 EUCTR2019-000667-24-GB United Kingdom;United States;
SUB195712
IntraBio Ltd
2019 Phase 2 EUCTR2018-004406-25-GB Germany;Spain;United Kingdom;United States;
2019 Phase 2 EUCTR2018-004331-71-GB Germany;Slovakia;Spain;United Kingdom;United States;
Saline Solution for Injection
Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
2017 Phase 1/Phase 2 NCT03153319 United States;
ScAAV9.CB.CLN6
Amicus Therapeutics, Inc.
2021 Phase 1;Phase 2 EUCTR2021-000706-21-DE Germany;United States;
ScAAV9.U1A.SGSH
Abeona Therapeutics Inc
2019 Phase 1;Phase 2 EUCTR2018-000504-42-ES Australia;Spain;United States;
2016 Phase 1;Phase 2 EUCTR2015-003904-21-ES Australia;Spain;United States;
ScAAV9.U1A.hSGSH
Abeona Therapeutics Inc
2020 Phase 1;Phase 2 EUCTR2019-002979-34-ES Australia;France;Germany;Spain;United States;
2019 Phase 1;Phase 2 EUCTR2015-003904-21-FR Australia;Brazil;France;Germany;Spain;United Kingdom;United States;
Sebelipase alfa
Alexion Pharmaceuticals
2015 - NCT02376751 -
2011 Phase 2 NCT01488097 Canada;Czech Republic;Czechia;France;United Kingdom;United States;
2011 Phase 1/Phase 2 NCT01307098 Czech Republic;Czechia;France;United Kingdom;United States;
Alexion Pharmaceuticals Inc.
2013 Phase 2;Phase 3 EUCTR2011-000032-28-IE Egypt;France;Germany;Ireland;Italy;Saudi Arabia;Taiwan;Turkey;United Kingdom;United States;
Alexion Pharmaceuticals, Inc.
2015 Phase 3 EUCTR2011-002750-31-CZ Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Japan;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;United States;
2013 Phase 3 EUCTR2011-002750-31-GB Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Japan;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;United States;
2013 Phase 3 EUCTR2011-002750-31-DE Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Japan;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;United States;
- Phase 3 EUCTR2011-002750-31-PL Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Japan;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;United States;
Synageva BioPharma Corporation
2013 Phase 3 EUCTR2011-002750-31-IT Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;
2013 Phase 3 EUCTR2011-002750-31-HR Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;
2013 Phase 3 EUCTR2011-002750-31-GR Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;
2013 Phase 3 EUCTR2011-002750-31-ES Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;
- Phase 3 EUCTR2011-002750-31-FR Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;
Synageva Biopharma Corp.
2013 - EUCTR2011-000032-28-DE France;Germany;Italy;Taiwan;United Kingdom;United States;
2011 Phase 2 EUCTR2011-001513-13-CZ Czech Republic;France;United Kingdom;United States;
Sebelipase alfa (SBC-102)
Alexion Pharmaceuticals
2011 Phase 2/Phase 3 NCT01371825 Egypt;France;Germany;India;Ireland;Italy;Saudi Arabia;Taiwan;Turkey;United Kingdom;United States;
Sirolimus
National Human Genome Research Institute (NHGRI)
2019 Phase 1/Phase 2 NCT03952637 United States;
Prevail Therapeutics
2021 Phase 1/Phase 2 NCT04411654 United Kingdom;United States;
Skeletal survey
National Human Genome Research Institute (NHGRI)
2019 Phase 1/Phase 2 NCT03952637 United States;
Skin biopsy
National Human Genome Research Institute (NHGRI)
2019 Phase 1/Phase 2 NCT03952637 United States;
Slit Lamp assessment and intra-ocular pressure measurement
Université de Montréal
2014 - NCT02023086 Canada;
Somatropin (DNA origin)
Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
2008 Phase 2/Phase 3 NCT00748969 United States;
Speech and modified barium swallow study
National Human Genome Research Institute (NHGRI)
2019 Phase 1/Phase 2 NCT03952637 United States;
Ss-mercaptoethylamine Hydrochloride & 2-aminoethanethiol Hydrochloride
Recordati Rare Diseases
2020 Phase 3 EUCTR2018-002984-24-IT Belgium;France;Germany;Italy;United Kingdom;
2020 Phase 3 EUCTR2018-002984-24-DE France;Germany;United Kingdom;
- Phase 3 EUCTR2018-002984-24-BE Belgium;France;Germany;Italy;United Kingdom;
Stem Cell Transplant
Masonic Cancer Center, University of Minnesota
2004 Phase 2 NCT00176891 United States;
1999 Phase 2 NCT00176917 United States;
1995 Phase 2/Phase 3 NCT00176904 United States;
Stem Cell Transplantation
Masonic Cancer Center, University of Minnesota
2014 Phase 2 NCT02171104 United States;
2008 Phase 2 NCT00668564 United States;
Surgery to implant human CNS stem cells (HuCNS-SC)
StemCells, Inc.
2006 Phase 1 NCT00337636 United States;
TBD
BioMarin Pharmaceutical Inc.
2015 - EUCTR2014-003480-37-IT Germany;Italy;Japan;United Kingdom;United States;
2014 Phase 1;Phase 2 EUCTR2012-005430-11-IT Germany;Italy;United Kingdom;
2013 Phase 1;Phase 2 EUCTR2012-005430-11-DE Germany;United Kingdom;
TSHA-101
Dr. Anupam Sehgal
2021 Phase 1/Phase 2 NCT04798235 Canada;
Taliglucerase alfa
Pfizer
2011 Phase 3 NCT01422187 -
2011 Phase 3 NCT01411228 Israel;Paraguay;South Africa;
2010 Phase 4 NCT01132690 Israel;Paraguay;South Africa;United States;
2008 Phase 3 NCT00712348 Australia;Canada;Israel;Serbia;Spain;United Kingdom;United States;
2008 Phase 3 NCT00705939 Australia;Canada;Chile;Israel;South Africa;Spain;United Kingdom;United States;
Protalix Biotherapeutics
2009 Phase 3 EUCTR2008-005826-35-GB Italy;Spain;United Kingdom;
2009 Phase 3 EUCTR2008-005825-12-GB Germany;Italy;Spain;United Kingdom;
2009 - EUCTR2008-005825-12-DE Germany;Italy;Spain;United Kingdom;
Taliglucerase alfa - Recombinant human glucocerebrosidase
Protalix Biotherapeutics
- - EUCTR2008-005826-35-Outside-EU/EEA Australia;Canada;Chile;Israel;South Africa;Spain;United Kingdom;United States;
- - EUCTR2008-005825-12-Outside-EU/EEA Canada;Israel;United Kingdom;United States;
- - EUCTR2007-000498-42-Outside-EU/EEA Israel;United States;
Temperature sensitivity measurement with Advanced Thermal Stimulation
Universitaire Ziekenhuizen Leuven
2014 Phase 0 NCT02533076 Belgium;
Thiotepa--escalated dose
University of Florida
2018 Phase 1/Phase 2 NCT03513328 United States;
Thiotepa--single daily dose
University of Florida
2018 Phase 1/Phase 2 NCT03513328 United States;
To be added after EV code is available
Passage Bio, Inc.
- Phase 1;Phase 2 EUCTR2020-005229-95-NL Brazil;Canada;Israel;Netherlands;United Kingdom;United States;
Total body Irradiation
Masonic Cancer Center, University of Minnesota
2006 Phase 2 NCT00383448 United States;
Trappsol Cyclo
CTD Holdings, Inc.
2016 Phase 1;Phase 2 EUCTR2015-005761-23-GB Italy;Sweden;United Kingdom;
Cyclo Therapeutics, Inc.
2017 Phase 1;Phase 2 EUCTR2015-005761-23-SE Italy;Sweden;United Kingdom;
Trappsol® Cyclo™
Cyclo Therapeutics, Inc.
2022 Phase 3 EUCTR2020-003136-25-PL Australia;France;Germany;Israel;Italy;Poland;United Kingdom;United States;
2022 Phase 3 EUCTR2020-003136-25-DE Australia;France;Germany;Israel;Italy;Poland;United Kingdom;United States;
2021 Phase 3 EUCTR2020-003136-25-FR Australia;France;Israel;United Kingdom;United States;
- Phase 3 EUCTR2020-003136-25-IT Australia;France;Germany;Israel;Italy;Poland;Spain;United Kingdom;United States;
Treosulfan
Tokai University School of Medicine
2013 Phase 1 JPRN-UMIN000009758 Japan;
Triapin& triapin Mite
Salford Royal NHS Foundation Trust
2009 Phase 4 EUCTR2007-007482-21-GB United Kingdom;
Tropicamide
Université de Montréal
2014 - NCT02023086 Canada;
UX003
Ultragenyx Pharmaceutical Inc
2015 Phase 3 NCT02432144 Brazil;Mexico;Portugal;United States;
2015 Phase 2 NCT02418455 Portugal;Spain;United States;
2014 Phase 3 NCT02230566 United States;
Ultragenyx Pharmaceutical Inc.
2016 Phase 3 EUCTR2015-001875-32-PT Brazil;Mexico;Portugal;United States;
2016 Phase 2 EUCTR2015-000104-26-PT Portugal;Spain;United States;
2016 Phase 2 EUCTR2015-000104-26-ES Portugal;Spain;United States;
2014 Phase 1;Phase 2 EUCTR2013-001152-35-ES Spain;United Kingdom;
2013 Phase 1;Phase 2 EUCTR2013-001152-35-GB Spain;United Kingdom;
- - EUCTR2015-001875-32-Outside-EU/EEA United States;
- - EUCTR2015-000104-26-Outside-EU/EEA United States;
- - EUCTR2014-005638-71-Outside-EU/EEA United States;
Unrelated Umbilical Cord Blood Transplant
Duke University
2008 Phase 1 NCT00744692 United States;
Urine collection
Baylor Research Institute
2018 - NCT04002531 United States;
VPRIV - 400 U - POLVERE PER SOLUZIONE PER INFUSIONE - USO ENDOVENOSO - FLACONCINO(VETRO) 1 FLACONCINO
SHIRE HUMAN GENETIC THERAPIES, INC
2018 Phase 4 EUCTR2015-001578-17-IT Germany;Israel;Italy;Spain;United Kingdom;United States;
VPRIV 400 Units powder for solution of infusion
Shire Human Genetic Therapies, Inc.
2016 Phase 4 EUCTR2015-001578-17-GB Canada;Germany;Israel;Spain;United Kingdom;United States;
2016 Phase 4 EUCTR2015-001578-17-DE Germany;India;Israel;Italy;Spain;Turkey;United Kingdom;United States;
2016 - EUCTR2015-001578-17-ES Canada;Germany;Israel;Italy;Spain;United Kingdom;United States;
VPRIV ®,
Shire
2007 Phase 3 NCT00430625 Argentina;Israel;Paraguay;Russian Federation;Tunisia;United States;
VPRIV®
Shire
2008 Phase 3 NCT00635427 Argentina;India;Israel;Korea, Republic of;Paraguay;Poland;Russian Federation;Spain;Tunisia;United Kingdom;United States;
VTS-270
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
2020 Phase 1/Phase 2 NCT03887533 United States;
Mandos LLC
2019 Phase 2/Phase 3 NCT03879655 Costa Rica;
Vtesse, Inc., a Mallinckrodt Pharmaceuticals Company
2020 Phase 2 NCT03687476 -
Vtesse, LLC, a Mallinckrodt Pharmaceuticals Company
2013 Phase 1 NCT01747135 United States;
Vector viral adenoasociado de serotipo 9 que contiene el gen de la sulfamidasa humana
Laboratorios del Dr. Esteve, S.A.
2016 Phase 1;Phase 2 EUCTR2015-000359-26-ES Spain;
Velaglucerase alfa
Baylor Research Institute
2015 Phase 4 NCT02528617 United States;
SHIRE HUMAN GENETIC THERAPIES, INC
2018 Phase 4 EUCTR2015-001578-17-IT Germany;Israel;Italy;Spain;United Kingdom;United States;
Shire
2016 Phase 4 NCT02574286 Israel;Spain;United Kingdom;United States;
2013 Phase 3 NCT01842841 Japan;
2012 Phase 3 NCT01614574 Japan;
2012 Phase 1/Phase 2 NCT01685216 Egypt;India;Tunisia;
2009 - NCT00954460 United States;
2008 Phase 3 NCT00553631 Argentina;India;Israel;Paraguay;Russian Federation;Spain;Tunisia;United Kingdom;United States;
Shire Human Genetic Therapies Inc
- Phase 2;Phase 3 EUCTR2006-006304-11-DE Germany;Italy;Spain;United Kingdom;
Shire Human Genetic Therapies, Inc.
2008 - EUCTR2008-001965-27-ES Spain;United Kingdom;
2007 - EUCTR2007-002840-21-GB Italy;Spain;United Kingdom;
Takeda
2021 Phase 4 NCT04718779 United States;
Velaglucerase alfa (VPRIV)
Shire
2021 - NCT04429984 India;
Velaglucerasi alfa
SHIRE HUMAN GENETIC THERAPIES, INC
2018 Phase 4 EUCTR2015-001578-17-IT Germany;Israel;Italy;Spain;United Kingdom;United States;
Velmanase Alfa (e.g. Lamazym)
Chiesi Farmaceutici S.p.A.
2016 Phase 2 NCT02998879 Austria;Denmark;France;Germany;Italy;
Velmanase alfa
Chiesi Farmaceutici S.p.A.
2015 Phase 3 EUCTR2013-000336-97-DK Denmark;France;
Venglustat
GENZYME CORPORATION
2020 Phase 3 EUCTR2019-002375-34-IT Argentina;Austria;Brazil;Czech Republic;Czechia;France;Germany;Italy;Japan;Portugal;Russian Federation;Spain;Turkey;United Kingdom;United States;
Genzyme Corporation
2021 Phase 3 EUCTR2019-002375-34-AT Argentina;Austria;Brazil;Czech Republic;Czechia;France;Germany;Italy;Japan;Portugal;Russian Federation;Spain;Turkey;United Kingdom;United States;
2020 Phase 3 EUCTR2019-002375-34-PT Argentina;Austria;Brazil;Czech Republic;Czechia;France;Germany;Italy;Japan;Portugal;Russian Federation;Spain;Turkey;United Kingdom;United States;
2020 Phase 3 EUCTR2019-002375-34-GB Argentina;Austria;Brazil;Czech Republic;France;Germany;Italy;Japan;Portugal;Russian Federation;Spain;Turkey;United Kingdom;United States;
2020 Phase 3 EUCTR2019-002375-34-DE Argentina;Austria;Brazil;Czech Republic;France;Germany;Italy;Japan;Portugal;Russian Federation;Spain;Turkey;United Kingdom;United States;
2020 Phase 3 EUCTR2019-002375-34-CZ Argentina;Austria;Brazil;Czech Republic;France;Germany;Italy;Japan;Portugal;Russian Federation;Spain;Turkey;United Kingdom;United States;
2016 Phase 2 EUCTR2014-002550-39-DE Germany;Japan;United Kingdom;United States;
- Phase 3 EUCTR2019-002375-34-FR Argentina;Austria;Brazil;Czech Republic;France;Germany;Italy;Japan;Portugal;Russian Federation;Spain;Turkey;United Kingdom;United States;
Genzyme, a Sanofi Company
2022 Phase 3 NCT05222906 -
Tanaka Tomoyuki
2020 Phase 3 JPRN-jRCT2021200023 Argentina;Austria;Brazil;Czechia;France;Germany;Italy;Japan;Portugal;Russian Federation;Spain;Turkey;United Kingdom;United States;
Venglustat (GZ402671)
Genzyme, a Sanofi Company
2022 Phase 3 NCT05206773 United States;
2017 Phase 2 NCT02843035 Germany;Japan;United Kingdom;United States;
Venglustat GZ402671
Genzyme, a Sanofi Company
2020 Phase 3 NCT04221451 Argentina;Austria;Brazil;Czechia;France;Germany;Italy;Japan;Portugal;Russian Federation;Spain;Turkey;United Kingdom;United States;
Venglustat malate
Genzyme Corporation
2021 Phase 2;Phase 3 EUCTR2020-003120-17-HU Argentina;Brazil;Canada;China;Egypt;France;Germany;Hungary;Japan;Sweden;Taiwan;Turkey;United States;
2021 Phase 2;Phase 3 EUCTR2020-003120-17-FR Argentina;Brazil;Canada;China;Egypt;France;Germany;Hungary;Japan;Sweden;Taiwan;Turkey;United States;
2021 Phase 2;Phase 3 EUCTR2020-003120-17-DE Argentina;Brazil;Canada;China;Egypt;France;Germany;Hungary;Japan;Sweden;Taiwan;Turkey;United States;
- Phase 2;Phase 3 EUCTR2020-003120-17-SE Argentina;Brazil;Canada;China;Egypt;France;Germany;Hungary;Japan;Sweden;Taiwan;Turkey;United States;
Sanofi-Aventis Recherche & Développement
2021 Phase 3 EUCTR2021-002350-90-GR Argentina;Austria;Brazil;Bulgaria;Canada;China;Denmark;Finland;France;Germany;Greece;Italy;Mexico;Netherlands;Norway;Poland;Romania;Russian Federation;Turkey;Ukraine;United Kingdom;
Sanofi-aventis recherche & développement
- Phase 3 EUCTR2021-005402-10-FR Argentina;Brazil;Canada;China;Egypt;France;Germany;Hungary;Japan;Taiwan;Turkey;United Kingdom;United States;
- Phase 3 EUCTR2021-005402-10-DE Argentina;Brazil;Canada;China;Egypt;France;Germany;Hungary;Japan;Taiwan;Turkey;United Kingdom;United States;
sanofi-aventis recherche et développement
- Phase 3 EUCTR2021-002320-20-ES Austria;Canada;China;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Turkey;United Kingdom;United States;
Venglustat malato
SANOFI-AVENTIS RECHERCHE E DEVELOPPEMENT
2022 Phase 3 EUCTR2021-002320-20-IT Austria;Canada;China;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Turkey;United Kingdom;United States;
2021 Phase 3 EUCTR2021-002350-90-IT Argentina;Austria;Brazil;Bulgaria;Canada;China;Denmark;Finland;France;Germany;Greece;Italy;Mexico;Netherlands;Norway;Poland;Romania;Russian Federation;Turkey;Ukraine;United Kingdom;
Vestronidase alfa
Hamazaki Takashi
2020 Phase 3 JPRN-jRCT2051190085 Japan;
Vimizim® (elosulfase alfa)
BioMarin Pharmaceutical
2014 - NCT02294877 Australia;Austria;Belgium;Canada;Czechia;Denmark;France;Germany;Ireland;Italy;Malaysia;Netherlands;Poland;Portugal;Puerto Rico;Taiwan;United Kingdom;United States;
Visual field testing
Université de Montréal
2014 - NCT02023086 Canada;
Vitamin D
Shire
2016 Phase 4 NCT02574286 Israel;Spain;United Kingdom;United States;
Vorinostat
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
2014 Phase 1/Phase 2 NCT02124083 United States;
Warfarin
The Cooper Health System
2007 - NCT00683189 United States;
Water
Amicus Therapeutics
2011 Phase 1 NCT01853852 Australia;
Whole body Protein turnover
University of Texas Southwestern Medical Center
2006 - NCT00286689 United States;
ZAVESCA 84CPS
Actelion Registration Ltd
2006 - EUCTR2005-001651-37-IT Czech Republic;Germany;Hungary;Italy;Spain;United Kingdom;
ZESTORIC
Salford Royal NHS Foundation Trust
2009 Phase 4 EUCTR2007-007482-21-GB United Kingdom;
Zavesca
Actelion Pharmaceuticals Ltd
2007 - EUCTR2005-001651-37-HU Czech Republic;Germany;Hungary;Italy;Spain;United Kingdom;
2006 - EUCTR2005-001651-37-ES Czech Republic;Germany;Hungary;Italy;Spain;United Kingdom;
2006 - EUCTR2005-001651-37-CZ Czech Republic;Germany;Hungary;Italy;Spain;United Kingdom;
2005 Phase 4 EUCTR2005-001651-37-GB Czech Republic;Germany;Hungary;Italy;Spain;United Kingdom;
Orphazyme ApS
2015 Phase 1 EUCTR2014-005194-37-ES Germany;Spain;Switzerland;
2015 Phase 1 EUCTR2014-005194-37-DE Germany;Spain;Switzerland;
Zavesca (Miglustat)
Children's Research Institute
2004 Phase 3 NCT00672022 United States;
Zestoretic 10,20
Salford Royal NHS Foundation Trust
2009 Phase 4 EUCTR2007-007482-21-GB United Kingdom;
Zestril
Salford Royal NHS Foundation Trust
2009 Phase 4 EUCTR2007-007482-21-GB United Kingdom;
[14C] AT1001
Amicus Therapeutics
2011 Phase 1 NCT01730482 -
sebelipase alpha
Alexion Pharmaceuitcals Inc
2014 Phase 2 EUCTR2011-004287-30-HR Australia;Belgium;Brazil;Canada;Croatia;Denmark;Germany;Italy;Japan;Mexico;Netherlands;Russian Federation;Spain;Turkey;United Kingdom;United States;
2014 Phase 2 EUCTR2011-004287-30-DE Australia;Belgium;Brazil;Canada;Croatia;Denmark;Germany;Italy;Japan;Mexico;Netherlands;Russian Federation;Spain;Turkey;United Kingdom;United States;
Alexion Pharmaceuticals Inc
2015 Phase 2 EUCTR2014-000533-22-FI Finland;Italy;United Kingdom;United States;
2015 Phase 2 EUCTR2011-004287-30-NL Australia;Belgium;Brazil;Canada;Croatia;Denmark;Germany;Italy;Mexico;Netherlands;Russian Federation;Spain;Turkey;United Kingdom;United States;
2015 Phase 2 EUCTR2011-004287-30-GB Australia;Belgium;Brazil;Canada;Croatia;Denmark;Germany;Italy;Japan;Mexico;Netherlands;Poland;Russian Federation;Spain;Turkey;United Kingdom;United States;
2014 Phase 2 EUCTR2014-000533-22-GB Finland;Italy;United Kingdom;United States;
Alexion Pharmaceuticals inc
2014 Phase 2 EUCTR2011-004287-30-DK Australia;Belgium;Brazil;Canada;Croatia;Denmark;Germany;Italy;Japan;Mexico;Netherlands;Poland;Russian Federation;Spain;Turkey;United Kingdom;United States;
Alexion Pharmaceuticals, Inc.
2015 Phase 3 EUCTR2011-002750-31-CZ Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Japan;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;United States;
2013 Phase 3 EUCTR2011-002750-31-GB Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Japan;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;United States;
2013 Phase 3 EUCTR2011-002750-31-DE Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Japan;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;United States;
- Phase 3 EUCTR2011-002750-31-PL Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Japan;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;United States;
Synageva BioPharma Corp.
2014 Phase 2 EUCTR2014-000533-22-IT Finland;Italy;United Kingdom;United States;
2014 Phase 2 EUCTR2011-004287-30-IT Australia;Belgium;Brazil;Canada;Croatia;Denmark;Germany;Italy;Japan;Mexico;Netherlands;Poland;Russian Federation;Spain;Turkey;United Kingdom;United States;
2014 Phase 2 EUCTR2011-004287-30-BE Australia;Belgium;Brazil;Canada;Croatia;Denmark;Germany;Italy;Japan;Mexico;Netherlands;Russian Federation;Spain;Turkey;United Kingdom;United States;
Synageva BioPharma Corporation
2013 Phase 3 EUCTR2011-002750-31-IT Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;
2013 Phase 3 EUCTR2011-002750-31-HR Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;
2013 Phase 3 EUCTR2011-002750-31-GR Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;
2013 Phase 3 EUCTR2011-002750-31-ES Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;
- Phase 3 EUCTR2011-002750-31-FR Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;
Recordati Rare Diseases
2020 Phase 3 EUCTR2018-002984-24-IT Belgium;France;Germany;Italy;United Kingdom;
1- Deoxygalactonojirimycin
Amicus Therapeutics
2005 Phase 2 EUCTR2005-004384-33-GB United Kingdom;
1-deoxygalactonojirimycin hydrochloride
AMICUS THERAPEUTICS, INC
2011 - EUCTR2010-022636-37-IT Argentina;Australia;Austria;Belgium;Brazil;Denmark;Germany;Greece;Italy;Japan;Mexico;Russian Federation;Switzerland;Taiwan;Turkey;United Kingdom;United States;
Amicus Therapeutics, Inc.
2012 - EUCTR2010-022709-16-GB Belgium;United Kingdom;
2012 - EUCTR2010-022636-37-DE Argentina;Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Hungary;Israel;Italy;Japan;Mexico;Poland;Russian Federation;Slovakia;Switzerland;Taiwan;Turkey;United Kingdom;United States;
2011 Phase 3 EUCTR2010-022636-37-GR Argentina;Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Hungary;Israel;Italy;Japan;Mexico;Poland;Russian Federation;Slovakia;Switzerland;Taiwan;Turkey;United Kingdom;United States;
2011 Phase 2 EUCTR2010-022709-16-BE Australia;Belgium;Canada;France;Netherlands;Switzerland;United Kingdom;
2011 - EUCTR2010-022636-37-GB Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;United Kingdom;United States;
2011 - EUCTR2010-022636-37-DK Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;United Kingdom;United States;
2011 - EUCTR2010-022636-37-BE Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;United Kingdom;United States;
2011 - EUCTR2010-022636-37-AT Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;United Kingdom;United States;
2010 Phase 3 EUCTR2009-013459-31-ES Belgium;Denmark;France;Germany;Italy;Netherlands;Spain;United Kingdom;
2010 Phase 3 EUCTR2009-013459-31-BE Belgium;Denmark;France;Germany;Italy;Netherlands;Spain;United Kingdom;
2010 - EUCTR2009-013459-31-NL Belgium;Denmark;France;Germany;Italy;Netherlands;Spain;United Kingdom;
2010 - EUCTR2009-013459-31-DK Argentina;Australia;Belgium;Brazil;Canada;Denmark;Egypt;France;Germany;Italy;Netherlands;South Africa;Spain;Turkey;United Kingdom;United States;
2009 Phase 3 EUCTR2009-013459-31-GB Argentina;Australia;Belgium;Brazil;Canada;Denmark;Egypt;France;Germany;Italy;Netherlands;South Africa;Spain;Turkey;United Kingdom;United States;
2009 Phase 3 EUCTR2009-013459-31-FR Belgium;Denmark;France;Germany;Italy;Netherlands;Spain;United Kingdom;
2009 Phase 2 EUCTR2007-001838-13-GB Australia;Brazil;United Kingdom;United States;
2006 Phase 2 EUCTR2006-000181-36-GB United Kingdom;
- - EUCTR2009-013459-31-DE Belgium;Denmark;France;Germany;Italy;Netherlands;Spain;United Kingdom;
12 lead electrocardiogram
Baylor Research Institute
2018 - NCT04002531 United States;
12S-IT
Shire HGT Inc
2012 Phase 1;Phase 2 EUCTR2011-000212-25-GB Canada;United Kingdom;United States;
15 O Water
National Institute of Neurological Disorders and Stroke (NINDS)
1994 - NCT00001972 United States;
2(S)-(ACETYLAMINO)-4-METHYLPENTANOIC ACID
IntraBio Ltd
2022 Phase 3 EUCTR2021-005356-10-SK Australia;Czechia;Germany;Netherlands;Slovakia;Switzerland;United Kingdom;United States;
2022 Phase 3 EUCTR2021-005356-10-DE Australia;Czechia;Germany;Netherlands;Slovakia;Spain;Switzerland;United Kingdom;United States;
2019 Phase 2 EUCTR2018-004406-25-ES Germany;Spain;United Kingdom;United States;
2019 Phase 2 EUCTR2018-004406-25-DE Germany;Spain;United Kingdom;United States;
2019 Phase 2 EUCTR2018-004331-71-SK Germany;Slovakia;Spain;United Kingdom;United States;
2019 Phase 2 EUCTR2018-004331-71-ES Germany;Slovakia;Spain;United Kingdom;United States;
2019 Phase 2 EUCTR2018-004331-71-DE Germany;Slovakia;Spain;United Kingdom;United States;
- Phase 3 EUCTR2021-005356-10-NL Australia;Czechia;Germany;Netherlands;Slovakia;Spain;Switzerland;United Kingdom;United States;
2-HYDROXYPROPYL-BETA-CYCLODEXTRIN
Jichi Medical University, Saitama Medical Center
2014 - JPRN-UMIN000020032 Japan;
Nara Medical University
2016 - JPRN-UMIN000023517 Japan;
2014 - JPRN-UMIN000023094 Japan;
2-hour Holter Monitor
Baylor Research Institute
2018 - NCT04002531 United States;
4D-310
4D Molecular Therapeutics
2020 Phase 1/Phase 2 NCT04519749 United States;
500 0500 0
Manchester University NHS Foundation Trust
2014 Phase 3 EUCTR2013-001479-18-GB United Kingdom;
89 Zirconium Recombinant Human Alpha-N-Acetylglucosaminidase (rhNAGLU) (radiolabeled)
Alexion Pharmaceuticals, Inc.
2016 Phase 2 EUCTR2015-000753-20-NL Netherlands;
89Zr-SBC-103
Alexion Pharmaceuticals, Inc.
2016 Phase 2 EUCTR2015-000753-20-NL Netherlands;
A (migalastat)
Amicus Therapeutics
2011 Phase 1 NCT01489995 United States;
A16AB04
FONDAZIONE POLICLINICO UNIVERSITARIO AGOSTINO GEMELLI IRCCS UNIVERSITA' CATTOLICA DEL SACRO CUORE
2018 Phase 4 EUCTR2018-000368-27-IT Italy;
A16AX06
Orphazyme ApS
2015 Phase 1 EUCTR2014-005194-37-ES Germany;Spain;Switzerland;
2015 Phase 1 EUCTR2014-005194-37-DE Germany;Spain;Switzerland;
AAV2/8.TBG.hARSB
FONDAZIONE TELETHON
2018 Phase 1 EUCTR2016-002328-10-NL Italy;Netherlands;Turkey;
2017 Phase 1 EUCTR2016-002328-10-IT Italy;Netherlands;Turkey;
Fondazione Telethon
2017 Phase 1/Phase 2 NCT03173521 Italy;Netherlands;Turkey;
AAV2CUhCLN2 (3x10^12 particle units)
Weill Medical College of Cornell University
2004 Phase 1 NCT00151216 United States;
AAV9-CAG-coh-SGSH
Laboratorios del Dr. Esteve, S.A.
2016 Phase 1;Phase 2 EUCTR2015-000359-26-ES Spain;
AAV9-GLB1
National Human Genome Research Institute (NHGRI)
2019 Phase 1/Phase 2 NCT03952637 United States;
AAVrh.10-hMPSIIIA
SANFILIPPO Therapeutics SAS
- - EUCTR2010-019962-10-FR France;
AAVrh.10CUCLN2
Weill Medical College of Cornell University
2010 Phase 1/Phase 2 NCT01414985 United States;
AAVrh.10CUhCLN2 vector 2.85x10^11 genome copies
Weill Medical College of Cornell University
2010 Phase 1 NCT01161576 United States;
AAVrh.10CUhCLN2 vector 9.0x10^11 genome copies
Weill Medical College of Cornell University
2010 Phase 1 NCT01161576 United States;
AAVrh.10cuARSA
Inserm
2012 - EUCTR2011-004410-42-FR France;
ABO-101
Abeona Therapeutics Europe SL.
2021 Phase 1;Phase 2 EUCTR2019-002936-97-FR France;Germany;United States;
2019 Phase 1;Phase 2 EUCTR2014-001411-39-GB France;Germany;Italy;Netherlands;Spain;United Kingdom;United States;
2019 Phase 1;Phase 2 EUCTR2014-001411-39-DE France;Germany;Spain;United Kingdom;United States;
- Phase 1;Phase 2 EUCTR2019-002936-97-DE France;Germany;Spain;United States;
Abeona Therapeutics, Inc
2020 - NCT04655911 France;Germany;United States;
ABO-102
Abeona Therapeutics Inc
2020 Phase 1;Phase 2 EUCTR2019-002979-34-ES Australia;France;Germany;Spain;United States;
2019 Phase 1;Phase 2 EUCTR2018-000504-42-ES Australia;Spain;United States;
2019 Phase 1;Phase 2 EUCTR2015-003904-21-FR Australia;Brazil;France;Germany;Spain;United Kingdom;United States;
Abeona Therapeutics, Inc
2020 - NCT04360265 Australia;Spain;United States;
2019 Phase 1/Phase 2 NCT04088734 Australia;Spain;United States;
2016 Phase 1/Phase 2 NCT02716246 Australia;France;Germany;Spain;United States;
ACT-434964
IDORSIA PHARMACEUTICALS LTD
2020 Phase 3 EUCTR2018-002210-12-IT Australia;Austria;Belgium;Canada;France;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;United Kingdom;United States;
2020 Phase 3 EUCTR2017-003369-85-IT Australia;Austria;Belgium;Canada;Finland;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;Switzerland;United Kingdom;United States;
Idorsia Pharmaceuticals Japan Ltd
2019 Phase 3 JPRN-JapicCTI-194731 Japan;
Idorsia Pharmaceuticals Ltd
2021 Phase 3 EUCTR2018-002210-12-NO Australia;Austria;Belgium;Canada;France;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;United Kingdom;United States;
2021 Phase 3 EUCTR2018-002210-12-FR Austria;Belgium;Canada;Czech Republic;France;Germany;Netherlands;Poland;United Kingdom;United States;
2020 Phase 3 EUCTR2018-002210-12-BE Australia;Austria;Belgium;Canada;France;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;United Kingdom;United States;
2020 Phase 3 EUCTR2017-003369-85-NO Australia;Austria;Belgium;Canada;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;Switzerland;United Kingdom;United States;
2020 Phase 3 EUCTR2017-003369-85-IE Australia;Austria;Belgium;Canada;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;Switzerland;United Kingdom;United States;
2020 Phase 3 EUCTR2017-003369-85-ES Australia;Austria;Belgium;Canada;Germany;Ireland;Italy;Netherlands;Norway;Poland;Russian Federation;Spain;Switzerland;United Kingdom;United States;
2019 Phase 3 EUCTR2018-002210-12-NL Australia;Austria;Belgium;Canada;France;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;United Kingdom;United States;
2019 Phase 3 EUCTR2018-002210-12-GB Australia;Austria;Belgium;Canada;Germany;Ireland;Netherlands;Poland;United Kingdom;United States;
2019 Phase 3 EUCTR2018-002210-12-DE Australia;Austria;Belgium;Canada;France;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;United Kingdom;United States;
2019 Phase 3 EUCTR2018-002210-12-AT Australia;Austria;Belgium;Canada;France;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;United Kingdom;United States;
2018 Phase 3 EUCTR2017-003369-85-NL Australia;Austria;Belgium;Canada;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;Switzerland;United Kingdom;United States;
2018 Phase 3 EUCTR2017-003369-85-GB Australia;Austria;Belgium;Canada;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;Switzerland;United Kingdom;United States;
2018 Phase 3 EUCTR2017-003369-85-AT Australia;Austria;Belgium;Canada;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;Switzerland;United Kingdom;United States;
ADENO-ASSOCIATED VIRAL (AAV) SEROTYPE 8 (AAV2/8) VECTOR WITH LIVER-SPECIFIC THYROXINE-BINDING GLOBULIN (TBG) PROMOTER, DRIVING THE EXPRESSION OF THE HUMAN ARSB GENE
FONDAZIONE TELETHON
2018 Phase 1 EUCTR2016-002328-10-NL Italy;Netherlands;Turkey;
ADMINISTRATION
Osaka City University HospitalTakashi Hamazaki
2019 Phase 2 JPRN-JMA-IIA00416 Japan;
Tottori University Hospital, Departrment of Child Neurology
2019 Phase 2-3 JPRN-JMA-IIA00421 Japan;
AGALSIDASI ALFA
SANOFI-AVENTIS RECHERCHE E DEVELOPPEMENT
2022 Phase 3 EUCTR2021-002320-20-IT Austria;Canada;China;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Turkey;United Kingdom;United States;
AGALSIDASI BETA
FONDAZIONE POLICLINICO UNIVERSITARIO AGOSTINO GEMELLI IRCCS UNIVERSITA' CATTOLICA DEL SACRO CUORE
2018 Phase 4 EUCTR2018-000368-27-IT Italy;
SANOFI-AVENTIS RECHERCHE E DEVELOPPEMENT
2022 Phase 3 EUCTR2021-002320-20-IT Austria;Canada;China;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Turkey;United Kingdom;United States;
AGT-181
ArmaGen, Inc
2016 Phase 1/Phase 2 NCT03071341 Brazil;
2015 Phase 1/Phase 2 NCT03053089 Brazil;
2015 Phase 1 NCT02597114 -
AGT-181 (HIRMAb-IDUA)
ArmaGen, Inc
2015 Phase 1 NCT02371226 United States;
AGT-182
ArmaGen, Inc
2015 Phase 1 NCT02262338 Germany;Netherlands;Philippines;United States;
ALDURAZYME*INFUS 1FL 5ML 500U
Genzyme Europe BV
2010 - EUCTR2007-007003-33-IT Italy;
ARIMOCLOMOL CITRATE
Orphazyme A/S
2016 Phase 2;Phase 3 EUCTR2015-004438-93-GB Denmark;Germany;Italy;Poland;Spain;Switzerland;United Kingdom;United States;
AT-GTX-501
Amicus Therapeutics, Inc.
2021 Phase 1;Phase 2 EUCTR2021-000706-21-DE Germany;United States;
AT1001
AMICUS THERAPEUTICS, INC
2011 - EUCTR2010-022636-37-IT Argentina;Australia;Austria;Belgium;Brazil;Denmark;Germany;Greece;Italy;Japan;Mexico;Russian Federation;Switzerland;Taiwan;Turkey;United Kingdom;United States;
Amicus Therapeutics
2011 Phase 1 NCT01730469 United States;
2005 Phase 2 EUCTR2005-004384-33-GB United Kingdom;
Amicus Therapeutics UK, Ltd
2020 Phase 3 EUCTR2017-000146-21-IT Australia;Germany;Italy;Spain;United Kingdom;United States;
Amicus Therapeutics, Inc.
2015 Phase 3 EUCTR2014-002701-38-GB Argentina;Australia;Austria;Belgium;Brazil;Canada;Denmark;Egypt;France;Italy;Japan;Spain;Turkey;United Kingdom;United States;
2015 Phase 3 EUCTR2014-002701-38-ES Argentina;Australia;Austria;Belgium;Brazil;Canada;Denmark;Egypt;France;Italy;Japan;Spain;Turkey;United Kingdom;United States;
2015 Phase 3 EUCTR2014-002701-38-DK Argentina;Australia;Austria;Belgium;Brazil;Canada;Denmark;Egypt;France;Italy;Japan;Spain;Turkey;United Kingdom;United States;
2015 Phase 3 EUCTR2014-002701-38-BE Argentina;Australia;Austria;Belgium;Brazil;Canada;Denmark;Egypt;France;Italy;Japan;Spain;Turkey;United Kingdom;United States;
2014 Phase 3 EUCTR2014-002701-38-AT Argentina;Australia;Austria;Belgium;Brazil;Canada;Denmark;Egypt;France;Italy;Japan;Spain;Turkey;United Kingdom;United States;
2014 Phase 3 EUCTR2011-004800-40-AT Argentina;Australia;Austria;Belgium;Brazil;Canada;Denmark;Egypt;France;Italy;Spain;Turkey;United Kingdom;United States;
2013 Phase 3 EUCTR2011-004800-40-BE Argentina;Australia;Austria;Belgium;Brazil;Canada;Denmark;Egypt;France;Italy;Spain;Turkey;United Kingdom;United States;
2012 Phase 3 EUCTR2011-004800-40-GB Argentina;Australia;Austria;Belgium;Brazil;Canada;Denmark;Egypt;France;Italy;Spain;Turkey;United Kingdom;United States;
2012 Phase 3 EUCTR2011-004800-40-DK Argentina;Australia;Austria;Belgium;Brazil;Canada;Denmark;Egypt;France;Italy;Spain;Turkey;United Kingdom;United States;
2012 - EUCTR2010-022709-16-GB Belgium;United Kingdom;
2012 - EUCTR2010-022636-37-DE Argentina;Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Hungary;Israel;Italy;Japan;Mexico;Poland;Russian Federation;Slovakia;Switzerland;Taiwan;Turkey;United Kingdom;United States;
2011 Phase 3 EUCTR2010-022636-37-GR Argentina;Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Hungary;Israel;Italy;Japan;Mexico;Poland;Russian Federation;Slovakia;Switzerland;Taiwan;Turkey;United Kingdom;United States;
2011 Phase 2 EUCTR2010-022709-16-BE Australia;Belgium;Canada;France;Netherlands;Switzerland;United Kingdom;
2011 - EUCTR2010-022636-37-GB Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;United Kingdom;United States;
2011 - EUCTR2010-022636-37-DK Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;United Kingdom;United States;
2011 - EUCTR2010-022636-37-BE Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;United Kingdom;United States;
2011 - EUCTR2010-022636-37-AT Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;United Kingdom;United States;
2010 Phase 3 EUCTR2009-013459-31-ES Belgium;Denmark;France;Germany;Italy;Netherlands;Spain;United Kingdom;
2010 Phase 3 EUCTR2009-013459-31-BE Belgium;Denmark;France;Germany;Italy;Netherlands;Spain;United Kingdom;
2010 - EUCTR2009-013459-31-NL Belgium;Denmark;France;Germany;Italy;Netherlands;Spain;United Kingdom;
2010 - EUCTR2009-013459-31-DK Argentina;Australia;Belgium;Brazil;Canada;Denmark;Egypt;France;Germany;Italy;Netherlands;South Africa;Spain;Turkey;United Kingdom;United States;
2009 Phase 3 EUCTR2009-013459-31-GB Argentina;Australia;Belgium;Brazil;Canada;Denmark;Egypt;France;Germany;Italy;Netherlands;South Africa;Spain;Turkey;United Kingdom;United States;
2009 Phase 3 EUCTR2009-013459-31-FR Belgium;Denmark;France;Germany;Italy;Netherlands;Spain;United Kingdom;
2009 Phase 2 EUCTR2007-001838-13-GB Australia;Brazil;United Kingdom;United States;
2006 Phase 2 EUCTR2006-000181-36-GB United Kingdom;
- - EUCTR2009-013459-31-DE Belgium;Denmark;France;Germany;Italy;Netherlands;Spain;United Kingdom;
Amicus Therapeutics, UK Limited
2020 Phase 3 EUCTR2019-000222-21-GB United Kingdom;United States;
Amicus Therapeutics, UK Ltd
2019 Phase 3 EUCTR2017-000146-21-GB Australia;Germany;Italy;Spain;United Kingdom;United States;
2019 Phase 3 EUCTR2017-000146-21-ES Australia;Germany;Italy;Spain;United Kingdom;United States;
- Phase 3 EUCTR2017-000146-21-DE Australia;Germany;Italy;Spain;United Kingdom;United States;
AT2101
Amicus Therapeutics
2009 Phase 1 NCT00875160 United Kingdom;United States;
Amicus Therapeutics, Inc.
2009 - EUCTR2008-007158-36-GB United Kingdom;
2007 - EUCTR2007-003359-35-GB Germany;United Kingdom;
2007 - EUCTR2007-003359-35-DE Germany;United Kingdom;
AUTOLOGOUS CD34+ CELLS TRANSDUCED WITH A LENTIVIRAL VECTOR CONTAINING THE HUMAN SGSH GENE
University of Manchester
2019 Phase 1;Phase 2 EUCTR2019-002051-42-GB United Kingdom;
AVR-RD-01
AvroBio
2018 Phase 1/Phase 2 NCT03454893 Australia;Canada;United States;
AVR-RD-02
AvroBio
2019 Phase 1/Phase 2 NCT04145037 Australia;Canada;United States;
AX 250
Allievex Corporation
2018 Phase 2 NCT03784287 Colombia;Germany;Spain;Taiwan;Turkey;United Kingdom;United States;
2018 Phase 2 EUCTR2017-003083-13-GB Colombia;Germany;Spain;Taiwan;Turkey;United Kingdom;United States;
2016 Phase 1;Phase 2 EUCTR2015-001985-25-DE Australia;Colombia;Germany;Spain;Taiwan;Turkey;United Kingdom;
2016 Phase 1/Phase 2 NCT02754076 Colombia;Germany;Spain;Taiwan;Turkey;United Kingdom;United States;
2015 Phase 1;Phase 2 EUCTR2015-001985-25-GB Australia;Colombia;Germany;Spain;Taiwan;Turkey;United Kingdom;
- Phase 2 EUCTR2017-003083-13-DE Colombia;Germany;Spain;Taiwan;Turkey;United Kingdom;United States;
AXO-AAV-GM2 High Dose
Sio Gene Therapies
2021 Phase 1 NCT04669535 United States;
AXO-AAV-GM2 Low Dose
Sio Gene Therapies
2021 Phase 1 NCT04669535 United States;
AXO-AAV-GM2 Middle Dose
Sio Gene Therapies
2021 Phase 1 NCT04669535 United States;
AXO-AAV-GM2 Starting Dose
Sio Gene Therapies
2021 Phase 1 NCT04669535 United States;
Abatacept
Emory University
2014 Phase 1 NCT01917708 United States;
Abcertin
ISU Abxis Co., Ltd.
2020 Phase 1 NCT04787887 Australia;
Accupro
Salford Royal NHS Foundation Trust
2009 Phase 4 EUCTR2007-007482-21-GB United Kingdom;
Accuretic
Salford Royal NHS Foundation Trust
2009 Phase 4 EUCTR2007-007482-21-GB United Kingdom;
Adalimumab
Los Angeles Biomedical Research Institute
2015 Phase 1/Phase 2 NCT02437253 United States;
Adalimumab Injection [Humira]
Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
2017 Phase 1/Phase 2 NCT03153319 United States;
Adeno-associated viral vector serotype 9 containing the human N-acetyl-alpha-glucosaminidase gene
Abeona Therapeutics Europe SL.
2021 Phase 1;Phase 2 EUCTR2019-002936-97-FR France;Germany;United States;
- Phase 1;Phase 2 EUCTR2019-002936-97-DE France;Germany;Spain;United States;
Adeno-associated virus (AAV) serotype rh.10 expressing human betagalactosidase (AAVrh.10-CAG-betagal)
Lysogene
- Phase 1;Phase 2 EUCTR2019-004949-32-FR France;United Kingdom;United States;
Adeno-associated virus (AAV) serotype rh.10 expressing human betagalactosidase (AAVrh.10-CAG-ßgal)
Lysogene
- Phase 1;Phase 2 EUCTR2019-004949-32-FR France;United Kingdom;United States;
Adeno-associated virus serotype 2/6 encoding Human Iduronate 2-sulfatase (hIDS) gene donor vector, SB-A6P-HNT Donor Vector
Sangamo Therapeutics, Inc.
2018 Phase 1;Phase 2 EUCTR2018-000192-33-GB United Kingdom;United States;
Adeno-associated virus serotype 2/6 encoding Left side-zinc finger nuclease (ZFN1), SB-A6P-ZLEFT Vector
Sangamo Therapeutics, Inc.
2018 Phase 1;Phase 2 EUCTR2018-000206-28-GB United Kingdom;United States;
2018 Phase 1;Phase 2 EUCTR2018-000192-33-GB United Kingdom;United States;
Adeno-associated virus serotype 2/6 encoding Right side-zinc finger nuclease (ZFN2), SB-A6P-ZRIGHT Vector
Sangamo Therapeutics, Inc.
2018 Phase 1;Phase 2 EUCTR2018-000206-28-GB United Kingdom;United States;
2018 Phase 1;Phase 2 EUCTR2018-000192-33-GB United Kingdom;United States;
Adeno-associated virus serotype 2/6 encoding human alpha-galactosidase A cDNA
Sangamo Therapeutics, Inc.
2019 Phase 1;Phase 2 EUCTR2019-000667-24-GB United Kingdom;United States;
Adeno-associated virus serotype 2/6 encoding human iduronidase (hIDUA) gene donor vector, SB-A6P-HRL Donor Vector
Sangamo Therapeutics, Inc.
2018 Phase 1;Phase 2 EUCTR2018-000206-28-GB United Kingdom;United States;
Adeno-associated virus serotype hu68 containing the human GALC gene
Passage Bio, Inc.
- Phase 1;Phase 2 EUCTR2020-005229-95-NL Brazil;Canada;Israel;Netherlands;United Kingdom;United States;
Adeno-associated virus serotype rh.10 expressing beta-galactosidase
Lysogene
- Phase 1;Phase 2 EUCTR2019-004949-32-FR France;United Kingdom;United States;
Administration Kit
BioMarin Pharmaceutical
2020 - NCT04476862 United States;
Adrabetadex
Mandos LLC
2018 Phase 3 NCT03643562 United States;
2015 Phase 2/Phase 3 NCT04958642 Australia;France;Germany;New Zealand;Singapore;Switzerland;Turkey;United Kingdom;United States;
Afegostat tartrate
Amicus Therapeutics
2009 Phase 2 NCT00813865 Paraguay;United Kingdom;United States;
2008 Phase 2 NCT00446550 Germany;Israel;South Africa;United Kingdom;United States;
2007 Phase 2 NCT00433147 United States;
Amicus Therapeutics, Inc.
2009 - EUCTR2008-007158-36-GB United Kingdom;
Agalsidase
Amicus Therapeutics
2011 Phase 3 NCT01218659 Argentina;Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;Poland;Switzerland;Taiwan;Turkey;United Kingdom;United States;
Agalsidase alfa
AMICUS THERAPEUTICS, INC
2011 - EUCTR2010-022636-37-IT Argentina;Australia;Austria;Belgium;Brazil;Denmark;Germany;Greece;Italy;Japan;Mexico;Russian Federation;Switzerland;Taiwan;Turkey;United Kingdom;United States;
Sanofi
2020 Phase 4 NCT04143958 Czechia;
Shire
2011 Phase 3 NCT01298141 Canada;
2011 Phase 2 NCT01304277 Canada;
2010 - NCT01031173 United States;
2004 Phase 2 NCT00084084 Canada;United States;
TKT Inc
2004 - EUCTR2004-000772-14-CZ Czech Republic;
sanofi-aventis recherche et développement
- Phase 3 EUCTR2021-002320-20-ES Austria;Canada;China;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Turkey;United Kingdom;United States;
Agalsidase alpha
Shire Human Genetic Therapies, Inc. (Shire HGT)
2011 - EUCTR2007-005543-22-SI Australia;Czech Republic;Finland;Paraguay;Slovenia;United Kingdom;United States;
2010 Phase 3;Phase 4 EUCTR2007-005543-22-GB Australia;Czech Republic;Finland;Paraguay;Slovenia;United Kingdom;United States;
2010 Phase 3;Phase 4 EUCTR2007-005543-22-FI Australia;Czech Republic;Finland;Paraguay;Slovenia;United Kingdom;United States;
2008 - EUCTR2007-005543-22-CZ Australia;Czech Republic;Finland;Paraguay;Slovenia;United Kingdom;United States;
Agalsidase beta
AMICUS THERAPEUTICS, INC
2011 - EUCTR2010-022636-37-IT Argentina;Australia;Austria;Belgium;Brazil;Denmark;Germany;Greece;Italy;Japan;Mexico;Russian Federation;Switzerland;Taiwan;Turkey;United Kingdom;United States;
Genzyme Europe BV
2010 - EUCTR2006-001910-33-AT Austria;United Kingdom;
2009 Phase 3 EUCTR2007-005668-28-FR Czech Republic;France;Germany;Netherlands;Portugal;United Kingdom;
2008 - EUCTR2007-005668-28-PT Czech Republic;France;Germany;Netherlands;Portugal;United Kingdom;
2008 - EUCTR2007-005668-28-NL Czech Republic;France;Germany;Netherlands;Portugal;United Kingdom;
2008 - EUCTR2007-005668-28-GB Argentina;Brazil;Canada;Czech Republic;France;Germany;Netherlands;Norway;Poland;Portugal;United Kingdom;United States;
2008 - EUCTR2007-005668-28-DE Czech Republic;France;Germany;Netherlands;Portugal;United Kingdom;
2008 - EUCTR2007-005668-28-CZ Czech Republic;France;Germany;Netherlands;Portugal;United Kingdom;
2007 - EUCTR2006-001910-33-GB Austria;United Kingdom;
Genzyme, a Sanofi Company
2021 Phase 4 NCT05054387 China;
2012 Phase 4 NCT01650779 United States;
2008 Phase 3 NCT00701415 Argentina;Brazil;Canada;Chile;Czech Republic;Netherlands;Norway;Poland;Portugal;United Kingdom;United States;
2006 Phase 4 NCT00230607 Austria;United Kingdom;United States;
2005 Phase 4 NCT00140621 Japan;
2004 Phase 4 NCT00081497 Canada;Czech Republic;Hungary;Poland;United Kingdom;United States;
Protalix
2016 Phase 3 NCT02795676 Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Czechia;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Switzerland;Turkey;United Kingdom;United States;
sanofi-aventis recherche et développement
- Phase 3 EUCTR2021-002320-20-ES Austria;Canada;China;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Turkey;United Kingdom;United States;
Agalsidase beta (GZ419828)
Sanofi
2020 Phase 4 NCT04143958 Czechia;
Agalsidase beta (recombinant form)
Genzyme, a Sanofi Company
2004 - NCT00233870 Japan;
Aldurazyme
Genzyme, a Sanofi Company
2001 Phase 3 NCT00146770 Brazil;Canada;Germany;Italy;Netherlands;United Kingdom;United States;
HUS, Hospital for Children and Adolescents
2007 - EUCTR2006-005216-27-FI Finland;
Aldurazyme (Recombinant Human Alpha-L-Iduronidase)
Genzyme, a Sanofi Company
2005 Phase 3 NCT00258011 Japan;
2004 Phase 4 NCT00144781 Brazil;Canada;
2002 Phase 2 NCT00146757 France;Germany;Netherlands;United Kingdom;
Aldurazyme (laronidase)
Genzyme, a Sanofi Company
2010 Phase 4 NCT00418821 Italy;United States;
University of California, San Francisco
2021 Phase 1 NCT04532047 United States;
Alemtuzumab
Masonic Cancer Center, University of Minnesota
2006 Phase 2 NCT00383448 United States;
Alendronate sodium
Children's Hospital Medical Center, Cincinnati
1998 Phase 2 NCT00004488 Israel;United States;
Alfa-manosidadas humana recombinante
Zymenex A/S
2012 - EUCTR2011-004355-40-ES Belgium;Denmark;Germany;Spain;United Kingdom;
Allogeneic stem cell transplantation
Masonic Cancer Center, University of Minnesota
2009 Phase 2 NCT01043640 United States;
Ambroxol
Exsar Corporation
2012 Phase 1/Phase 2 NCT01463215 United States;
Ioroi Minoru
2021 Phase 1 JPRN-jRCT2051210024 Japan;
Maegaki Yoshihiro
2010 Phase 2 JPRN-jRCTs061180090 Japan;
Narita Aya
2019 Phase 3 JPRN-jRCTs061190017 Japan;
Shaare Zedek Medical Center
2019 Phase 2 NCT03950050 Israel;
Tottori University, Faculty of Medicine, Institute of Neurological Science
2012 Phase 2,3 JPRN-UMIN000009392 Japan;
Anakinra
Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
2019 Phase 2/Phase 3 NCT04018755 United States;
Arimoclomol
Orphazyme
2022 - NCT04316637 United States;
2018 Phase 2 NCT03746587 India;
2016 Phase 2/Phase 3 NCT02612129 Denmark;France;Germany;Italy;Poland;Spain;Switzerland;United Kingdom;United States;
Orphazyme ApS
2016 Phase 2;Phase 3 EUCTR2015-004438-93-IT Denmark;France;Germany;Italy;Poland;Spain;Switzerland;United Kingdom;United States;
2016 Phase 2;Phase 3 EUCTR2015-004438-93-ES Denmark;Spain;Switzerland;United States;
Arimoclomol (HPMC capsule)
Orphazyme A/S
2016 Phase 2;Phase 3 EUCTR2015-004438-93-DE Denmark;France;Germany;Italy;Poland;Spain;Switzerland;United Kingdom;United States;
Arimoclomol (hard gelatine capsule)
Orphazyme A/S
2016 Phase 2;Phase 3 EUCTR2015-004438-93-DE Denmark;France;Germany;Italy;Poland;Spain;Switzerland;United Kingdom;United States;
Arimoclomol citrate
Orphazyme A/S
2016 Phase 2;Phase 3 EUCTR2015-004438-93-DK Denmark;Germany;Italy;Poland;Spain;Switzerland;United Kingdom;United States;
2016 Phase 2;Phase 3 EUCTR2015-004438-93-DE Denmark;France;Germany;Italy;Poland;Spain;Switzerland;United Kingdom;United States;
Arylsulfatase A
Shire Pharmaceuticals Ireland Limited
2006 - EUCTR2006-005341-11-DK Denmark;
Autologous CD34+ cells transduced with a lentiviral vector containing the human SGSH gene
University of Manchester
2020 Phase 1/Phase 2 NCT04201405 United Kingdom;
Autologous CD34+ cells transduced with a lentiviral vector encoding the ARSA cDNA
FONDAZIONE CENTRO S. RAFFAELE DEL MONTE TABOR
2010 - EUCTR2009-017349-77-IT Italy;
Autologous Plasmablasts
Masonic Cancer Center, University of Minnesota
2021 Phase 1/Phase 2 NCT04284254 -
Azathioprine (Aza)
Genzyme, a Sanofi Company
2008 Phase 1/Phase 2 NCT00741338 Brazil;Russian Federation;Ukraine;
B (migalastat)
Amicus Therapeutics
2011 Phase 1 NCT01489995 United States;
BHP001
Torayuki Okuyama
2016 Phase 1-2 JPRN-JMA-IIA00350 Japan;
BM110
BIOMARIN PHARMACEUTICAL INC.
2011 - EUCTR2010-020199-45-IT Argentina;Australia;Brazil;Canada;Colombia;Denmark;Germany;Italy;Japan;Korea, Democratic People's Republic of;Portugal;Qatar;Saudi Arabia;Spain;Switzerland;Taiwan;Turkey;United Kingdom;United States;
BMN 110
BioMarin Pharmaceutical
2013 Phase 3 NCT01966029 Australia;
2012 Phase 2 NCT01697319 Germany;United Kingdom;United States;
2012 Phase 2 NCT01609062 Canada;Germany;United Kingdom;United States;
2011 Phase 2 NCT01515956 Italy;Taiwan;United Kingdom;United States;
2010 Phase 1/Phase 2 NCT01242111 United Kingdom;
2009 Phase 1/Phase 2 NCT00884949 United Kingdom;
2009 - NCT01858103 Puerto Rico;United States;
BMN 110 - Every Other Week
BioMarin Pharmaceutical
2011 Phase 3 NCT01415427 Argentina;Brazil;Canada;Colombia;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Netherlands;Norway;Portugal;Qatar;Saudi Arabia;Spain;Taiwan;Turkey;United Kingdom;United States;
BMN 110 - Weekly
BioMarin Pharmaceutical
2011 Phase 3 NCT01415427 Argentina;Brazil;Canada;Colombia;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Netherlands;Norway;Portugal;Qatar;Saudi Arabia;Spain;Taiwan;Turkey;United Kingdom;United States;
BMN 110 Every Other Week
BioMarin Pharmaceutical
2011 Phase 3 NCT01275066 Argentina;Australia;Brazil;Canada;Colombia;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Portugal;Qatar;Saudi Arabia;Switzerland;Taiwan;Turkey;United Kingdom;United States;
BMN 110 Weekly
BioMarin Pharmaceutical
2011 Phase 3 NCT01275066 Argentina;Australia;Brazil;Canada;Colombia;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Portugal;Qatar;Saudi Arabia;Switzerland;Taiwan;Turkey;United Kingdom;United States;
BMN 190
BioMarin Pharmaceutical
2015 Phase 1/Phase 2 NCT02485899 Germany;Italy;United Kingdom;United States;
2013 Phase 1/Phase 2 NCT01907087 Germany;Italy;United Kingdom;United States;
BioMarin Pharmaceutical Inc
2017 Phase 2 EUCTR2015-000891-85-GB Germany;Italy;United Kingdom;United States;
BioMarin Pharmaceutical Inc.
2016 Phase 2 EUCTR2015-000891-85-DE Germany;Italy;United Kingdom;
2015 Phase 2 EUCTR2014-003480-37-GB Germany;Italy;United Kingdom;United States;
2015 Phase 2 EUCTR2014-003480-37-DE Germany;Italy;United Kingdom;United States;
2015 - EUCTR2014-003480-37-IT Germany;Italy;Japan;United Kingdom;United States;
2014 Phase 1;Phase 2 EUCTR2012-005430-11-IT Germany;Italy;United Kingdom;
2013 Phase 1;Phase 2 EUCTR2012-005430-11-GB Germany;Italy;United Kingdom;United States;
2013 Phase 1;Phase 2 EUCTR2012-005430-11-DE Germany;United Kingdom;
BMN 250
BioMarin Pharmaceutical Inc.
2017 Phase 2 EUCTR2017-003083-13-ES Argentina;Australia;Colombia;Germany;Spain;Taiwan;Turkey;United Kingdom;United States;
2016 Phase 1;Phase 2 EUCTR2015-001985-25-ES Australia;Brazil;Colombia;Germany;Spain;Taiwan;Turkey;United Kingdom;
BMN190 recombinant human tripeptidyl peptidase-1 (rhTPP1)
BioMarin Pharmaceutical
2016 Phase 2 NCT02678689 Germany;Italy;United Kingdom;United States;
BUSILVEX - 6 MG/ML - CONCENTRATO PER SOLUZIONE PER INFUSIONE - USO ENDOVENOSO - FLACONCINO - 10 ML 8 FLACONCINI
OSPEDALE SAN RAFFAELE
2018 Phase 1 EUCTR2017-002430-23-IT Italy;
Beta-mercaptoethylamine Hydrochloride & 2-aminoethanethiol Hydrochloride
Recordati Rare Diseases
2020 Phase 3 EUCTR2018-002984-24-IT Belgium;France;Germany;Italy;United Kingdom;
2020 Phase 3 EUCTR2018-002984-24-DE France;Germany;United Kingdom;
- Phase 3 EUCTR2018-002984-24-BE Belgium;France;Germany;Italy;United Kingdom;
Biological assays
University Hospital, Bordeaux
2016 - NCT03123523 France;
Biopsy
University Hospital, Grenoble
2013 - NCT02841358 France;
Blood draw
Baylor Research Institute
2018 - NCT04002531 United States;
Blood sampling
University Hospital, Grenoble
2013 - NCT02841358 France;
Brain MRI/MRS/fMRI
National Human Genome Research Institute (NHGRI)
2019 Phase 1/Phase 2 NCT03952637 United States;
Brineura
BioMarin Pharmaceutical Inc
2017 Phase 2 EUCTR2015-000891-85-GB Germany;Italy;United Kingdom;United States;
BioMarin Pharmaceutical Inc.
2016 Phase 2 EUCTR2015-000891-85-DE Germany;Italy;United Kingdom;
2015 Phase 2 EUCTR2014-003480-37-GB Germany;Italy;United Kingdom;United States;
2015 Phase 2 EUCTR2014-003480-37-DE Germany;Italy;United Kingdom;United States;
Busulfan
FONDAZIONE CENTRO S. RAFFAELE DEL MONTE TABOR
2010 - EUCTR2009-017349-77-IT Italy;
Masonic Cancer Center, University of Minnesota
2009 Phase 2 NCT01043640 United States;
2008 Phase 2 NCT00668564 United States;
Busulfan, Cyclophosphamide, ATG
Masonic Cancer Center, University of Minnesota
1999 Phase 2 NCT00176917 United States;
Busulfan, Cyclophosphamide, Antithymocyte Globulin
Masonic Cancer Center, University of Minnesota
1995 Phase 2/Phase 3 NCT00176904 United States;
C (migalastat)
Amicus Therapeutics
2011 Phase 1 NCT01489995 United States;
CALD HR-D (High-Risk, Regimen C)
Masonic Cancer Center, University of Minnesota
2014 Phase 2 NCT02171104 United States;
CALD HR-D (High-Risk, Regimen D)
Masonic Cancer Center, University of Minnesota
2014 Phase 2 NCT02171104 United States;
CALD SR-A (Standard-Risk, Regimen A)
Masonic Cancer Center, University of Minnesota
2014 Phase 2 NCT02171104 United States;
CALD SR-B (Standard-Risk, Regimen B)
Masonic Cancer Center, University of Minnesota
2014 Phase 2 NCT02171104 United States;
CEREDASE™
National Institute of Neurological Disorders and Stroke (NINDS)
1993 Phase 2 NCT00001416 United States;
CRYOPRESERVED AUTOLOGOUS CD34+ CELLS TRANSDUCED WITH A LENTIVIRAL VECTOR CONTAINING HUMAN SGSH GENE
University of Manchester
2019 Phase 1;Phase 2 EUCTR2019-002051-42-GB United Kingdom;
CYSTADROPS eye drops, solution
Orphan Europe SARL
2013 - EUCTR2009-012564-13-FR France;
CYSTEAMINE HYDROCHLORIDE
Orphan Europe SARL
2013 - EUCTR2009-012564-13-FR France;
Calcium carbonate
Children's Hospital Medical Center, Cincinnati
1998 Phase 2 NCT00004488 Israel;United States;
Campath-1H
Masonic Cancer Center, University of Minnesota
2012 - NCT01626092 United States;
2009 Phase 2 NCT01043640 United States;
2008 Phase 2 NCT00668564 United States;
Cannabis sativa L., folium cum flore
Albina Nowak, MD
2021 - NCT04820361 -
Capsaicin
Universitaire Ziekenhuizen Leuven
2014 Phase 0 NCT02533076 Belgium;
Cellule CD34+ autologhe trasdotte con IDUA LV codificante per il cDNA dell'alfa-L-iduronidasi
OSPEDALE SAN RAFFAELE
2018 Phase 1 EUCTR2017-002430-23-IT Italy;
Cerdelga
Genzyme Corporation
2019 Phase 3 EUCTR2016-000301-37-NL Argentina;Canada;Egypt;France;Italy;Netherlands;Russian Federation;Spain;Sweden;Turkey;United Kingdom;
2019 Phase 3 EUCTR2016-000301-37-GB Argentina;Canada;Egypt;France;Italy;Netherlands;Russian Federation;Spain;Sweden;Turkey;United Kingdom;
2018 Phase 3 EUCTR2016-000301-37-SE Argentina;Canada;Egypt;France;Italy;Japan;Netherlands;Russian Federation;Spain;Sweden;Taiwan;Turkey;United Kingdom;
2018 Phase 3 EUCTR2016-000301-37-IT Argentina;Canada;Egypt;France;Italy;Netherlands;Russian Federation;Spain;Sweden;Turkey;United Kingdom;
2018 Phase 3 EUCTR2016-000301-37-ES Argentina;Canada;Egypt;France;Italy;Netherlands;Russian Federation;Spain;Sweden;Turkey;United Kingdom;
Cerezyme
Genzyme Corporation
2019 Phase 3 EUCTR2016-000301-37-NL Argentina;Canada;Egypt;France;Italy;Netherlands;Russian Federation;Spain;Sweden;Turkey;United Kingdom;
2019 Phase 3 EUCTR2016-000301-37-GB Argentina;Canada;Egypt;France;Italy;Netherlands;Russian Federation;Spain;Sweden;Turkey;United Kingdom;
2018 Phase 3 EUCTR2016-000301-37-SE Argentina;Canada;Egypt;France;Italy;Japan;Netherlands;Russian Federation;Spain;Sweden;Taiwan;Turkey;United Kingdom;
2018 Phase 3 EUCTR2016-000301-37-IT Argentina;Canada;Egypt;France;Italy;Netherlands;Russian Federation;Spain;Sweden;Turkey;United Kingdom;
2018 Phase 3 EUCTR2016-000301-37-ES Argentina;Canada;Egypt;France;Italy;Netherlands;Russian Federation;Spain;Sweden;Turkey;United Kingdom;
Genzyme Europe B.V.
2010 - EUCTR2008-005223-28-DE Argentina;Australia;Brazil;Canada;Czech Republic;Egypt;France;Germany;Italy;Netherlands;Russian Federation;Spain;Turkey;United Kingdom;United States;
2009 Phase 3 EUCTR2008-005223-28-GB Argentina;Australia;Brazil;Canada;Czech Republic;Egypt;France;Germany;Italy;Netherlands;Russian Federation;Spain;Turkey;United Kingdom;United States;
2009 Phase 3 EUCTR2008-005223-28-FR Czech Republic;France;Germany;Italy;Netherlands;Spain;United Kingdom;
2009 - EUCTR2008-005223-28-NL Czech Republic;France;Germany;Italy;Netherlands;Spain;United Kingdom;
2009 - EUCTR2008-005223-28-CZ Czech Republic;France;Germany;Italy;Netherlands;Spain;United Kingdom;
Genzyme, a Sanofi Company
2001 Phase 4 NCT00364858 Brazil;Canada;Italy;Poland;Spain;United Kingdom;United States;
Shire Human Genetic Therapies Inc
2007 - EUCTR2007-002840-21-ES Italy;Spain;United Kingdom;
Shire Human Genetic Therapies, Inc.
2007 - EUCTR2007-002840-21-GB Italy;Spain;United Kingdom;
Cerezyme (imiglucerase for injection)
Genzyme, a Sanofi Company
1997 Phase 4 NCT00365131 United States;
Cerezyme 400 Units Powder for concentrate for solution for infusion
Sanofi-aventis recherche & développement
- Phase 3 EUCTR2021-005402-10-FR Argentina;Brazil;Canada;China;Egypt;France;Germany;Hungary;Japan;Taiwan;Turkey;United Kingdom;United States;
- Phase 3 EUCTR2021-005402-10-DE Argentina;Brazil;Canada;China;Egypt;France;Germany;Hungary;Japan;Taiwan;Turkey;United Kingdom;United States;
Cerezyme®
ISU Abxis Co., Ltd.
2011 Phase 3 NCT01161914 -
Cerezyme® / Imiglucerase
Sanofi
2021 Phase 4 NCT04656600 China;
Cerliponase alfa
BioMarin Pharmaceutical Inc
2017 Phase 2 EUCTR2015-000891-85-GB Germany;Italy;United Kingdom;United States;
BioMarin Pharmaceutical Inc.
2016 Phase 2 EUCTR2015-000891-85-DE Germany;Italy;United Kingdom;
2015 Phase 2 EUCTR2014-003480-37-GB Germany;Italy;United Kingdom;United States;
2015 Phase 2 EUCTR2014-003480-37-DE Germany;Italy;United Kingdom;United States;
2013 Phase 1;Phase 2 EUCTR2012-005430-11-GB Germany;Italy;United Kingdom;United States;
Cholecalciferol
Children's Hospital Medical Center, Cincinnati
1998 Phase 2 NCT00004488 Israel;United States;
Clofarabine
Masonic Cancer Center, University of Minnesota
2012 - NCT01626092 United States;
2006 Phase 2 NCT00383448 United States;
Contrast sensitivity measurement
Université de Montréal
2014 - NCT02023086 Canada;
Coversyl PLUS
Salford Royal NHS Foundation Trust
2009 Phase 4 EUCTR2007-007482-21-GB United Kingdom;
Cyclophosphamide
Masonic Cancer Center, University of Minnesota
2009 Phase 2 NCT01043640 United States;
2008 Phase 2 NCT00668564 United States;
Cyclosporine A
Masonic Cancer Center, University of Minnesota
2012 - NCT01626092 United States;
2009 Phase 2 NCT01043640 United States;
2006 Phase 2 NCT00383448 United States;
Cyclosporine A (CsA)
Genzyme, a Sanofi Company
2008 Phase 1/Phase 2 NCT00741338 Brazil;Russian Federation;Ukraine;
Cystadane anhydrous
Minna Laine
2017 Phase 2 EUCTR2017-000645-48-FI Finland;
Cystadrops
RECORDATI Rare Diseases SARL
2019 Phase 3 EUCTR2018-002984-24-GB France;United Kingdom;
Recordati Rare Diseases
2020 Phase 3 EUCTR2018-002984-24-IT Belgium;France;Germany;Italy;United Kingdom;
2020 Phase 3 EUCTR2018-002984-24-DE France;Germany;United Kingdom;
- Phase 3 EUCTR2018-002984-24-FR France;United Kingdom;
- Phase 3 EUCTR2018-002984-24-BE Belgium;France;Germany;Italy;United Kingdom;
Cystagon
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
2001 Phase 4 NCT00028262 United States;
Horizon Pharma USA, Inc.
2014 Phase 3 EUCTR2012-002773-64-GB Belgium;France;Germany;Italy;Netherlands;United Kingdom;United States;
2013 Phase 3 EUCTR2012-002773-64-NL Belgium;France;Germany;Italy;Netherlands;United Kingdom;United States;
Raptor Therapeutics Inc.
2014 Phase 3 EUCTR2012-002773-64-BE Belgium;France;Germany;Italy;Netherlands;United Kingdom;United States;
2013 Phase 3 EUCTR2012-002773-64-FR Belgium;France;Italy;Netherlands;United Kingdom;United States;
2010 Phase 3 EUCTR2009-017882-42-FR France;Netherlands;
2010 - EUCTR2009-017882-42-NL France;Netherlands;
Cystagon®
Horizon Pharma USA, Inc.
2013 Phase 3 NCT01733316 Belgium;France;Italy;Netherlands;United Kingdom;United States;
2009 Phase 1/Phase 2 NCT00872729 United States;
Cystagon® (Cysteamine Bitartrate)
Horizon Pharma USA, Inc.
2010 Phase 3 NCT01000961 France;Netherlands;United States;
Cysteamine
Hospices Civils de Lyon
2011 - NCT02012114 France;
National Eye Institute (NEI)
1998 Phase 1 NCT00001736 United States;
1986 Phase 2 NCT00001213 United States;
Cysteamine (mercaptamine) viscous solution
Fundació Hospital Universitari Vall d'Hebron - Institut de Recerca (VHIR)
2014 - EUCTR2013-003228-35-ES Spain;
Cysteamine Bitartrate
Chiesi SA/NV
2020 - NCT04246060 Belgium;
Cysteamine Bitartrate Delayed-release Capsules
Horizon Pharma USA, Inc.
2010 Phase 3 NCT01197378 France;Netherlands;United States;
Cysteamine Bitartrate Delayed-release Capsules (RP103)
Horizon Pharma USA, Inc.
2010 Phase 3 NCT01000961 France;Netherlands;United States;
Cysteamine bitartrate
University of California, San Diego
2011 - NCT01432561 United States;
Cysteamine bitartrate (INN: mercaptamine bitartrate
Raptor Therapeutics Inc.
2010 - EUCTR2009-017882-42-NL France;Netherlands;
Cysteamine bitartrate (INN: mercaptamine bitartrate )
Horizon Pharma USA, Inc.
2014 Phase 3 EUCTR2012-002773-64-GB Belgium;France;Germany;Italy;Netherlands;United Kingdom;United States;
2010 Phase 3 EUCTR2010-018365-34-NL France;Netherlands;United States;
Raptor Therapeutics Inc.
2014 Phase 3 EUCTR2012-002773-64-IT Belgium;France;Germany;Italy;Netherlands;United Kingdom;United States;
2014 Phase 3 EUCTR2012-002773-64-BE Belgium;France;Germany;Italy;Netherlands;United Kingdom;United States;
2013 Phase 3 EUCTR2012-002773-64-FR Belgium;France;Italy;Netherlands;United Kingdom;United States;
2010 Phase 3 EUCTR2009-017882-42-FR France;Netherlands;
- Phase 3 EUCTR2010-018365-34-FR France;Netherlands;
Cysteamine bitartrate (INN: mercaptamine bitartrate)
Horizon Pharma USA, Inc.
2013 Phase 3 EUCTR2012-002773-64-NL Belgium;France;Germany;Italy;Netherlands;United Kingdom;United States;
Cysteamine hydrochloride
FDA Office of Orphan Products Development
1999 - NCT00010426 -
Orphan Europe SARL
2008 - EUCTR2007-006024-35-FR France;
RECORDATI Rare Diseases SARL
2019 Phase 3 EUCTR2018-002984-24-GB France;United Kingdom;
Recordati Rare Diseases
2020 Phase 3 EUCTR2018-002984-24-DE France;Germany;United Kingdom;
- Phase 3 EUCTR2018-002984-24-BE Belgium;France;Germany;Italy;United Kingdom;
D (migalastat)
Amicus Therapeutics
2011 Phase 1 NCT01489995 United States;
DEXA scan
University of Texas Southwestern Medical Center
2006 - NCT00286689 United States;
DNL310
Denali Therapeutics Inc.
2020 Phase 1/Phase 2 NCT04251026 Netherlands;United States;
DNL310 Drug Substance
Denali Therapeutics Inc.
2021 Phase 1;Phase 2 EUCTR2019-004909-27-NL Germany;Italy;Netherlands;United Kingdom;United States;
2020 Phase 1;Phase 2 EUCTR2019-004909-27-GB Germany;Italy;Netherlands;United Kingdom;United States;
DRX005B
National Institute of Neurological Disorders and Stroke (NINDS)
2002 Phase 2 NCT00048906 United States;
DUOC-01
Joanne Kurtzberg, MD
2014 Phase 1 NCT02254863 United States;
Dried blood spot (DBS) sampling
Hospices Civils de Lyon
2016 - NCT02843334 France;
E (migalastat)
Amicus Therapeutics
2011 Phase 1 NCT01489995 United States;
ELIGLUSTAT TARTRATE
Genzyme Corporation
2019 Phase 3 EUCTR2016-000301-37-NL Argentina;Canada;Egypt;France;Italy;Netherlands;Russian Federation;Spain;Sweden;Turkey;United Kingdom;
2019 Phase 3 EUCTR2016-000301-37-GB Argentina;Canada;Egypt;France;Italy;Netherlands;Russian Federation;Spain;Sweden;Turkey;United Kingdom;
2018 Phase 3 EUCTR2016-000301-37-IT Argentina;Canada;Egypt;France;Italy;Netherlands;Russian Federation;Spain;Sweden;Turkey;United Kingdom;
2018 Phase 3 EUCTR2016-000301-37-ES Argentina;Canada;Egypt;France;Italy;Netherlands;Russian Federation;Spain;Sweden;Turkey;United Kingdom;
ELX-02
Eloxx Pharmaceuticals, Inc.
2019 Phase 2 NCT04069260 Canada;
ERT
National Taiwan University Hospital
2015 - NCT02520934 Taiwan;
EU-sourced Cerezyme
ISU Abxis Co., Ltd.
2020 Phase 1 NCT04787887 Australia;
Echocardiogram
Baylor Research Institute
2018 - NCT04002531 United States;
Elaprase
Shire
2022 Phase 4 NCT05058391 India;
2015 Phase 2/Phase 3 NCT02412787 Argentina;Australia;Canada;Colombia;France;Mexico;Spain;United Kingdom;United States;
2010 Phase 1/Phase 2 NCT01506141 Canada;United Kingdom;United States;
Shire Human Genetic Therapies, Inc
2016 Phase 4 EUCTR2014-004804-31-DE Costa Rica;Dominican Republic;Germany;Malaysia;Oman;Philippines;Saudi Arabia;Serbia;Thailand;United States;Vietnam;
Elaprase 2mg/ml
Shire Human Genetic Therapies, Inc. (Shire HGT)
- Phase 4 EUCTR2007-006044-22-PL Poland;
Elaprase for intravenous (IV) infusion
Shire
2015 Phase 4 NCT02455622 Dominican Republic;Germany;Malaysia;Oman;Philippines;Saudi Arabia;Serbia;Thailand;United States;Vietnam;
Elaprase_
Green Cross Corporation
2010 Phase 1/Phase 2 NCT01301898 Korea, Republic of;
Elelyso
Ari Zimran
2020 Phase 4 NCT04002830 India;Israel;Turkey;
Shaare Zedek Medical Center
2017 - NCT04353466 -
Elelyso 60 units
Pfizer
2019 Phase 4 NCT03021941 United States;
Eliglustat
Genzyme Europe B.V.
2011 Phase 3 EUCTR2009-015811-42-PT Argentina;Australia;Austria;Brazil;Canada;China;Croatia;France;Greece;India;Japan;Netherlands;Portugal;Romania;Russian Federation;Serbia;Sweden;United States;
2011 Phase 3 EUCTR2009-015811-42-AT Argentina;Australia;Austria;Brazil;Canada;China;Croatia;France;Greece;India;Japan;Netherlands;Portugal;Romania;Russian Federation;Serbia;Sweden;United States;
2011 - EUCTR2009-015811-42-GR Austria;Greece;Portugal;Sweden;
2010 Phase 3 EUCTR2009-015811-42-SE Argentina;Australia;Austria;Brazil;Canada;China;Croatia;France;Greece;India;Japan;Netherlands;Portugal;Romania;Russian Federation;Serbia;Sweden;United States;
2010 - EUCTR2008-005223-28-DE Argentina;Australia;Brazil;Canada;Czech Republic;Egypt;France;Germany;Italy;Netherlands;Russian Federation;Spain;Turkey;United Kingdom;United States;
2010 - EUCTR2008-005222-37-BG Bulgaria;Canada;Colombia;India;Israel;Jordan;Lebanon;Mexico;Netherlands;Russian Federation;Saudi Arabia;Serbia;Tunisia;United Kingdom;United States;
2009 Phase 3 EUCTR2008-005223-28-GB Argentina;Australia;Brazil;Canada;Czech Republic;Egypt;France;Germany;Italy;Netherlands;Russian Federation;Spain;Turkey;United Kingdom;United States;
2009 Phase 3 EUCTR2008-005222-37-GB Bulgaria;Canada;Colombia;India;Israel;Jordan;Lebanon;Mexico;Netherlands;Russian Federation;Saudi Arabia;Serbia;Tunisia;United Kingdom;United States;
2009 - EUCTR2008-005223-28-NL Czech Republic;France;Germany;Italy;Netherlands;Spain;United Kingdom;
2009 - EUCTR2008-005222-37-NL Bulgaria;Netherlands;United Kingdom;
Genzyme, a Sanofi Company
2015 Phase 1 NCT02536911 United States;
2015 Phase 1 NCT02422654 United States;
National Taiwan University Hospital
2018 - NCT03519646 Taiwan;
Sanofi
2015 Phase 1 NCT02536937 United States;
Eliglustat GZ385660
Genzyme, a Sanofi Company
2015 Phase 3 NCT02536755 Canada;Russian Federation;Tunisia;
Sanofi
2018 Phase 3 NCT03485677 Argentina;Canada;France;Italy;Japan;Russian Federation;Spain;Sweden;Turkey;United Kingdom;
Eliglustat Tartrate – Powder for Oral Suspension
Genzyme Corporation
2018 Phase 3 EUCTR2016-000301-37-SE Argentina;Canada;Egypt;France;Italy;Japan;Netherlands;Russian Federation;Spain;Sweden;Taiwan;Turkey;United Kingdom;
Eliglustat tartrate
Genzyme Europe B.V.
2009 - EUCTR2008-005223-28-CZ Czech Republic;France;Germany;Italy;Netherlands;Spain;United Kingdom;
Genzyme, a Sanofi Company
2010 Phase 3 NCT01074944 Australia;Austria;Brazil;Canada;China;Croatia;France;Greece;India;Japan;Netherlands;Portugal;Romania;Russian Federation;Serbia;Sweden;United States;
2009 Phase 3 NCT00943111 Argentina;Australia;Brazil;Canada;Czech Republic;Egypt;France;Germany;Italy;Netherlands;Russian Federation;Spain;Turkey;United Kingdom;United States;
2009 Phase 3 NCT00891202 Bulgaria;Canada;Chile;Colombia;Georgia;India;Israel;Jordan;Lebanon;Mexico;Netherlands;Russian Federation;Saudi Arabia;Serbia;Tunisia;United Kingdom;United States;
2006 Phase 2 NCT00358150 Argentina;Israel;Italy;Mexico;Russian Federation;United States;
Elosulfase Alfa 1 MG/ML Intravenous Solution [VIMIZIM]
GOIZET
2018 - NCT03204370 France;
Enalapril and other angiotensin converting enzyme inhibitors
University of Alabama at Birmingham
2007 - NCT00446862 Germany;Slovenia;United States;
Enriched Hematopoetic Stem Cell Infusion
Talaris Therapeutics Inc.
2014 - NCT02084121 United States;
2013 - NCT02021266 United States;
Enzyme replacement
Rigshospitalet, Denmark
2001 Phase 4 NCT01997489 Denmark;
Enzyme replacement therapy
Ulla Feldt-Rasmussen
2015 - NCT02969200 Denmark;
Enzyme replacement therapy (Agalsidase alpha (Replagal®))
University Hospital, Rouen
2017 - NCT02956954 France;
Enzyme replacement therapy (ERT)
Idorsia Pharmaceuticals Ltd.
2015 Phase 1 NCT02930655 Germany;
FABRAZYME - 35 MG POLVERE PER CONCENTRATO PER SOLUZIONE PER INFUSIONE ENDOVENOSA 1 FLACONCINO
FONDAZIONE POLICLINICO UNIVERSITARIO AGOSTINO GEMELLI IRCCS UNIVERSITA' CATTOLICA DEL SACRO CUORE
2018 Phase 4 EUCTR2018-000368-27-IT Italy;
PROTALIX LTD
2017 Phase 3 EUCTR2016-000378-38-IT Australia;Belgium;Canada;Czech Republic;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Turkey;United Kingdom;United States;
FBX-101
Forge Biologics, Inc
2021 Phase 1/Phase 2 NCT04693598 United States;
FLT190
Freeline Therapeutics Limited
2019 Phase 1;Phase 2 EUCTR2018-002097-51-IT Denmark;France;Germany;Italy;Norway;United Kingdom;United States;
Freeline Therapeutics Ltd
2021 Phase 1;Phase 2 EUCTR2018-002097-51-AT Austria;Denmark;France;Germany;Italy;Norway;Switzerland;United Kingdom;United States;
2020 Phase 2 EUCTR2019-004645-32-GB Denmark;France;Germany;Italy;Norway;United Kingdom;United States;
2020 Phase 1;Phase 2 EUCTR2018-002097-51-DK Denmark;France;Germany;Italy;Norway;Spain;United Kingdom;United States;
2020 Phase 1;Phase 2 EUCTR2018-002097-51-DE Austria;Denmark;France;Germany;Italy;Norway;Switzerland;United Kingdom;United States;
FLUDARABINA ACCORD - 25 MG/ML CONCENTRATO PER SOLUZIONE INIETTABILE O PER INFUSIONE 5 FLACONCINI IN VETRO DA 2 ML
OSPEDALE SAN RAFFAELE
2018 Phase 1 EUCTR2017-002430-23-IT Italy;
FLUDARABINA FOSFATO
OSPEDALE SAN RAFFAELE
2018 Phase 1 EUCTR2017-002430-23-IT Italy;
Fabrazyme
Amicus Therapeutics, Inc.
2012 - EUCTR2010-022709-16-GB Belgium;United Kingdom;
2012 - EUCTR2010-022636-37-DE Argentina;Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Hungary;Israel;Italy;Japan;Mexico;Poland;Russian Federation;Slovakia;Switzerland;Taiwan;Turkey;United Kingdom;United States;
2011 Phase 3 EUCTR2010-022636-37-GR Argentina;Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Hungary;Israel;Italy;Japan;Mexico;Poland;Russian Federation;Slovakia;Switzerland;Taiwan;Turkey;United Kingdom;United States;
2011 Phase 2 EUCTR2010-022709-16-BE Australia;Belgium;Canada;France;Netherlands;Switzerland;United Kingdom;
2011 - EUCTR2010-022636-37-GB Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;United Kingdom;United States;
2011 - EUCTR2010-022636-37-DK Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;United Kingdom;United States;
2011 - EUCTR2010-022636-37-BE Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;United Kingdom;United States;
2011 - EUCTR2010-022636-37-AT Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;United Kingdom;United States;
Genzyme Europe BV
2010 - EUCTR2006-001910-33-AT Austria;United Kingdom;
2009 Phase 3 EUCTR2007-005668-28-FR Czech Republic;France;Germany;Netherlands;Portugal;United Kingdom;
2008 - EUCTR2007-005668-28-PT Czech Republic;France;Germany;Netherlands;Portugal;United Kingdom;
2008 - EUCTR2007-005668-28-NL Czech Republic;France;Germany;Netherlands;Portugal;United Kingdom;
2008 - EUCTR2007-005668-28-GB Argentina;Brazil;Canada;Czech Republic;France;Germany;Netherlands;Norway;Poland;Portugal;United Kingdom;United States;
2008 - EUCTR2007-005668-28-DE Czech Republic;France;Germany;Netherlands;Portugal;United Kingdom;
2008 - EUCTR2007-005668-28-CZ Czech Republic;France;Germany;Netherlands;Portugal;United Kingdom;
2007 - EUCTR2006-001910-33-GB Austria;United Kingdom;
Protalix Ltd.
2019 Phase 3 EUCTR2016-000378-38-FR Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Denmark;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Switzerland;Turkey;United Kingdom;United States;
2019 Phase 3 EUCTR2016-000378-38-FI Argentina;Australia;Belgium;Brazil;Canada;Czechia;Denmark;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Slovenia;Spain;Sweden;Switzerland;Turkey;United Kingdom;United States;
2017 Phase 3 EUCTR2016-000378-38-SI Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Denmark;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Switzerland;Turkey;United Kingdom;United States;
2017 Phase 3 EUCTR2016-000378-38-NL Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Turkey;United Kingdom;United States;
2016 Phase 3 EUCTR2016-000378-38-HU Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Turkey;United Kingdom;United States;
2016 Phase 3 EUCTR2016-000378-38-GB Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Germany;Hungary;Netherlands;Norway;Paraguay;Slovenia;Spain;Turkey;United Kingdom;United States;
2016 Phase 3 EUCTR2016-000378-38-ES Australia;Belgium;Canada;Czech Republic;Germany;Hungary;Netherlands;Norway;Paraguay;Spain;Turkey;United Kingdom;United States;
2016 Phase 3 EUCTR2016-000378-38-BE Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Turkey;United Kingdom;United States;
SANOFI-AVENTIS RECHERCHE E DEVELOPPEMENT
2022 Phase 3 EUCTR2021-002320-20-IT Austria;Canada;China;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Turkey;United Kingdom;United States;
Sanofi Aventis Groupe (SAG)
2019 Phase 4 EUCTR2019-000064-21-GB Austria;Canada;Czech Republic;Denmark;France;Germany;Italy;Norway;Spain;Turkey;United Kingdom;
- Phase 4 EUCTR2019-000065-20-NO Canada;France;Norway;United States;
- Phase 4 EUCTR2019-000064-21-NO Austria;Canada;Czech Republic;Denmark;France;Germany;Italy;Norway;Spain;Turkey;United Kingdom;
- Phase 4 EUCTR2019-000064-21-CZ Austria;Canada;Czech Republic;Denmark;France;Germany;Italy;Norway;Spain;Turkey;United Kingdom;
sanofi-aventis recherche et développement
- Phase 3 EUCTR2021-002320-20-ES Austria;Canada;China;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Turkey;United Kingdom;United States;
Fabrazyme (agalsidase beta)
Genzyme, a Sanofi Company
2006 Phase 4 NCT00312767 United States;
2003 Phase 2 NCT00196716 Czech Republic;Estonia;Poland;Slovakia;
2002 Phase 2 NCT00837824 United States;
2002 Phase 2 NCT00074958 France;Italy;Poland;United Kingdom;United States;
2001 Phase 4 NCT00074984 Canada;Czech Republic;Hungary;Poland;United Kingdom;United States;
1999 Phase 3 NCT00074971 France;Netherlands;Puerto Rico;United Kingdom;United States;
Fluorescein
Ohio State University
2010 - NCT01165697 United States;
GA-GCB
SHIRE HUMAN GENETIC THERAPIES, INC.
2008 - EUCTR2007-002840-21-IT Italy;Spain;United Kingdom;
2008 - EUCTR2006-006304-11-IT Italy;Spain;United Kingdom;
Shire
2005 Phase 1/Phase 2 NCT00391625 Former Serbia and Montenegro;Israel;Romania;Serbia;
Shire Human Genetic Therapies
- - EUCTR2012-003427-38-Outside-EU/EEA Egypt;India;Tunisia;
Shire Human Genetic Therapies Inc
2007 Phase 2;Phase 3 EUCTR2006-006304-11-GB Germany;Italy;Spain;United Kingdom;
2007 - EUCTR2007-002840-21-ES Italy;Spain;United Kingdom;
2007 - EUCTR2006-006304-11-ES Italy;Spain;United Kingdom;
- Phase 2;Phase 3 EUCTR2006-006304-11-DE Germany;Italy;Spain;United Kingdom;
Shire Human Genetic Therapies, Inc.
2008 Phase 3 EUCTR2008-001965-27-GB Spain;United Kingdom;
2008 - EUCTR2008-001965-27-ES Spain;United Kingdom;
2007 - EUCTR2007-002840-21-GB Italy;Spain;United Kingdom;
GA-GCB (velaglucerase alfa)
Shire
2007 Phase 2/Phase 3 NCT00478647 Israel;Poland;Spain;United Kingdom;United States;
GC1111_
Green Cross Corporation
2010 Phase 1/Phase 2 NCT01301898 Korea, Republic of;
GC1119
Green Cross Corporation
2012 Phase 1 NCT01653444 Korea, Republic of;
GLA gene
Chiayi Christian Hospital
2016 - NCT03596398 -
GLA genetic sequencing.
Chang Gung Memorial Hospital
2018 - NCT05056636 Taiwan;
GLUCOCEREBROSIDASE UMANO GENE-ATTIVATO
SHIRE HUMAN GENETIC THERAPIES, INC
2018 Phase 4 EUCTR2015-001578-17-IT Germany;Israel;Italy;Spain;United Kingdom;United States;
GNR-055
AO GENERIUM
2020 Phase 1 NCT04539340 Russian Federation;
GR181413
GLAXOSMITHKLINE RESEARCH & DEVELOPMENT LTD.
2012 - EUCTR2011-004800-40-IT Australia;Austria;Belgium;Brazil;Denmark;Egypt;Italy;Spain;Turkey;United Kingdom;United States;
GlaxoSmithKline Research & Development Limited
2012 Phase 3 EUCTR2011-004800-40-ES Australia;Austria;Belgium;Brazil;Denmark;Egypt;France;Italy;Spain;Turkey;United Kingdom;United States;
GR181413A
Amicus Therapeutics, Inc.
2014 Phase 3 EUCTR2011-004800-40-AT Argentina;Australia;Austria;Belgium;Brazil;Canada;Denmark;Egypt;France;Italy;Spain;Turkey;United Kingdom;United States;
2013 Phase 3 EUCTR2011-004800-40-BE Argentina;Australia;Austria;Belgium;Brazil;Canada;Denmark;Egypt;France;Italy;Spain;Turkey;United Kingdom;United States;
2012 Phase 3 EUCTR2011-004800-40-GB Argentina;Australia;Austria;Belgium;Brazil;Canada;Denmark;Egypt;France;Italy;Spain;Turkey;United Kingdom;United States;
2012 Phase 3 EUCTR2011-004800-40-DK Argentina;Australia;Austria;Belgium;Brazil;Canada;Denmark;Egypt;France;Italy;Spain;Turkey;United Kingdom;United States;
GLAXOSMITHKLINE RESEARCH & DEVELOPMENT LTD.
2012 - EUCTR2011-004800-40-IT Australia;Austria;Belgium;Brazil;Denmark;Egypt;Italy;Spain;Turkey;United Kingdom;United States;
GlaxoSmithKline Research & Development Limited
2012 Phase 3 EUCTR2011-004800-40-ES Australia;Austria;Belgium;Brazil;Denmark;Egypt;France;Italy;Spain;Turkey;United Kingdom;United States;
GR181413A/AT1001 capsule
Amicus Therapeutics
2011 Phase 1 NCT01853852 Australia;
GR181413A/AT1001 solution
Amicus Therapeutics
2011 Phase 1 NCT01853852 Australia;
GSK2788723
GlaxoSmithKline
2012 - NCT01602601 Japan;
GZ/SAR402671
Genzyme, a Sanofi Company
2015 Phase 2 NCT02489344 France;Poland;Russian Federation;United Kingdom;United States;
2014 Phase 2 NCT02228460 Czech Republic;Czechia;France;Poland;Russian Federation;United Kingdom;United States;
Sanofi-aventis recherche & développement
- Phase 3 EUCTR2021-005402-10-FR Argentina;Brazil;Canada;China;Egypt;France;Germany;Hungary;Japan;Taiwan;Turkey;United Kingdom;United States;
- Phase 3 EUCTR2021-005402-10-DE Argentina;Brazil;Canada;China;Egypt;France;Germany;Hungary;Japan;Taiwan;Turkey;United Kingdom;United States;
GZ385660
Genzyme Corporation
2019 Phase 3 EUCTR2016-000301-37-NL Argentina;Canada;Egypt;France;Italy;Netherlands;Russian Federation;Spain;Sweden;Turkey;United Kingdom;
2019 Phase 3 EUCTR2016-000301-37-GB Argentina;Canada;Egypt;France;Italy;Netherlands;Russian Federation;Spain;Sweden;Turkey;United Kingdom;
2018 Phase 3 EUCTR2016-000301-37-SE Argentina;Canada;Egypt;France;Italy;Japan;Netherlands;Russian Federation;Spain;Sweden;Taiwan;Turkey;United Kingdom;
2018 Phase 3 EUCTR2016-000301-37-IT Argentina;Canada;Egypt;France;Italy;Netherlands;Russian Federation;Spain;Sweden;Turkey;United Kingdom;
2018 Phase 3 EUCTR2016-000301-37-ES Argentina;Canada;Egypt;France;Italy;Netherlands;Russian Federation;Spain;Sweden;Turkey;United Kingdom;
GZ402665
Genzyme Corporation
2015 Phase 2 EUCTR2014-003198-40-IT Brazil;Chile;France;Germany;Italy;United Kingdom;United States;
2015 Phase 2 EUCTR2014-003198-40-GB Brazil;France;Germany;Italy;United Kingdom;United States;
2015 Phase 2 EUCTR2014-003198-40-DE Brazil;France;Germany;Italy;United Kingdom;United States;
- Phase 2 EUCTR2014-003198-40-FR Brazil;Chile;France;Germany;Italy;United Kingdom;United States;
GZ402671
GENZYME CORPORATION
2020 Phase 3 EUCTR2019-002375-34-IT Argentina;Austria;Brazil;Czech Republic;Czechia;France;Germany;Italy;Japan;Portugal;Russian Federation;Spain;Turkey;United Kingdom;United States;
Genzyme Corporation
2021 Phase 3 EUCTR2019-002375-34-AT Argentina;Austria;Brazil;Czech Republic;Czechia;France;Germany;Italy;Japan;Portugal;Russian Federation;Spain;Turkey;United Kingdom;United States;
2021 Phase 2;Phase 3 EUCTR2020-003120-17-HU Argentina;Brazil;Canada;China;Egypt;France;Germany;Hungary;Japan;Sweden;Taiwan;Turkey;United States;
2021 Phase 2;Phase 3 EUCTR2020-003120-17-FR Argentina;Brazil;Canada;China;Egypt;France;Germany;Hungary;Japan;Sweden;Taiwan;Turkey;United States;
2021 Phase 2;Phase 3 EUCTR2020-003120-17-DE Argentina;Brazil;Canada;China;Egypt;France;Germany;Hungary;Japan;Sweden;Taiwan;Turkey;United States;
2020 Phase 3 EUCTR2019-002375-34-PT Argentina;Austria;Brazil;Czech Republic;Czechia;France;Germany;Italy;Japan;Portugal;Russian Federation;Spain;Turkey;United Kingdom;United States;
2020 Phase 3 EUCTR2019-002375-34-GB Argentina;Austria;Brazil;Czech Republic;France;Germany;Italy;Japan;Portugal;Russian Federation;Spain;Turkey;United Kingdom;United States;
2020 Phase 3 EUCTR2019-002375-34-DE Argentina;Austria;Brazil;Czech Republic;France;Germany;Italy;Japan;Portugal;Russian Federation;Spain;Turkey;United Kingdom;United States;
2020 Phase 3 EUCTR2019-002375-34-CZ Argentina;Austria;Brazil;Czech Republic;France;Germany;Italy;Japan;Portugal;Russian Federation;Spain;Turkey;United Kingdom;United States;
2016 Phase 2 EUCTR2014-002550-39-DE Germany;Japan;United Kingdom;United States;
- Phase 3 EUCTR2019-002375-34-FR Argentina;Austria;Brazil;Czech Republic;France;Germany;Italy;Japan;Portugal;Russian Federation;Spain;Turkey;United Kingdom;United States;
- Phase 2;Phase 3 EUCTR2020-003120-17-SE Argentina;Brazil;Canada;China;Egypt;France;Germany;Hungary;Japan;Sweden;Taiwan;Turkey;United States;
GZ402671 / SAR402671
Genzyme Corporation
2014 Phase 2 EUCTR2013-005324-41-GB Czech Republic;France;Poland;Russian Federation;United Kingdom;United States;
2014 Phase 2 EUCTR2013-005324-41-CZ Czech Republic;France;Poland;Russian Federation;United Kingdom;United States;
Gabapentin
University of Minnesota
2012 Phase 2 NCT01588314 United States;
Galafold
Amicus Therapeutics UK, Ltd
2020 Phase 3 EUCTR2017-000146-21-IT Australia;Germany;Italy;Spain;United Kingdom;United States;
Amicus Therapeutics, UK Ltd
2019 Phase 3 EUCTR2017-000146-21-GB Australia;Germany;Italy;Spain;United Kingdom;United States;
2019 Phase 3 EUCTR2017-000146-21-ES Australia;Germany;Italy;Spain;United Kingdom;United States;
- Phase 3 EUCTR2017-000146-21-DE Australia;Germany;Italy;Spain;United Kingdom;United States;
SANOFI-AVENTIS RECHERCHE E DEVELOPPEMENT
2022 Phase 3 EUCTR2021-002320-20-IT Austria;Canada;China;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Turkey;United Kingdom;United States;
Sanofi Aventis Groupe (SAG)
- Phase 4 EUCTR2019-000065-20-NO Canada;France;Norway;United States;
sanofi-aventis recherche et développement
- Phase 3 EUCTR2021-002320-20-ES Austria;Canada;China;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Turkey;United Kingdom;United States;
Galsulfase
BioMarin Pharmaceutical Inc.
2007 Phase 4 EUCTR2005-003512-30-FR France;Portugal;
Gene Activated Human Glucocerebrosidase
SHIRE HUMAN GENETIC THERAPIES, INC.
2008 - EUCTR2007-002840-21-IT Italy;Spain;United Kingdom;
Gene Activated a-Galactosidase A
Shire Human Genetic Therapies, Inc.
2021 Phase 3 EUCTR2018-004689-32-SE Argentina;Australia;Belgium;Bosnia and Herzegovina;Brazil;Canada;Croatia;Finland;Germany;Greece;Hungary;Italy;Latvia;Poland;Portugal;Romania;Slovenia;Spain;Sweden;Ukraine;
2021 Phase 3 EUCTR2018-004689-32-PT Argentina;Australia;Belgium;Bosnia and Herzegovina;Brazil;Canada;Croatia;Finland;Germany;Greece;Hungary;Italy;Latvia;Poland;Portugal;Romania;Slovenia;Spain;Sweden;Ukraine;
2021 Phase 3 EUCTR2018-004689-32-PL Argentina;Australia;Belgium;Bosnia and Herzegovina;Brazil;Canada;Croatia;Finland;Germany;Greece;Hungary;Italy;Latvia;Poland;Portugal;Romania;Slovenia;Spain;Sweden;Ukraine;
2021 Phase 3 EUCTR2018-004689-32-GR Argentina;Australia;Belgium;Bosnia and Herzegovina;Brazil;Canada;Croatia;Finland;Germany;Greece;Hungary;Italy;Latvia;Poland;Portugal;Romania;Slovenia;Spain;Sweden;Ukraine;
2021 Phase 3 EUCTR2018-004689-32-FI Argentina;Australia;Belgium;Bosnia and Herzegovina;Brazil;Canada;Croatia;Finland;Germany;Greece;Hungary;Italy;Latvia;Poland;Portugal;Romania;Slovenia;Spain;Sweden;Ukraine;
Gene activated human glucocerebrosidase
Shire Human Genetic Therapies Inc
2007 Phase 2;Phase 3 EUCTR2006-006304-11-GB Germany;Italy;Spain;United Kingdom;
2007 - EUCTR2006-006304-11-ES Italy;Spain;United Kingdom;
- Phase 2;Phase 3 EUCTR2006-006304-11-DE Germany;Italy;Spain;United Kingdom;
Shire Human Genetic Therapies, Inc.
2008 - EUCTR2008-001965-27-ES Spain;United Kingdom;
Gene activated human glucocerebrosidase, velaglucerase alfa
Shire Human Genetic Therapies, Inc.
2016 Phase 4 EUCTR2015-001578-17-GB Canada;Germany;Israel;Spain;United Kingdom;United States;
2016 Phase 4 EUCTR2015-001578-17-DE Germany;India;Israel;Italy;Spain;Turkey;United Kingdom;United States;
2016 - EUCTR2015-001578-17-ES Canada;Germany;Israel;Italy;Spain;United Kingdom;United States;
2008 Phase 3 EUCTR2008-001965-27-GB Spain;United Kingdom;
Gene-Activated Human Glucocerebrosidase
SHIRE HUMAN GENETIC THERAPIES, INC.
2008 - EUCTR2006-006304-11-IT Italy;Spain;United Kingdom;
Shire Human Genetic Therapies Inc
2007 Phase 2;Phase 3 EUCTR2006-006304-11-GB Germany;Italy;Spain;United Kingdom;
2007 - EUCTR2006-006304-11-ES Italy;Spain;United Kingdom;
- Phase 2;Phase 3 EUCTR2006-006304-11-DE Germany;Italy;Spain;United Kingdom;
Shire Human Genetic Therapies, Inc.
2008 - EUCTR2008-001965-27-ES Spain;United Kingdom;
Gene-Activated Human Glucocerebrosidase 200U/vial
Shire Human Genetic Therapies, Inc.
2008 Phase 3 EUCTR2008-001965-27-GB Spain;United Kingdom;
Gene-Activated Human Glucocerebrosidase 400U/vial
Shire Human Genetic Therapies
- - EUCTR2012-003427-38-Outside-EU/EEA Egypt;India;Tunisia;
Shire Human Genetic Therapies, Inc.
2008 Phase 3 EUCTR2008-001965-27-GB Spain;United Kingdom;
Genetic HMI-203
Homology Medicines, Inc
2022 Phase 1 NCT05238324 United States;
Genistein aglycone
Manchester University NHS Foundation Trust
2014 Phase 3 EUCTR2013-001479-18-GB United Kingdom;
Genz-112638
Genzyme Europe B.V.
2011 Phase 3 EUCTR2009-015811-42-PT Argentina;Australia;Austria;Brazil;Canada;China;Croatia;France;Greece;India;Japan;Netherlands;Portugal;Romania;Russian Federation;Serbia;Sweden;United States;
2011 Phase 3 EUCTR2009-015811-42-AT Argentina;Australia;Austria;Brazil;Canada;China;Croatia;France;Greece;India;Japan;Netherlands;Portugal;Romania;Russian Federation;Serbia;Sweden;United States;
2011 - EUCTR2009-015811-42-GR Austria;Greece;Portugal;Sweden;
2010 Phase 3 EUCTR2009-015811-42-SE Argentina;Australia;Austria;Brazil;Canada;China;Croatia;France;Greece;India;Japan;Netherlands;Portugal;Romania;Russian Federation;Serbia;Sweden;United States;
2010 - EUCTR2008-005223-28-ES Czech Republic;France;Germany;Italy;Netherlands;Spain;United Kingdom;
2010 - EUCTR2008-005223-28-DE Argentina;Australia;Brazil;Canada;Czech Republic;Egypt;France;Germany;Italy;Netherlands;Russian Federation;Spain;Turkey;United Kingdom;United States;
2010 - EUCTR2008-005222-37-BG Bulgaria;Canada;Colombia;India;Israel;Jordan;Lebanon;Mexico;Netherlands;Russian Federation;Saudi Arabia;Serbia;Tunisia;United Kingdom;United States;
2009 Phase 3 EUCTR2008-005223-28-GB Argentina;Australia;Brazil;Canada;Czech Republic;Egypt;France;Germany;Italy;Netherlands;Russian Federation;Spain;Turkey;United Kingdom;United States;
2009 Phase 3 EUCTR2008-005223-28-FR Czech Republic;France;Germany;Italy;Netherlands;Spain;United Kingdom;
2009 Phase 3 EUCTR2008-005222-37-GB Bulgaria;Canada;Colombia;India;Israel;Jordan;Lebanon;Mexico;Netherlands;Russian Federation;Saudi Arabia;Serbia;Tunisia;United Kingdom;United States;
2009 - EUCTR2008-005223-28-NL Czech Republic;France;Germany;Italy;Netherlands;Spain;United Kingdom;
2009 - EUCTR2008-005223-28-CZ Czech Republic;France;Germany;Italy;Netherlands;Spain;United Kingdom;
2009 - EUCTR2008-005222-37-NL Bulgaria;Netherlands;United Kingdom;
Genzyme Europe BV
2010 - EUCTR2008-005223-28-IT Czech Republic;France;Germany;Italy;Netherlands;Spain;United Kingdom;
2006 - EUCTR2005-004732-42-IT Italy;
Genz-682452-AA
Genzyme Corporation
2014 Phase 2 EUCTR2013-005324-41-GB Czech Republic;France;Poland;Russian Federation;United Kingdom;United States;
2014 Phase 2 EUCTR2013-005324-41-CZ Czech Republic;France;Poland;Russian Federation;United Kingdom;United States;
Genz-682452-AU
GENZYME CORPORATION
2020 Phase 3 EUCTR2019-002375-34-IT Argentina;Austria;Brazil;Czech Republic;Czechia;France;Germany;Italy;Japan;Portugal;Russian Federation;Spain;Turkey;United Kingdom;United States;
Genzyme Corporation
2021 Phase 3 EUCTR2019-002375-34-AT Argentina;Austria;Brazil;Czech Republic;Czechia;France;Germany;Italy;Japan;Portugal;Russian Federation;Spain;Turkey;United Kingdom;United States;
2021 Phase 2;Phase 3 EUCTR2020-003120-17-HU Argentina;Brazil;Canada;China;Egypt;France;Germany;Hungary;Japan;Sweden;Taiwan;Turkey;United States;
2021 Phase 2;Phase 3 EUCTR2020-003120-17-FR Argentina;Brazil;Canada;China;Egypt;France;Germany;Hungary;Japan;Sweden;Taiwan;Turkey;United States;
2021 Phase 2;Phase 3 EUCTR2020-003120-17-DE Argentina;Brazil;Canada;China;Egypt;France;Germany;Hungary;Japan;Sweden;Taiwan;Turkey;United States;
2020 Phase 3 EUCTR2019-002375-34-PT Argentina;Austria;Brazil;Czech Republic;Czechia;France;Germany;Italy;Japan;Portugal;Russian Federation;Spain;Turkey;United Kingdom;United States;
2020 Phase 3 EUCTR2019-002375-34-GB Argentina;Austria;Brazil;Czech Republic;France;Germany;Italy;Japan;Portugal;Russian Federation;Spain;Turkey;United Kingdom;United States;
2020 Phase 3 EUCTR2019-002375-34-DE Argentina;Austria;Brazil;Czech Republic;France;Germany;Italy;Japan;Portugal;Russian Federation;Spain;Turkey;United Kingdom;United States;
2020 Phase 3 EUCTR2019-002375-34-CZ Argentina;Austria;Brazil;Czech Republic;France;Germany;Italy;Japan;Portugal;Russian Federation;Spain;Turkey;United Kingdom;United States;
2016 Phase 2 EUCTR2014-002550-39-DE Germany;Japan;United Kingdom;United States;
2015 Phase 2 EUCTR2014-004995-49-PL Czech Republic;France;Poland;Russian Federation;United Kingdom;United States;
2015 Phase 2 EUCTR2014-004995-49-GB Czech Republic;France;Poland;Russian Federation;United Kingdom;United States;
2015 Phase 2 EUCTR2014-004995-49-FR Czech Republic;France;Poland;Russian Federation;United Kingdom;United States;
- Phase 3 EUCTR2019-002375-34-FR Argentina;Austria;Brazil;Czech Republic;France;Germany;Italy;Japan;Portugal;Russian Federation;Spain;Turkey;United Kingdom;United States;
- Phase 2;Phase 3 EUCTR2020-003120-17-SE Argentina;Brazil;Canada;China;Egypt;France;Germany;Hungary;Japan;Sweden;Taiwan;Turkey;United States;
Glucocerebrosidase
National Center for Research Resources (NCRR)
1999 Phase 2 NCT00004293 United States;
HGT-1110
Shire
2013 Phase 1/Phase 2 NCT01887938 Argentina;Australia;Brazil;Czech Republic;Czechia;Denmark;France;Germany;Japan;
Shire Human Genetics Therapies Inc
2012 Phase 1;Phase 2 EUCTR2011-002044-28-DE Australia;Brazil;Denmark;France;Germany;Japan;
2011 Phase 1;Phase 2 EUCTR2011-002044-28-DK Australia;Brazil;Denmark;France;Germany;Japan;
- Phase 1;Phase 2 EUCTR2012-003775-20-FR Argentina;Australia;Brazil;Czech Republic;Denmark;France;Germany;United Kingdom;
HGT-1111
Shire
2008 Phase 2 NCT00681811 Denmark;
Shire Human Genetic Therapies Inc
2010 Phase 2 EUCTR2008-000084-41-BE Belgium;Denmark;France;Italy;
2009 Phase 2 EUCTR2008-000084-41-IT Belgium;Denmark;France;Italy;
2009 - EUCTR2008-000084-41-FR Belgium;Denmark;France;Italy;
2008 Phase 2 EUCTR2008-000084-41-DK Belgium;Denmark;France;Italy;
HGT-1410
SHIRE HUMAN GENETIC THERAPIES, INC
2015 Phase 2 EUCTR2014-003960-20-IT France;Germany;Italy;Netherlands;Spain;United Kingdom;United States;
Shire
2015 Phase 2 NCT02350816 France;Germany;Italy;Netherlands;Spain;United Kingdom;United States;
Shire Human Genetic Therapies, Inc
2014 Phase 2 EUCTR2013-003450-24-NL Argentina;France;Germany;Italy;Netherlands;Spain;United Kingdom;United States;
2014 Phase 2 EUCTR2013-003450-24-IT France;Germany;Italy;Netherlands;Spain;United Kingdom;
2014 Phase 2 EUCTR2013-003450-24-GB Argentina;Brazil;France;Germany;Italy;Netherlands;Spain;United Kingdom;United States;
2014 Phase 2 EUCTR2013-003450-24-ES Argentina;France;Germany;Italy;Netherlands;Spain;United Kingdom;United States;
2014 Phase 2 EUCTR2013-003450-24-DE Argentina;Brazil;France;Germany;Italy;Netherlands;Spain;United Kingdom;United States;
2011 Phase 1;Phase 2 EUCTR2010-021348-16-NL Netherlands;United Kingdom;
2011 Phase 1;Phase 2 EUCTR2010-021348-16-GB Netherlands;United Kingdom;
- Phase 2 EUCTR2013-003450-24-FR Argentina;Brazil;France;Germany;Italy;Netherlands;Spain;United Kingdom;United States;
Shire Human Genetic Therapies, Inc.
2015 Phase 2 EUCTR2014-003960-20-NL France;Germany;Italy;Netherlands;Spain;United Kingdom;United States;
2015 Phase 2 EUCTR2014-003960-20-GB France;Germany;Italy;Netherlands;Spain;United Kingdom;United States;
2015 Phase 2 EUCTR2014-003960-20-DE France;Germany;Italy;Netherlands;Spain;United Kingdom;United States;
Shire Human Genetic Therapies, Inc. (Shire HGT)
2010 Phase 1;Phase 2 EUCTR2009-015984-15-GB United Kingdom;
Shire human Genetic Therapies, Inc.
2015 Phase 2 EUCTR2014-003960-20-ES Argentina;France;Germany;Italy;Netherlands;Spain;United Kingdom;United States;
- Phase 2 EUCTR2014-003960-20-FR Argentina;France;Germany;Italy;Netherlands;Spain;United Kingdom;United States;
HGT-2310
Shire HGT Inc
2013 Phase 2;Phase 3 EUCTR2013-002885-38-GB Argentina;Australia;Canada;Colombia;France;Germany;Mexico;Spain;United Kingdom;United States;
2013 Phase 2;Phase 3 EUCTR2013-002885-38-ES Argentina;Mexico;Spain;United Kingdom;United States;
- Phase 2;Phase 3 EUCTR2013-002885-38-FR Argentina;Australia;France;Germany;Mexico;Spain;United Kingdom;United States;
Shire Human Genetic Therapies, Inc.
2015 Phase 2;Phase 3 EUCTR2014-004143-13-GB Argentina;Australia;Canada;Colombia;France;Mexico;Spain;United Kingdom;United States;
2015 - EUCTR2014-004143-13-ES Argentina;Canada;Colombia;Mexico;Spain;United Kingdom;United States;
- Phase 2;Phase 3 EUCTR2014-004143-13-FR Argentina;Australia;Canada;Colombia;France;Mexico;Spain;United Kingdom;United States;
HYDROXYPROPYLBETADEX
Cyclo Therapeutics, Inc.
2022 Phase 3 EUCTR2020-003136-25-PL Australia;France;Germany;Israel;Italy;Poland;United Kingdom;United States;
2022 Phase 3 EUCTR2020-003136-25-DE Australia;France;Germany;Israel;Italy;Poland;United Kingdom;United States;
2021 Phase 3 EUCTR2020-003136-25-FR Australia;France;Israel;United Kingdom;United States;
- Phase 3 EUCTR2020-003136-25-IT Australia;France;Germany;Israel;Italy;Poland;Spain;United Kingdom;United States;
Hematopoetic Stem Cell Transplantation
University of Pittsburgh
2012 - NCT03333200 United States;
Hematopoietic Stem Cell Transplantation
Masonic Cancer Center, University of Minnesota
2006 Phase 2 NCT00383448 United States;
Hematopoietic stem cell infusion
Talaris Therapeutics Inc.
2011 Phase 1/Phase 2 NCT01372228 United States;
Hematopoietic stem cell transplantation
Masonic Cancer Center, University of Minnesota
2012 - NCT01626092 United States;
Heparán N sulfatasa humana recombinante (rhHNS)
Shire Human Genetic Therapies, Inc
2014 Phase 2 EUCTR2013-003450-24-ES Argentina;France;Germany;Italy;Netherlands;Spain;United Kingdom;United States;
HuCNS-SC
StemCells, Inc.
2010 Phase 1 NCT01238315 United States;
Human Glucocerebrosidase (prGCD)
Protalix
2005 Phase 1 NCT00258778 Israel;
Human Placental Derived Stem Cell
New York Medical College
2013 Phase 1 NCT01586455 United States;
Hunterase
Green Cross Corporation
2012 Phase 3 NCT01645189 Korea, Republic of;
Hydroxypropyl-beta-cyclodextrin
CTD Holdings, Inc.
2016 Phase 1;Phase 2 EUCTR2015-005761-23-GB Italy;Sweden;United Kingdom;
Cyclo Therapeutics, Inc.
2021 Phase 3 NCT04860960 United States;
2019 Phase 1 NCT03893071 United States;
2017 Phase 1;Phase 2 EUCTR2015-005761-23-SE Italy;Sweden;United Kingdom;
2017 Phase 1/Phase 2 NCT02912793 Israel;Sweden;United Kingdom;
2017 Phase 1 NCT02939547 United States;
Hydroxypropyl-ß-cyclodextrin
Cyclo Therapeutics, Inc.
2019 Phase 1 NCT03893071 United States;
Hydroxyurea
Masonic Cancer Center, University of Minnesota
2006 Phase 2 NCT00383448 United States;
I2S
TKT INC
2005 - EUCTR2004-002743-27-IT Germany;Italy;Spain;Sweden;United Kingdom;
I2S-IT
Shire HGT Inc
- - EUCTR2011-000212-25-Outside-EU/EEA Canada;United States;
Shire HGT, Inc
2011 Phase 1;Phase 2 EUCTR2010-020048-36-GB United Kingdom;United States;
IB1001
IntraBio Inc
2019 Phase 2 NCT03759665 Germany;Spain;United Kingdom;United States;
2019 Phase 2 NCT03759639 Germany;Slovakia;Spain;United Kingdom;United States;
IntraBio Ltd
2022 Phase 3 EUCTR2021-005356-10-SK Australia;Czechia;Germany;Netherlands;Slovakia;Switzerland;United Kingdom;United States;
2022 Phase 3 EUCTR2021-005356-10-DE Australia;Czechia;Germany;Netherlands;Slovakia;Spain;Switzerland;United Kingdom;United States;
2019 Phase 2 EUCTR2018-004406-25-GB Germany;Spain;United Kingdom;United States;
2019 Phase 2 EUCTR2018-004406-25-ES Germany;Spain;United Kingdom;United States;
2019 Phase 2 EUCTR2018-004406-25-DE Germany;Spain;United Kingdom;United States;
2019 Phase 2 EUCTR2018-004331-71-SK Germany;Slovakia;Spain;United Kingdom;United States;
2019 Phase 2 EUCTR2018-004331-71-GB Germany;Slovakia;Spain;United Kingdom;United States;
2019 Phase 2 EUCTR2018-004331-71-ES Germany;Slovakia;Spain;United Kingdom;United States;
2019 Phase 2 EUCTR2018-004331-71-DE Germany;Slovakia;Spain;United Kingdom;United States;
- Phase 3 EUCTR2021-005356-10-NL Australia;Czechia;Germany;Netherlands;Slovakia;Spain;Switzerland;United Kingdom;United States;
IDURONATE-2-SULFATASE FUSED TO A FC POLYPEPTIDE THAT BINDS TO THE HUMAN TRANSFERRIN RECEPTOR
Denali Therapeutics Inc.
2021 Phase 1;Phase 2 EUCTR2019-004909-27-NL Germany;Italy;Netherlands;United Kingdom;United States;
2020 Phase 1;Phase 2 EUCTR2019-004909-27-GB Germany;Italy;Netherlands;United Kingdom;United States;
IMD Preparative Regimen
Masonic Cancer Center, University of Minnesota
2014 Phase 2 NCT02171104 United States;
INN Not available
Genzyme Europe B.V.
2010 - EUCTR2008-005223-28-ES Czech Republic;France;Germany;Italy;Netherlands;Spain;United Kingdom;
IRT Laronidase
Masonic Cancer Center, University of Minnesota
2008 Phase 1 NCT00638547 United States;
ISU302
ISU Abxis Co., Ltd.
2011 Phase 3 NCT02770625 -
2011 Phase 3 NCT01161914 -
2010 Phase 1 NCT01881633 -
IV migalastat HCl
Amicus Therapeutics
2014 Phase 1 NCT02082327 Netherlands;
IVA 336
Inventiva S.A.
2019 Phase 2 EUCTR2017-002158-35-PT France;Germany;Portugal;United Kingdom;
2018 Phase 2 EUCTR2017-002158-35-DE France;Germany;Portugal;United Kingdom;
2017 Phase 2 EUCTR2017-002158-35-GB France;Germany;Portugal;United Kingdom;
- Phase 2 EUCTR2017-002158-35-FR France;Germany;Portugal;United Kingdom;
Iduronate-2-sulfatase enzyme replacement therapy
Shire
2003 Phase 2/Phase 3 NCT00069641 Brazil;Germany;United Kingdom;United States;
Idursulfase
GlaxoSmithKline
2012 - NCT01602601 Japan;
Shire
2008 - NCT00882921 Brazil;United Kingdom;United States;
2007 Phase 4 NCT00607386 Brazil;Poland;Taiwan;
2004 Phase 2/Phase 3 NCT00630747 Brazil;Canada;France;Germany;Italy;Romania;Spain;Sweden;United Kingdom;United States;
TKT INC
2005 - EUCTR2004-002743-27-IT Germany;Italy;Spain;Sweden;United Kingdom;
Idursulfase (I2S)
Shire Human Genetic Therapies INC.
2005 - EUCTR2004-002743-27-SE Germany;Italy;Spain;Sweden;United Kingdom;
2004 - EUCTR2004-002743-27-DE Germany;Italy;Spain;Sweden;United Kingdom;
Shire Human Genetic Therapies, Inc. (Shire HGT)
- Phase 4 EUCTR2007-006044-22-PL Poland;
TKT Inc.
2005 Phase 3 EUCTR2004-002743-27-GB Germany;Italy;Spain;Sweden;United Kingdom;
2005 - EUCTR2004-002743-27-ES Germany;Italy;Spain;Sweden;United Kingdom;
Idursulfase (I2S)-IT
Shire HGT, Inc
2011 Phase 1;Phase 2 EUCTR2010-020048-36-GB United Kingdom;United States;
Idursulfase IT (1 mg)
Shire
2009 Phase 1/Phase 2 NCT00920647 United Kingdom;United States;
Idursulfase IT (10 mg)
Shire
2009 Phase 1/Phase 2 NCT00920647 United Kingdom;United States;
Idursulfase IT (30 mg)
Shire
2009 Phase 1/Phase 2 NCT00920647 United Kingdom;United States;
Idursulfase beta
Green Cross Corporation
2016 Phase 2 NCT02663024 -
Idursulfase(12S)-IT
Shire HGT Inc
2012 Phase 1;Phase 2 EUCTR2011-000212-25-GB Canada;United Kingdom;United States;
Idursulfase(I2S)-IT
Shire HGT Inc
- - EUCTR2011-000212-25-Outside-EU/EEA Canada;United States;
Idursulfase-IT
Shire
2015 Phase 2/Phase 3 NCT02412787 Argentina;Australia;Canada;Colombia;France;Mexico;Spain;United Kingdom;United States;
2014 Phase 2/Phase 3 NCT02055118 Argentina;Australia;Canada;Colombia;France;Mexico;Spain;United Kingdom;United States;
2010 Phase 1/Phase 2 NCT01506141 Canada;United Kingdom;United States;
Shire HGT Inc
2013 Phase 2;Phase 3 EUCTR2013-002885-38-GB Argentina;Australia;Canada;Colombia;France;Germany;Mexico;Spain;United Kingdom;United States;
2013 Phase 2;Phase 3 EUCTR2013-002885-38-ES Argentina;Mexico;Spain;United Kingdom;United States;
2012 Phase 1;Phase 2 EUCTR2011-000212-25-GB Canada;United Kingdom;United States;
- Phase 2;Phase 3 EUCTR2013-002885-38-FR Argentina;Australia;France;Germany;Mexico;Spain;United Kingdom;United States;
- - EUCTR2011-000212-25-Outside-EU/EEA Canada;United States;
Shire HGT, Inc
2011 Phase 1;Phase 2 EUCTR2010-020048-36-GB United Kingdom;United States;
Shire Human Genetic Therapies, Inc.
2015 Phase 2;Phase 3 EUCTR2014-004143-13-GB Argentina;Australia;Canada;Colombia;France;Mexico;Spain;United Kingdom;United States;
2015 - EUCTR2014-004143-13-ES Argentina;Canada;Colombia;Mexico;Spain;United Kingdom;United States;
- Phase 2;Phase 3 EUCTR2014-004143-13-FR Argentina;Australia;Canada;Colombia;France;Mexico;Spain;United Kingdom;United States;
Imiglucerase
Genzyme Europe BV
2010 - EUCTR2008-005223-28-IT Czech Republic;France;Germany;Italy;Netherlands;Spain;United Kingdom;
Genzyme, a Sanofi Company
2022 Phase 3 NCT05222906 -
2017 Phase 2 NCT02843035 Germany;Japan;United Kingdom;United States;
2009 Phase 3 NCT00943111 Argentina;Australia;Brazil;Canada;Czech Republic;Egypt;France;Germany;Italy;Netherlands;Russian Federation;Spain;Turkey;United Kingdom;United States;
ISTITUTO GIANNINA GASLINI
2009 - EUCTR2007-005516-61-IT Italy;
SHIRE HUMAN GENETIC THERAPIES, INC.
2008 - EUCTR2007-002840-21-IT Italy;Spain;United Kingdom;
Sanofi-aventis recherche & développement
- Phase 3 EUCTR2021-005402-10-FR Argentina;Brazil;Canada;China;Egypt;France;Germany;Hungary;Japan;Taiwan;Turkey;United Kingdom;United States;
- Phase 3 EUCTR2021-005402-10-DE Argentina;Brazil;Canada;China;Egypt;France;Germany;Hungary;Japan;Taiwan;Turkey;United Kingdom;United States;
Shire
2008 Phase 3 NCT00553631 Argentina;India;Israel;Paraguay;Russian Federation;Spain;Tunisia;United Kingdom;United States;
University Research Foundation for Lysosomal Storage Diseases, Inc.
2010 - NCT01136304 United States;
Imiglucerase GZ437843
Sanofi
2018 Phase 3 NCT03485677 Argentina;Canada;France;Italy;Japan;Russian Federation;Spain;Sweden;Turkey;United Kingdom;
Imiglucerasi
SHIRE HUMAN GENETIC THERAPIES, INC.
2008 - EUCTR2007-002840-21-IT Italy;Spain;United Kingdom;
Imiglucérase (drug) pharmacokinetics
University Hospital, Clermont-Ferrand
2012 Phase 2 NCT01951989 France;
Inactive Reminder Capsules
Amicus Therapeutics, Inc.
2015 Phase 3 EUCTR2014-002701-38-GB Argentina;Australia;Austria;Belgium;Brazil;Canada;Denmark;Egypt;France;Italy;Japan;Spain;Turkey;United Kingdom;United States;
2015 Phase 3 EUCTR2014-002701-38-DK Argentina;Australia;Austria;Belgium;Brazil;Canada;Denmark;Egypt;France;Italy;Japan;Spain;Turkey;United Kingdom;United States;
2015 Phase 3 EUCTR2014-002701-38-BE Argentina;Australia;Austria;Belgium;Brazil;Canada;Denmark;Egypt;France;Italy;Japan;Spain;Turkey;United Kingdom;United States;
2014 Phase 3 EUCTR2014-002701-38-AT Argentina;Australia;Austria;Belgium;Brazil;Canada;Denmark;Egypt;France;Italy;Japan;Spain;Turkey;United Kingdom;United States;
2012 - EUCTR2010-022636-37-DE Argentina;Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Hungary;Israel;Italy;Japan;Mexico;Poland;Russian Federation;Slovakia;Switzerland;Taiwan;Turkey;United Kingdom;United States;
2011 Phase 3 EUCTR2010-022636-37-GR Argentina;Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Hungary;Israel;Italy;Japan;Mexico;Poland;Russian Federation;Slovakia;Switzerland;Taiwan;Turkey;United Kingdom;United States;
2011 - EUCTR2010-022636-37-GB Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;United Kingdom;United States;
2011 - EUCTR2010-022636-37-DK Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;United Kingdom;United States;
2011 - EUCTR2010-022636-37-BE Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;United Kingdom;United States;
2011 - EUCTR2010-022636-37-AT Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;United Kingdom;United States;
Inactive Reminder Product
Amicus Therapeutics, Inc.
2010 - EUCTR2009-013459-31-DK Argentina;Australia;Belgium;Brazil;Canada;Denmark;Egypt;France;Germany;Italy;Netherlands;South Africa;Spain;Turkey;United Kingdom;United States;
2009 Phase 3 EUCTR2009-013459-31-GB Argentina;Australia;Belgium;Brazil;Canada;Denmark;Egypt;France;Germany;Italy;Netherlands;South Africa;Spain;Turkey;United Kingdom;United States;
Intrathecal recombinant human alpha iduronidase
agnes chen
2014 - NCT02232477 United States;
Intraventricular Access Device
BioMarin Pharmaceutical
2015 Phase 1/Phase 2 NCT02485899 Germany;Italy;United Kingdom;United States;
Intraventricular access device
BioMarin Pharmaceutical
2016 Phase 2 NCT02678689 Germany;Italy;United Kingdom;United States;
Iohexol
Genzyme, a Sanofi Company
2013 - NCT01839526 Argentina;Austria;Belgium;Brazil;Canada;Finland;France;Hungary;Norway;Poland;Spain;Taiwan;United Kingdom;United States;
2012 - EUCTR2012-001966-14-ES Canada;Netherlands;Norway;Spain;United Kingdom;United States;
Isofagamine tartrate
Amicus Therapeutics, Inc.
2007 - EUCTR2007-003359-35-GB Germany;United Kingdom;
2007 - EUCTR2007-003359-35-DE Germany;United Kingdom;
JR-141
JCR Pharmaceuticals Co., Ltd.
2019 Phase 2/Phase 3 NCT04348136 Japan;
2019 Phase 2 NCT03708965 Brazil;
2018 Phase 2/Phase 3 NCT03568175 Japan;
2018 Phase 2 NCT03359213 Brazil;
2017 Phase 1/Phase 2 NCT03128593 Japan;
- Phase 3 EUCTR2020-003200-14-FR Brazil;France;Germany;United Kingdom;United States;
- Phase 3 EUCTR2020-003200-14-DE Brazil;France;Germany;United Kingdom;United States;
JR-141 or Idursulfase
JCR Pharmaceuticals Co., Ltd.
2022 Phase 3 NCT04573023 United States;
JR-171
JCR Pharmaceuticals Co., Ltd.
2021 Phase 1/Phase 2 NCT04453085 Brazil;Japan;
2020 Phase 1/Phase 2 NCT04227600 Brazil;Japan;United States;
Kanuma
Alexion Pharmaceuticals, Inc.
2013 Phase 3 EUCTR2011-002750-31-DE Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Japan;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;United States;
LYS-GM101
LYSOGENE
2021 Phase 1/Phase 2 NCT04273269 France;United Kingdom;United States;
Lysogene
- Phase 1;Phase 2 EUCTR2019-004949-32-FR France;United Kingdom;United States;
LYS-SAF302
Lysogene SA
2019 Phase 2;Phase 3 EUCTR2018-000195-15-NL France;Germany;Netherlands;United Kingdom;United States;
2019 Phase 2;Phase 3 EUCTR2018-000195-15-GB France;Germany;Netherlands;United Kingdom;United States;
2019 Phase 2;Phase 3 EUCTR2018-000195-15-DE France;Germany;Netherlands;United Kingdom;United States;
2018 Phase 2;Phase 3 EUCTR2018-000195-15-FR France;Germany;Netherlands;United Kingdom;United States;
Lamazym
Chiesi Farmaceutici S.p.A.
2013 Phase 3 NCT01908725 Denmark;
2013 Phase 3 NCT01908712 France;
Zymenex A/S
2015 Phase 3 NCT02478840 Denmark;
2013 Phase 3 EUCTR2012-000979-17-ES Belgium;Denmark;Germany;Spain;Sweden;
2013 - EUCTR2012-000979-17-SE Belgium;Denmark;Germany;Spain;Sweden;
2013 - EUCTR2012-000979-17-BE Belgium;Denmark;Germany;Spain;Sweden;
2012 Phase 3 NCT01681953 Denmark;France;Germany;Poland;United Kingdom;
2012 Phase 2 NCT01681940 Belgium;Denmark;Spain;United Kingdom;
2012 Phase 2 EUCTR2011-004355-40-DK Belgium;Denmark;Germany;Spain;United Kingdom;
2012 - EUCTR2012-000979-17-DK Belgium;Denmark;Germany;Spain;Sweden;
2012 - EUCTR2012-000979-17-DE Belgium;Denmark;Germany;Spain;Sweden;
2012 - EUCTR2011-004355-40-GB Belgium;Denmark;Germany;Spain;United Kingdom;
2012 - EUCTR2011-004355-40-ES Belgium;Denmark;Germany;Spain;United Kingdom;
2012 - EUCTR2011-004355-40-DE Belgium;Denmark;Germany;Spain;United Kingdom;
2012 - EUCTR2011-004355-40-BE Belgium;Denmark;Germany;Spain;United Kingdom;
2011 Phase 2 NCT01285700 Denmark;
2010 Phase 1 NCT01268358 -
2010 - EUCTR2010-022085-26-DK Denmark;
2010 - EUCTR2010-022084-36-DK Denmark;
Laronidase
Genzyme Europe BV
2010 - EUCTR2007-007003-33-IT Italy;
Genzyme, a Sanofi Company
2021 Phase 4 NCT05134571 China;
2008 Phase 1/Phase 2 NCT00741338 Brazil;Russian Federation;Ukraine;
2004 Phase 4 NCT00144768 United States;
Masonic Cancer Center, University of Minnesota
2012 Phase 1 NCT01173016 United States;
2012 - NCT01572636 United States;
Patricia I. Dickson, M.D.
2009 - NCT00852358 United States;
2008 Phase 1 NCT00786968 Finland;United States;
2005 Phase 1 NCT00215527 Finland;United States;
Laronidase ERT
Masonic Cancer Center, University of Minnesota
2004 Phase 2 NCT00176891 United States;
Ledipasvir/Sofosbuvir
Mansoura University Children Hospital
2018 Phase 4 NCT03721627 Egypt;
Lentivirus Alpha-gal A transduced stem cells
University Health Network, Toronto
2016 Phase 1 NCT02800070 Canada;
Lentivirus-mediated delivery of ARSA to the CNS.
Shenzhen Geno-Immune Medical Institute
2018 - NCT03725670 China;
Leucovorin
The Hospital for Sick Children
2009 Phase 1/Phase 2 NCT01102686 Canada;
Lipasa ácida lisosómica, esterasa, colesterol (gen humano LIPA), lipasa ácida lisosómica (gen humano LIPA)
Synageva BioPharma Corp.
2014 Phase 2 EUCTR2011-004287-30-ES Australia;Belgium;Brazil;Canada;Croatia;Denmark;Germany;Italy;Japan;Mexico;Netherlands;Poland;Russian Federation;Spain;Turkey;United Kingdom;United States;
Lisinopril
Salford Royal NHS Foundation Trust
2009 Phase 4 EUCTR2007-007482-21-GB United Kingdom;
Lopace
Salford Royal NHS Foundation Trust
2009 Phase 4 EUCTR2007-007482-21-GB United Kingdom;
Losartan
Hospital de Clinicas de Porto Alegre
2018 Phase 2 NCT03632213 Brazil;
Losartan and other angiotensin receptor blockers
University of Alabama at Birmingham
2007 - NCT00446862 Germany;Slovenia;United States;
Lucerastat
IDORSIA PHARMACEUTICALS LTD
2020 Phase 3 EUCTR2018-002210-12-IT Australia;Austria;Belgium;Canada;France;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;United Kingdom;United States;
2020 Phase 3 EUCTR2017-003369-85-IT Australia;Austria;Belgium;Canada;Finland;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;Switzerland;United Kingdom;United States;
Idorsia Pharmaceuticals Japan Ltd
2019 Phase 3 JPRN-JapicCTI-194975 Japan;
2019 Phase 3 JPRN-JapicCTI-194731 Japan;
Idorsia Pharmaceuticals Ltd
2021 Phase 3 EUCTR2018-002210-12-NO Australia;Austria;Belgium;Canada;France;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;United Kingdom;United States;
2021 Phase 3 EUCTR2018-002210-12-FR Austria;Belgium;Canada;Czech Republic;France;Germany;Netherlands;Poland;United Kingdom;United States;
2020 Phase 3 EUCTR2018-002210-12-BE Australia;Austria;Belgium;Canada;France;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;United Kingdom;United States;
2020 Phase 3 EUCTR2017-003369-85-NO Australia;Austria;Belgium;Canada;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;Switzerland;United Kingdom;United States;
2020 Phase 3 EUCTR2017-003369-85-IE Australia;Austria;Belgium;Canada;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;Switzerland;United Kingdom;United States;
2020 Phase 3 EUCTR2017-003369-85-ES Australia;Austria;Belgium;Canada;Germany;Ireland;Italy;Netherlands;Norway;Poland;Russian Federation;Spain;Switzerland;United Kingdom;United States;
2019 Phase 3 EUCTR2018-002210-12-NL Australia;Austria;Belgium;Canada;France;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;United Kingdom;United States;
2019 Phase 3 EUCTR2018-002210-12-GB Australia;Austria;Belgium;Canada;Germany;Ireland;Netherlands;Poland;United Kingdom;United States;
2019 Phase 3 EUCTR2018-002210-12-DE Australia;Austria;Belgium;Canada;France;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;United Kingdom;United States;
2019 Phase 3 EUCTR2018-002210-12-AT Australia;Austria;Belgium;Canada;France;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;United Kingdom;United States;
2018 Phase 3 EUCTR2017-003369-85-NL Australia;Austria;Belgium;Canada;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;Switzerland;United Kingdom;United States;
2018 Phase 3 EUCTR2017-003369-85-GB Australia;Austria;Belgium;Canada;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;Switzerland;United Kingdom;United States;
2018 Phase 3 EUCTR2017-003369-85-AT Australia;Austria;Belgium;Canada;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;Switzerland;United Kingdom;United States;
Idorsia Pharmaceuticals Ltd.
2018 Phase 3 NCT03737214 Australia;Austria;Belgium;Canada;Czechia;France;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;Switzerland;United Kingdom;United States;
2018 Phase 3 NCT03425539 Australia;Austria;Belgium;Canada;Czechia;France;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;Switzerland;United Kingdom;United States;
2015 Phase 1 NCT02930655 Germany;
Lumbar puncture
National Human Genome Research Institute (NHGRI)
2019 Phase 1/Phase 2 NCT03952637 United States;
Lysodase
National Institute of Mental Health (NIMH)
1993 Phase 1 NCT00001410 United States;
Lysosomal acid lipase, Esterase cholesterol (human gene LIPA), Lysosomal acid lipase (human gene LIPA)
Synageva BioPharma Corp.
2014 Phase 2 EUCTR2014-000533-22-IT Finland;Italy;United Kingdom;United States;
Lysosomal acid lipase, Esterase, cholesterol (human gene LIPA), Lysosomal acid lipase (human gene LIPA)
Alexion Pharmaceuitcals Inc
2014 Phase 2 EUCTR2011-004287-30-HR Australia;Belgium;Brazil;Canada;Croatia;Denmark;Germany;Italy;Japan;Mexico;Netherlands;Russian Federation;Spain;Turkey;United Kingdom;United States;
2014 Phase 2 EUCTR2011-004287-30-DE Australia;Belgium;Brazil;Canada;Croatia;Denmark;Germany;Italy;Japan;Mexico;Netherlands;Russian Federation;Spain;Turkey;United Kingdom;United States;
Alexion Pharmaceuticals Inc
2015 Phase 2 EUCTR2014-000533-22-FI Finland;Italy;United Kingdom;United States;
2015 Phase 2 EUCTR2011-004287-30-NL Australia;Belgium;Brazil;Canada;Croatia;Denmark;Germany;Italy;Mexico;Netherlands;Russian Federation;Spain;Turkey;United Kingdom;United States;
2015 Phase 2 EUCTR2011-004287-30-GB Australia;Belgium;Brazil;Canada;Croatia;Denmark;Germany;Italy;Japan;Mexico;Netherlands;Poland;Russian Federation;Spain;Turkey;United Kingdom;United States;
2014 Phase 2 EUCTR2014-000533-22-GB Finland;Italy;United Kingdom;United States;
Alexion Pharmaceuticals inc
2014 Phase 2 EUCTR2011-004287-30-DK Australia;Belgium;Brazil;Canada;Croatia;Denmark;Germany;Italy;Japan;Mexico;Netherlands;Poland;Russian Federation;Spain;Turkey;United Kingdom;United States;
Alexion Pharmaceuticals, Inc.
2015 Phase 3 EUCTR2011-002750-31-CZ Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Japan;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;United States;
2013 Phase 3 EUCTR2011-002750-31-GB Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Japan;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;United States;
2013 Phase 3 EUCTR2011-002750-31-DE Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Japan;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;United States;
- Phase 3 EUCTR2011-002750-31-PL Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Japan;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;United States;
Synageva BioPharma Corp.
2014 Phase 2 EUCTR2011-004287-30-IT Australia;Belgium;Brazil;Canada;Croatia;Denmark;Germany;Italy;Japan;Mexico;Netherlands;Poland;Russian Federation;Spain;Turkey;United Kingdom;United States;
2014 Phase 2 EUCTR2011-004287-30-BE Australia;Belgium;Brazil;Canada;Croatia;Denmark;Germany;Italy;Japan;Mexico;Netherlands;Russian Federation;Spain;Turkey;United Kingdom;United States;
Synageva BioPharma Corporation
2013 Phase 3 EUCTR2011-002750-31-IT Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;
2013 Phase 3 EUCTR2011-002750-31-HR Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;
2013 Phase 3 EUCTR2011-002750-31-GR Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;
2013 Phase 3 EUCTR2011-002750-31-ES Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;
- Phase 3 EUCTR2011-002750-31-FR Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;
MABTHERA - 2 FIALE 100 MG 10 ML
OSPEDALE SAN RAFFAELE
2018 Phase 1 EUCTR2017-002430-23-IT Italy;
MERCAPTAMINE BITARTRATE
HOSPICES CIVILS DE LYON
2010 - EUCTR2010-020098-18-FR France;
MERCAPTAMINE HYDROCHLORIDE
Fundació Hospital Universitari Vall d'Hebron - Institut de Recerca (VHIR)
2014 - EUCTR2013-003228-35-ES Spain;
MIGALASTAT HYDROCHLORIDE
Amicus Therapeutics UK, Ltd
2020 Phase 3 EUCTR2017-000146-21-IT Australia;Germany;Italy;Spain;United Kingdom;United States;
Amicus Therapeutics, UK Limited
2020 Phase 3 EUCTR2019-000222-21-GB United Kingdom;United States;
Amicus Therapeutics, UK Ltd
2019 Phase 3 EUCTR2017-000146-21-GB Australia;Germany;Italy;Spain;United Kingdom;United States;
2019 Phase 3 EUCTR2017-000146-21-ES Australia;Germany;Italy;Spain;United Kingdom;United States;
- Phase 3 EUCTR2017-000146-21-DE Australia;Germany;Italy;Spain;United Kingdom;United States;
MIGLUSTAT (ZAVESCA)
INSTITUTO ARAGONÉS DE CIENCIAS DE LA SALUD
2008 - EUCTR2007-007164-20-ES Spain;
MOZOBIL - 20 MG/ML - SOLUZIONE INIETTABILE - USO SOTTOCUTANEO - F
OSPEDALE SAN RAFFAELE
2018 Phase 1 EUCTR2017-002430-23-IT Italy;
MPS
Mackay Memorial Hospital
2021 - NCT04958070 Taiwan;
National MPS Society
2011 - NCT01675674 United States;
MRI with contrast agent injection
University Hospital, Bordeaux
2016 - NCT03123523 France;
MRI without contrast agent injection
University Hospital, Bordeaux
2016 - NCT03123523 France;
MYELOSTIM - 34 1 FLACONCINO LIOFILIZZATO + SIRINGA PRERIEMPITA SOLVENTE 1 ML
OSPEDALE SAN RAFFAELE
2018 Phase 1 EUCTR2017-002430-23-IT Italy;
Magnetic Resonance Imaging
University Hospital, Rouen
2017 - NCT02956954 France;
Mechanical stimulation with Von Frey filaments
Universitaire Ziekenhuizen Leuven
2014 Phase 0 NCT02533076 Belgium;
Medication to suppress the immune system
StemCells, Inc.
2006 Phase 1 NCT00337636 United States;
Melphalan
Masonic Cancer Center, University of Minnesota
2012 - NCT01626092 United States;
2006 Phase 2 NCT00383448 United States;
Mepsevii
Ultragenyx Pharmaceutical Inc
2018 - NCT03775174 -
Mercaptamina
Recordati Rare Diseases
2020 Phase 3 EUCTR2018-002984-24-IT Belgium;France;Germany;Italy;United Kingdom;
Mercaptamine
Recordati Rare Diseases
2020 Phase 3 NCT04125927 France;
Mercaptamine hydrochloride
Orphan Europe SARL
2013 - EUCTR2009-012564-13-FR France;
RECORDATI Rare Diseases SARL
2019 Phase 3 EUCTR2018-002984-24-GB France;United Kingdom;
Mercaptamine, mercamine, 2-aminoethanethiol
Orphan Europe SARL
2008 - EUCTR2007-006024-35-FR France;
Metazym
Shire Pharmaceutical Ireland Limited
2008 - EUCTR2007-006345-40-DK Denmark;
Shire Pharmaceuticals Ireland Limited
2008 Phase 2 EUCTR2007-007165-20-DK Denmark;
2006 - EUCTR2006-005341-11-DK Denmark;
Methotrexate
Central Manchester University Hospitals NHS Foundation Trust
2015 Phase 3 EUCTR2015-003031-35-GB United Kingdom;
Methylprednisolone
National Human Genome Research Institute (NHGRI)
2019 Phase 1/Phase 2 NCT03952637 United States;
Prevail Therapeutics
2021 Phase 1/Phase 2 NCT04411654 United Kingdom;United States;
Migalastat
Amicus Therapeutics
2020 - NCT04252066 United States;
Manchester University NHS Foundation Trust
2019 - NCT03949920 United Kingdom;
SANOFI-AVENTIS RECHERCHE E DEVELOPPEMENT
2022 Phase 3 EUCTR2021-002320-20-IT Austria;Canada;China;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Turkey;United Kingdom;United States;
sanofi-aventis recherche et développement
- Phase 3 EUCTR2021-002320-20-ES Austria;Canada;China;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Turkey;United Kingdom;United States;
Migalastat HCl
Amicus Therapeutics
2022 Phase 3 NCT04020055 Belgium;France;Italy;Spain;United Kingdom;United States;
2019 Phase 3 NCT04049760 Spain;United Kingdom;United States;
2018 Phase 3 NCT03500094 Spain;United Kingdom;United States;
2014 Phase 3 NCT02194985 Argentina;Australia;Austria;Belgium;Brazil;Canada;Denmark;Egypt;France;Italy;Japan;Spain;Turkey;United Kingdom;United States;
2011 Phase 2 NCT01196871 Australia;Belgium;Canada;France;Netherlands;United Kingdom;United States;
2011 - NCT01476163 Australia;United States;
2007 Phase 2 NCT00526071 Australia;Brazil;France;United Kingdom;United States;
2006 Phase 2 NCT00304512 Australia;Brazil;Canada;France;United Kingdom;United States;
2006 Phase 2 NCT00283959 Australia;Brazil;
2006 Phase 2 NCT00283933 Canada;France;United Kingdom;
2006 Phase 2 NCT00214500 United States;
Migalastat Hydrochloride
Amicus Therapeutics, Inc.
2014 Phase 3 EUCTR2011-004800-40-AT Argentina;Australia;Austria;Belgium;Brazil;Canada;Denmark;Egypt;France;Italy;Spain;Turkey;United Kingdom;United States;
2013 Phase 3 EUCTR2011-004800-40-BE Argentina;Australia;Austria;Belgium;Brazil;Canada;Denmark;Egypt;France;Italy;Spain;Turkey;United Kingdom;United States;
2012 Phase 3 EUCTR2011-004800-40-GB Argentina;Australia;Austria;Belgium;Brazil;Canada;Denmark;Egypt;France;Italy;Spain;Turkey;United Kingdom;United States;
2012 Phase 3 EUCTR2011-004800-40-DK Argentina;Australia;Austria;Belgium;Brazil;Canada;Denmark;Egypt;France;Italy;Spain;Turkey;United Kingdom;United States;
GLAXOSMITHKLINE RESEARCH & DEVELOPMENT LTD.
2012 - EUCTR2011-004800-40-IT Australia;Austria;Belgium;Brazil;Denmark;Egypt;Italy;Spain;Turkey;United Kingdom;United States;
GlaxoSmithKline Research & Development Limited
2012 Phase 3 EUCTR2011-004800-40-ES Australia;Austria;Belgium;Brazil;Denmark;Egypt;France;Italy;Spain;Turkey;United Kingdom;United States;
Migalastat hydrochloride
AMICUS THERAPEUTICS, INC
2010 - EUCTR2009-013459-31-IT Belgium;Denmark;France;Germany;Italy;Netherlands;Spain;United Kingdom;
Amicus Therapeutics
2011 Phase 3 NCT01458119 Argentina;Australia;Austria;Belgium;Brazil;Canada;Denmark;Egypt;France;Italy;Japan;Spain;Turkey;United Kingdom;United States;
2011 Phase 3 NCT01218659 Argentina;Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;Poland;Switzerland;Taiwan;Turkey;United Kingdom;United States;
2009 Phase 3 NCT00925301 Argentina;Australia;Belgium;Brazil;Canada;Chile;Denmark;Egypt;France;Germany;Israel;Italy;Netherlands;Poland;South Africa;Spain;Turkey;United Kingdom;United States;
2005 Phase 2 EUCTR2005-004384-33-GB United Kingdom;
Amicus Therapeutics, Inc.
2012 - EUCTR2010-022709-16-GB Belgium;United Kingdom;
2012 - EUCTR2010-022636-37-DE Argentina;Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Hungary;Israel;Italy;Japan;Mexico;Poland;Russian Federation;Slovakia;Switzerland;Taiwan;Turkey;United Kingdom;United States;
2011 Phase 3 EUCTR2010-022636-37-GR Argentina;Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Hungary;Israel;Italy;Japan;Mexico;Poland;Russian Federation;Slovakia;Switzerland;Taiwan;Turkey;United Kingdom;United States;
2011 Phase 2 EUCTR2010-022709-16-BE Australia;Belgium;Canada;France;Netherlands;Switzerland;United Kingdom;
2011 - EUCTR2010-022636-37-GB Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;United Kingdom;United States;
2011 - EUCTR2010-022636-37-DK Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;United Kingdom;United States;
2011 - EUCTR2010-022636-37-BE Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;United Kingdom;United States;
2011 - EUCTR2010-022636-37-AT Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;United Kingdom;United States;
2010 Phase 3 EUCTR2009-013459-31-ES Belgium;Denmark;France;Germany;Italy;Netherlands;Spain;United Kingdom;
2010 Phase 3 EUCTR2009-013459-31-BE Belgium;Denmark;France;Germany;Italy;Netherlands;Spain;United Kingdom;
2010 - EUCTR2009-013459-31-NL Belgium;Denmark;France;Germany;Italy;Netherlands;Spain;United Kingdom;
2010 - EUCTR2009-013459-31-DK Argentina;Australia;Belgium;Brazil;Canada;Denmark;Egypt;France;Germany;Italy;Netherlands;South Africa;Spain;Turkey;United Kingdom;United States;
2009 Phase 3 EUCTR2009-013459-31-GB Argentina;Australia;Belgium;Brazil;Canada;Denmark;Egypt;France;Germany;Italy;Netherlands;South Africa;Spain;Turkey;United Kingdom;United States;
2009 Phase 3 EUCTR2009-013459-31-FR Belgium;Denmark;France;Germany;Italy;Netherlands;Spain;United Kingdom;
2009 Phase 2 EUCTR2007-001838-13-GB Australia;Brazil;United Kingdom;United States;
2006 Phase 2 EUCTR2006-000181-36-GB United Kingdom;
- - EUCTR2009-013459-31-DE Belgium;Denmark;France;Germany;Italy;Netherlands;Spain;United Kingdom;
Migalastat hydrocloride
AMICUS THERAPEUTICS, INC
2011 - EUCTR2010-022636-37-IT Argentina;Australia;Austria;Belgium;Brazil;Denmark;Germany;Greece;Italy;Japan;Mexico;Russian Federation;Switzerland;Taiwan;Turkey;United Kingdom;United States;
Miglustat
Actelion
2020 Phase 4 NCT03910621 China;
2006 Phase 3 NCT00319046 Australia;Brazil;Canada;Czech Republic;Czechia;France;Germany;Hungary;Italy;Netherlands;Spain;Taiwan;United Kingdom;United States;
2002 Phase 2 NCT00517153 United States;
Actelion Pharmaceuticals Ltd
2007 - EUCTR2005-001651-37-HU Czech Republic;Germany;Hungary;Italy;Spain;United Kingdom;
2006 - EUCTR2005-001651-37-ES Czech Republic;Germany;Hungary;Italy;Spain;United Kingdom;
2006 - EUCTR2005-001651-37-DE Czech Republic;Germany;Hungary;Italy;Spain;United Kingdom;
2006 - EUCTR2005-001651-37-CZ Czech Republic;Germany;Hungary;Italy;Spain;United Kingdom;
2005 Phase 4 EUCTR2005-001651-37-GB Czech Republic;Germany;Hungary;Italy;Spain;United Kingdom;
Actelion Registration Ltd
2006 - EUCTR2005-001651-37-IT Czech Republic;Germany;Hungary;Italy;Spain;United Kingdom;
Dipartimento di Pediatria Universita` di Napoli Federico II
2006 - EUCTR2006-005842-35-IT Italy;
ISTITUTO GIANNINA GASLINI
2009 - EUCTR2007-005516-61-IT Italy;
National Taiwan University Hospital
2015 - NCT02520934 Taiwan;
2008 Phase 3 NCT01760564 Taiwan;
Tehran University of Medical Sciences
2019 Phase 3 NCT03822013 Iran, Islamic Republic of;
The Hospital for Sick Children
2004 Phase 2 NCT00418847 Canada;
University of Minnesota
2015 Phase 4 NCT02030015 United States;
Moss-aGal (recombinant human alpha-galactosidase A produced in moss)
Greenovation Biotech GmbH
2016 Phase 1 NCT02995993 Germany;
Mycophenolate mofetil
Masonic Cancer Center, University of Minnesota
2012 - NCT01626092 United States;
University of Rochester
2011 Phase 2 NCT01399047 United States;
Mycophenylate mofetil
Masonic Cancer Center, University of Minnesota
2006 Phase 2 NCT00383448 United States;
N Acetyl L leucine
IntraBio Ltd
2022 Phase 3 EUCTR2021-005356-10-SK Australia;Czechia;Germany;Netherlands;Slovakia;Switzerland;United Kingdom;United States;
2019 Phase 2 EUCTR2018-004406-25-ES Germany;Spain;United Kingdom;United States;
N acetyl cysteine
University of Sao Paulo
2011 Phase 4 NCT01614431 -
N-Acetyl Cysteine
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
2009 Phase 1/Phase 2 NCT00975689 United States;
N-Acetyl-L Leucine
IntraBio Ltd
- Phase 3 EUCTR2021-005356-10-NL Australia;Czechia;Germany;Netherlands;Slovakia;Spain;Switzerland;United Kingdom;United States;
N-Acetyl-L-Leucine
IntraBio Inc
2022 Phase 3 NCT05163288 -
IntraBio Ltd
2019 Phase 2 EUCTR2018-004406-25-ES Germany;Spain;United Kingdom;United States;
2019 Phase 2 EUCTR2018-004406-25-DE Germany;Spain;United Kingdom;United States;
2019 Phase 2 EUCTR2018-004331-71-SK Germany;Slovakia;Spain;United Kingdom;United States;
N-acetyl-L-leucine
IntraBio Ltd
2022 Phase 3 EUCTR2021-005356-10-SK Australia;Czechia;Germany;Netherlands;Slovakia;Switzerland;United Kingdom;United States;
- Phase 3 EUCTR2021-005356-10-NL Australia;Czechia;Germany;Netherlands;Slovakia;Spain;Switzerland;United Kingdom;United States;
N-acetylcysteine
University of Minnesota
2015 Phase 2 NCT02583672 United States;
2011 Phase 1 NCT01427517 United States;
N-acetylgalactosamine 4-sulfatase
BioMarin Pharmaceutical
2004 Phase 3 NCT00104234 United States;
2003 Phase 3 NCT00067470 United States;
2002 Phase 2 NCT00048711 United States;
2000 Phase 1 NCT00048620 United States;
Naglazyme
BioMarin Pharmaceutical
2006 Phase 4 NCT00299000 France;Portugal;United States;
BioMarin Pharmaceutical Inc.
2007 Phase 4 EUCTR2005-003512-30-FR France;Portugal;
Naglazyme (galsulfase)
BioMarin Pharmaceutical Inc
2007 - EUCTR2005-003512-30-PT France;Portugal;
Naglazyme®
Masonic Cancer Center, University of Minnesota
2016 - NCT02156674 United States;
Non applicable
Inserm
2012 - EUCTR2011-004410-42-FR France;
OGT 918
National Institute of Neurological Disorders and Stroke (NINDS)
2002 Phase 2 NCT00041535 United Kingdom;United States;
OGT918
Actelion Pharmaceuticals Ltd
2007 - EUCTR2005-001651-37-HU Czech Republic;Germany;Hungary;Italy;Spain;United Kingdom;
2006 - EUCTR2005-001651-37-ES Czech Republic;Germany;Hungary;Italy;Spain;United Kingdom;
2006 - EUCTR2005-001651-37-DE Czech Republic;Germany;Hungary;Italy;Spain;United Kingdom;
2006 - EUCTR2005-001651-37-CZ Czech Republic;Germany;Hungary;Italy;Spain;United Kingdom;
2005 Phase 4 EUCTR2005-001651-37-GB Czech Republic;Germany;Hungary;Italy;Spain;United Kingdom;
National Eye Institute (NEI)
2002 Phase 1 NCT00316498 United Kingdom;United States;
OGT923
IDORSIA PHARMACEUTICALS LTD
2020 Phase 3 EUCTR2018-002210-12-IT Australia;Austria;Belgium;Canada;France;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;United Kingdom;United States;
2020 Phase 3 EUCTR2017-003369-85-IT Australia;Austria;Belgium;Canada;Finland;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;Switzerland;United Kingdom;United States;
Idorsia Pharmaceuticals Ltd
2021 Phase 3 EUCTR2018-002210-12-NO Australia;Austria;Belgium;Canada;France;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;United Kingdom;United States;
2021 Phase 3 EUCTR2018-002210-12-FR Austria;Belgium;Canada;Czech Republic;France;Germany;Netherlands;Poland;United Kingdom;United States;
2020 Phase 3 EUCTR2018-002210-12-BE Australia;Austria;Belgium;Canada;France;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;United Kingdom;United States;
2020 Phase 3 EUCTR2017-003369-85-NO Australia;Austria;Belgium;Canada;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;Switzerland;United Kingdom;United States;
2020 Phase 3 EUCTR2017-003369-85-IE Australia;Austria;Belgium;Canada;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;Switzerland;United Kingdom;United States;
2020 Phase 3 EUCTR2017-003369-85-ES Australia;Austria;Belgium;Canada;Germany;Ireland;Italy;Netherlands;Norway;Poland;Russian Federation;Spain;Switzerland;United Kingdom;United States;
2019 Phase 3 EUCTR2018-002210-12-NL Australia;Austria;Belgium;Canada;France;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;United Kingdom;United States;
2019 Phase 3 EUCTR2018-002210-12-GB Australia;Austria;Belgium;Canada;Germany;Ireland;Netherlands;Poland;United Kingdom;United States;
2019 Phase 3 EUCTR2018-002210-12-DE Australia;Austria;Belgium;Canada;France;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;United Kingdom;United States;
2019 Phase 3 EUCTR2018-002210-12-AT Australia;Austria;Belgium;Canada;France;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;United Kingdom;United States;
2018 Phase 3 EUCTR2017-003369-85-NL Australia;Austria;Belgium;Canada;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;Switzerland;United Kingdom;United States;
2018 Phase 3 EUCTR2017-003369-85-GB Australia;Austria;Belgium;Canada;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;Switzerland;United Kingdom;United States;
2018 Phase 3 EUCTR2017-003369-85-AT Australia;Austria;Belgium;Canada;Germany;Ireland;Italy;Netherlands;Norway;Poland;Spain;Switzerland;United Kingdom;United States;
OSOME
Université de Montréal
2014 - NCT02023086 Canada;
OTL-200 Dispersion for Infusion
Orchard Therapeutics (Europe) Limited
2019 Phase 3 EUCTR2019-002636-82-IT Italy;
Orchard Therapeutics (Europe) Ltd
2017 Phase 3 EUCTR2017-001730-26-IT Italy;
Odiparcil
Inventiva Pharma
2017 Phase 2 NCT03370653 France;Germany;Portugal;United Kingdom;
Inventiva S.A.
2019 Phase 2 EUCTR2017-002158-35-PT France;Germany;Portugal;United Kingdom;
2018 Phase 2 EUCTR2017-002158-35-DE France;Germany;Portugal;United Kingdom;
2017 Phase 2 EUCTR2017-002158-35-GB France;Germany;Portugal;United Kingdom;
- Phase 2 EUCTR2017-002158-35-FR France;Germany;Portugal;United Kingdom;
Olenasufligene relduparvovec
Lysogene SA
2019 Phase 2;Phase 3 EUCTR2018-000195-15-NL France;Germany;Netherlands;United Kingdom;United States;
2018 Phase 2;Phase 3 EUCTR2018-000195-15-FR France;Germany;Netherlands;United Kingdom;United States;
Olipudase alfa (rhASM)
Genzyme Corporation
2015 Phase 2 EUCTR2014-003198-40-GB Brazil;France;Germany;Italy;United Kingdom;United States;
2015 Phase 2 EUCTR2014-003198-40-DE Brazil;France;Germany;Italy;United Kingdom;United States;
Ophthalmology exam
National Human Genome Research Institute (NHGRI)
2019 Phase 1/Phase 2 NCT03952637 United States;
Oral migalastat HCl
Amicus Therapeutics
2014 Phase 1 NCT02082327 Netherlands;
Osteopetrosis Haploidentical Only Preparative Regimen
Masonic Cancer Center, University of Minnesota
2014 Phase 2 NCT02171104 United States;
Osteopetrosis Only Preparative Regimen
Masonic Cancer Center, University of Minnesota
2014 Phase 2 NCT02171104 United States;
Oxygen flow at the optic nerve head measurement
Université de Montréal
2014 - NCT02023086 Canada;
PBGM01
Passage Bio, Inc.
2021 Phase 1/Phase 2 NCT04713475 Brazil;Canada;Turkey;United Kingdom;United States;
PBKR03
Passage Bio, Inc.
2022 Phase 1/Phase 2 NCT04771416 Brazil;Canada;Israel;Netherlands;United Kingdom;United States;
- Phase 1;Phase 2 EUCTR2020-005229-95-NL Brazil;Canada;Israel;Netherlands;United Kingdom;United States;
PENTOSAN POLYSULFATE SODIUM
Multiplex Pharma Holdings LLC
2014 - EUCTR2014-000350-11-DE Germany;
PLX-200
Polaryx Therapeutics, Inc.
2021 Phase 3 NCT04637282 -
PR001
Prevail Therapeutics
2021 Phase 1/Phase 2 NCT04411654 United Kingdom;United States;
PRX-102
PROTALIX LTD
2020 Phase 3 EUCTR2018-001148-67-IT Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Finland;Hungary;Italy;Netherlands;Norway;Slovenia;Spain;Switzerland;Turkey;United Kingdom;United States;
2019 Phase 3 EUCTR2018-001947-30-IT Australia;Belgium;Canada;Czechia;Denmark;Finland;France;Italy;Netherlands;Norway;Spain;Sweden;Taiwan;Turkey;United Kingdom;United States;
2018 Phase 3 EUCTR2017-001528-23-IT Austria;Belgium;Canada;Czech Republic;Czechia;Denmark;Germany;Italy;Netherlands;Norway;Spain;Taiwan;Turkey;United Kingdom;United States;
2017 Phase 3 EUCTR2016-000378-38-IT Australia;Belgium;Canada;Czech Republic;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Turkey;United Kingdom;United States;
Protalix
2017 Phase 3 NCT02921620 -
2013 Phase 1/Phase 2 NCT01769001 Australia;Paraguay;Spain;United Kingdom;United States;
2012 Phase 1/Phase 2 NCT01678898 Australia;Paraguay;Serbia;Spain;United Kingdom;United States;
Protalix Ltd.
2021 Phase 3 EUCTR2018-001148-67-FI Australia;Canada;Czech Republic;Czechia;Finland;France;Hungary;Italy;Netherlands;Norway;Slovenia;Spain;United Kingdom;United States;
2020 Phase 3 EUCTR2018-001148-67-SI Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Hungary;Italy;Netherlands;Norway;Slovenia;Spain;Switzerland;Turkey;United Kingdom;United States;
2019 Phase 3 EUCTR2018-001947-30-GB Australia;Belgium;Canada;Czech Republic;Denmark;Italy;Netherlands;Norway;Spain;Taiwan;Turkey;United Kingdom;United States;
2019 Phase 3 EUCTR2018-001947-30-DK Belgium;Czechia;Denmark;Italy;Norway;United Kingdom;United States;
2019 Phase 3 EUCTR2018-001947-30-BE Belgium;Czechia;Denmark;Italy;Norway;United Kingdom;United States;
2019 Phase 3 EUCTR2018-001148-67-NL Australia;Canada;Czech Republic;Czechia;Finland;France;Hungary;Italy;Netherlands;Norway;Slovenia;Spain;United Kingdom;United States;
2019 Phase 3 EUCTR2018-001148-67-HU Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Hungary;Italy;Netherlands;Norway;Slovenia;Spain;Switzerland;Turkey;United Kingdom;United States;
2019 Phase 3 EUCTR2018-001148-67-ES Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Denmark;Germany;Hungary;Italy;Netherlands;Norway;Slovenia;Spain;Switzerland;Turkey;United Kingdom;United States;
2019 Phase 3 EUCTR2016-000378-38-FR Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Denmark;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Switzerland;Turkey;United Kingdom;United States;
2019 Phase 3 EUCTR2016-000378-38-FI Argentina;Australia;Belgium;Brazil;Canada;Czechia;Denmark;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Slovenia;Spain;Sweden;Switzerland;Turkey;United Kingdom;United States;
2018 Phase 3 EUCTR2018-001148-67-GB Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Denmark;Germany;Hungary;Italy;Netherlands;Norway;Slovenia;Spain;Switzerland;Turkey;United Kingdom;United States;
2018 Phase 3 EUCTR2018-001148-67-CZ Australia;Canada;Czech Republic;Czechia;Finland;France;Hungary;Italy;Netherlands;Norway;Slovenia;Spain;United Kingdom;United States;
2018 Phase 3 EUCTR2017-001528-23-DK Austria;Belgium;Canada;Czech Republic;Denmark;Germany;Netherlands;Norway;Spain;Taiwan;Turkey;United Kingdom;United States;
2018 Phase 3 EUCTR2017-001528-23-CZ Austria;Belgium;Canada;Czech Republic;Denmark;Germany;Netherlands;Norway;Spain;Taiwan;Turkey;United Kingdom;United States;
2018 Phase 3 EUCTR2016-001318-11-NL Australia;Canada;Czech Republic;Germany;Netherlands;Norway;Slovenia;Spain;United Kingdom;
2017 Phase 3 EUCTR2017-001528-23-GB Belgium;Canada;Czech Republic;Denmark;Germany;Netherlands;Spain;Turkey;United Kingdom;United States;
2017 Phase 3 EUCTR2017-001528-23-BE Belgium;Canada;Czech Republic;Denmark;Germany;Netherlands;Spain;Turkey;United Kingdom;United States;
2017 Phase 3 EUCTR2016-001318-11-SI Australia;Canada;Czech Republic;Germany;Netherlands;Norway;Slovenia;Spain;United Kingdom;
2017 Phase 3 EUCTR2016-001318-11-GB Australia;Canada;Czech Republic;Germany;Netherlands;Norway;Slovenia;Spain;United Kingdom;
2017 Phase 3 EUCTR2016-001318-11-ES Australia;Canada;Germany;Netherlands;Norway;Spain;United Kingdom;
2017 Phase 3 EUCTR2016-001318-11-DE Australia;Canada;Czech Republic;Germany;Netherlands;Norway;Slovenia;Spain;United Kingdom;
2017 Phase 3 EUCTR2016-001318-11-CZ Australia;Canada;Czech Republic;Germany;Netherlands;Norway;Slovenia;Spain;United Kingdom;
2017 Phase 3 EUCTR2016-000378-38-SI Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Denmark;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Switzerland;Turkey;United Kingdom;United States;
2017 Phase 3 EUCTR2016-000378-38-NL Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Turkey;United Kingdom;United States;
2016 Phase 3 EUCTR2016-000378-38-HU Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Turkey;United Kingdom;United States;
2016 Phase 3 EUCTR2016-000378-38-GB Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Germany;Hungary;Netherlands;Norway;Paraguay;Slovenia;Spain;Turkey;United Kingdom;United States;
2016 Phase 3 EUCTR2016-000378-38-ES Australia;Belgium;Canada;Czech Republic;Germany;Hungary;Netherlands;Norway;Paraguay;Spain;Turkey;United Kingdom;United States;
2016 Phase 3 EUCTR2016-000378-38-BE Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Turkey;United Kingdom;United States;
2015 Phase 1;Phase 2 EUCTR2014-005544-18-GB Paraguay;Spain;United Kingdom;United States;
2013 Phase 1;Phase 2 EUCTR2013-002554-78-GB Argentina;Australia;Chile;Israel;Paraguay;Spain;United Kingdom;United States;
2013 - EUCTR2012-004786-40-GB Argentina;Australia;Chile;Israel;Paraguay;Spain;United Kingdom;United States;
PRX-102 (pegunigalsidase alfa)
Protalix
2017 Phase 3 NCT03018730 Australia;Canada;Czech Republic;Czechia;Germany;Netherlands;Norway;Slovenia;Spain;United Kingdom;
2016 Phase 3 NCT02795676 Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Czechia;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Switzerland;Turkey;United Kingdom;United States;
2013 Phase 1/Phase 2 NCT01981720 Paraguay;Spain;United Kingdom;United States;
PRX-112
Protalix
2014 Phase 2 NCT02107846 Israel;
2013 Phase 1 NCT01747980 Israel;
PRX102
Protalix Ltd.
2015 - EUCTR2014-005544-18-ES Paraguay;Spain;United Kingdom;United States;
2013 Phase 1;Phase 2 EUCTR2013-002554-78-ES Australia;Israel;Paraguay;Serbia;Spain;United Kingdom;United States;
2013 - EUCTR2012-004786-40-ES Argentina;Australia;Israel;Paraguay;Serbia;Spain;United Kingdom;United States;
PTC124
PTC Therapeutics Inc.
2015 Phase 2 EUCTR2015-003105-41-GB United Kingdom;
2015 Phase 2 EUCTR2014-002596-28-GB Germany;United Kingdom;
Pabinafusp alfa
JCR Pharmaceuticals Co., Ltd.
- Phase 3 EUCTR2020-003200-14-FR Brazil;France;Germany;United Kingdom;United States;
- Phase 3 EUCTR2020-003200-14-DE Brazil;France;Germany;United Kingdom;United States;
Yamamoto Tatsuyoshi
2021 Phase 4 JPRN-jRCT2031210126 Japan;
Paricalcitol
Federico II University
2012 - NCT02090608 Italy;
Parts A/B: Adrabetadex
Mandos LLC
2015 Phase 2/Phase 3 NCT02534844 Australia;France;Germany;New Zealand;Singapore;Spain;Turkey;United Kingdom;United States;
Pegunigalsidase alfa
PROTALIX LTD
2018 Phase 3 EUCTR2017-001528-23-IT Austria;Belgium;Canada;Czech Republic;Czechia;Denmark;Germany;Italy;Netherlands;Norway;Spain;Taiwan;Turkey;United Kingdom;United States;
2017 Phase 3 EUCTR2016-000378-38-IT Australia;Belgium;Canada;Czech Republic;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Turkey;United Kingdom;United States;
Protalix
2018 Phase 3 NCT03614234 Belgium;Czechia;Denmark;Italy;Norway;United Kingdom;United States;
2018 Phase 3 NCT03566017 Australia;Canada;Czechia;Finland;France;Hungary;Italy;Netherlands;Norway;Slovenia;Spain;United Kingdom;United States;
2017 Phase 3 NCT03180840 Belgium;Canada;Czechia;Denmark;Italy;Netherlands;Norway;Spain;Taiwan;Turkey;United Kingdom;United States;
Protalix Ltd.
2019 Phase 3 EUCTR2016-000378-38-FR Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Denmark;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Switzerland;Turkey;United Kingdom;United States;
2019 Phase 3 EUCTR2016-000378-38-FI Argentina;Australia;Belgium;Brazil;Canada;Czechia;Denmark;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Slovenia;Spain;Sweden;Switzerland;Turkey;United Kingdom;United States;
2018 Phase 3 EUCTR2017-001528-23-DK Austria;Belgium;Canada;Czech Republic;Denmark;Germany;Netherlands;Norway;Spain;Taiwan;Turkey;United Kingdom;United States;
2018 Phase 3 EUCTR2017-001528-23-CZ Austria;Belgium;Canada;Czech Republic;Denmark;Germany;Netherlands;Norway;Spain;Taiwan;Turkey;United Kingdom;United States;
2018 Phase 3 EUCTR2016-001318-11-NL Australia;Canada;Czech Republic;Germany;Netherlands;Norway;Slovenia;Spain;United Kingdom;
2017 Phase 3 EUCTR2017-001528-23-GB Belgium;Canada;Czech Republic;Denmark;Germany;Netherlands;Spain;Turkey;United Kingdom;United States;
2017 Phase 3 EUCTR2017-001528-23-BE Belgium;Canada;Czech Republic;Denmark;Germany;Netherlands;Spain;Turkey;United Kingdom;United States;
2017 Phase 3 EUCTR2016-001318-11-SI Australia;Canada;Czech Republic;Germany;Netherlands;Norway;Slovenia;Spain;United Kingdom;
2017 Phase 3 EUCTR2016-001318-11-GB Australia;Canada;Czech Republic;Germany;Netherlands;Norway;Slovenia;Spain;United Kingdom;
2017 Phase 3 EUCTR2016-001318-11-ES Australia;Canada;Germany;Netherlands;Norway;Spain;United Kingdom;
2017 Phase 3 EUCTR2016-001318-11-DE Australia;Canada;Czech Republic;Germany;Netherlands;Norway;Slovenia;Spain;United Kingdom;
2017 Phase 3 EUCTR2016-001318-11-CZ Australia;Canada;Czech Republic;Germany;Netherlands;Norway;Slovenia;Spain;United Kingdom;
2017 Phase 3 EUCTR2016-000378-38-SI Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Denmark;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Switzerland;Turkey;United Kingdom;United States;
2017 Phase 3 EUCTR2016-000378-38-NL Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Turkey;United Kingdom;United States;
2016 Phase 3 EUCTR2016-000378-38-HU Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Turkey;United Kingdom;United States;
2016 Phase 3 EUCTR2016-000378-38-GB Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Germany;Hungary;Netherlands;Norway;Paraguay;Slovenia;Spain;Turkey;United Kingdom;United States;
2016 Phase 3 EUCTR2016-000378-38-ES Australia;Belgium;Canada;Czech Republic;Germany;Hungary;Netherlands;Norway;Paraguay;Spain;Turkey;United Kingdom;United States;
2016 Phase 3 EUCTR2016-000378-38-BE Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Turkey;United Kingdom;United States;
Pegunigalsidase alpha
PROTALIX LTD
2017 Phase 3 EUCTR2016-000378-38-IT Australia;Belgium;Canada;Czech Republic;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Turkey;United Kingdom;United States;
Protalix Ltd.
2019 Phase 3 EUCTR2016-000378-38-FR Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Denmark;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Switzerland;Turkey;United Kingdom;United States;
2019 Phase 3 EUCTR2016-000378-38-FI Argentina;Australia;Belgium;Brazil;Canada;Czechia;Denmark;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Slovenia;Spain;Sweden;Switzerland;Turkey;United Kingdom;United States;
2018 Phase 3 EUCTR2016-001318-11-NL Australia;Canada;Czech Republic;Germany;Netherlands;Norway;Slovenia;Spain;United Kingdom;
2017 Phase 3 EUCTR2016-001318-11-SI Australia;Canada;Czech Republic;Germany;Netherlands;Norway;Slovenia;Spain;United Kingdom;
2017 Phase 3 EUCTR2016-001318-11-GB Australia;Canada;Czech Republic;Germany;Netherlands;Norway;Slovenia;Spain;United Kingdom;
2017 Phase 3 EUCTR2016-001318-11-ES Australia;Canada;Germany;Netherlands;Norway;Spain;United Kingdom;
2017 Phase 3 EUCTR2016-001318-11-DE Australia;Canada;Czech Republic;Germany;Netherlands;Norway;Slovenia;Spain;United Kingdom;
2017 Phase 3 EUCTR2016-001318-11-CZ Australia;Canada;Czech Republic;Germany;Netherlands;Norway;Slovenia;Spain;United Kingdom;
2017 Phase 3 EUCTR2016-000378-38-SI Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Denmark;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Switzerland;Turkey;United Kingdom;United States;
2017 Phase 3 EUCTR2016-000378-38-NL Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Turkey;United Kingdom;United States;
2016 Phase 3 EUCTR2016-000378-38-HU Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Turkey;United Kingdom;United States;
2016 Phase 3 EUCTR2016-000378-38-GB Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Germany;Hungary;Netherlands;Norway;Paraguay;Slovenia;Spain;Turkey;United Kingdom;United States;
2016 Phase 3 EUCTR2016-000378-38-ES Australia;Belgium;Canada;Czech Republic;Germany;Hungary;Netherlands;Norway;Paraguay;Spain;Turkey;United Kingdom;United States;
2016 Phase 3 EUCTR2016-000378-38-BE Argentina;Australia;Belgium;Brazil;Canada;Czech Republic;Finland;France;Germany;Hungary;Italy;Netherlands;Norway;Paraguay;Slovenia;Spain;Turkey;United Kingdom;United States;
Pending
Ultragenyx Pharmaceutical Inc.
2016 Phase 3 EUCTR2015-001875-32-PT Brazil;Mexico;Portugal;United States;
- - EUCTR2015-001875-32-Outside-EU/EEA United States;
- - EUCTR2015-000104-26-Outside-EU/EEA United States;
Pentosan Polysulfat SP 54® injection solution
Multiplex Pharma Holdings LLC
2014 - EUCTR2014-000350-11-DE Germany;
Pentosan Polysulphate SP54®
Multiplex Pharma Holdings LLC
2014 - EUCTR2014-000350-11-DE Germany;
Perdix
Salford Royal NHS Foundation Trust
2009 Phase 4 EUCTR2007-007482-21-GB United Kingdom;
Plant Cell Recombinant Glucocerebrosidase
PROTALIX BIOTHERAPEUTICS
2009 - EUCTR2008-005826-35-IT Italy;Spain;United Kingdom;
2009 - EUCTR2008-005825-12-IT Germany;Italy;Spain;United Kingdom;
2008 - EUCTR2007-000498-42-IT Italy;Spain;United Kingdom;
Plant cell expressed recombinant glucocerebrosidase (prGCD)
Pfizer
2007 Phase 3 NCT00376168 Canada;Chile;Israel;Italy;South Africa;Spain;United Kingdom;United States;
2006 - NCT00962260 Israel;United States;
Plasma Lyso-GB3
Chang Gung Memorial Hospital
2018 - NCT05056636 Taiwan;
PrGCD
PROTALIX BIOTHERAPEUTICS
2009 - EUCTR2008-005826-35-IT Italy;Spain;United Kingdom;
2009 - EUCTR2008-005825-12-IT Germany;Italy;Spain;United Kingdom;
2008 - EUCTR2007-000498-42-IT Italy;Spain;United Kingdom;
Protalix Biotherapeutics
2012 - EUCTR2011-003685-32-ES Canada;Chile;Israel;Italy;Mexico;Serbia;South Africa;Spain;
2011 - EUCTR2011-003676-37-ES Israel;South Africa;Spain;
2009 - EUCTR2008-005826-35-ES Italy;Spain;United Kingdom;
2009 - EUCTR2008-005825-12-ES Germany;Italy;Spain;United Kingdom;
2008 Phase 3 EUCTR2007-000498-42-GB Italy;Spain;United Kingdom;
2008 - EUCTR2007-000498-42-ES Italy;Spain;United Kingdom;
- - EUCTR2008-005826-35-Outside-EU/EEA Australia;Canada;Chile;Israel;South Africa;Spain;United Kingdom;United States;
- - EUCTR2008-005825-12-Outside-EU/EEA Canada;Israel;United Kingdom;United States;
- - EUCTR2007-000498-42-Outside-EU/EEA Israel;United States;
Prednisone
National Human Genome Research Institute (NHGRI)
2019 Phase 1/Phase 2 NCT03952637 United States;
Prevail Therapeutics
2021 Phase 1/Phase 2 NCT04411654 United Kingdom;United States;
Prevenar13
Maastricht University
2016 Phase 4 EUCTR2015-004846-25-BE Belgium;
Pyrimethamine
Exsar Corporation
2008 Phase 1 NCT00679744 Canada;United States;
The Hospital for Sick Children
2009 Phase 1/Phase 2 NCT01102686 Canada;
Quantitative chemical shift imaging (QCSI)
Shaare Zedek Medical Center
2017 - NCT04353466 -
RAAV 2/6 hIDS DONOR
Sangamo Therapeutics, Inc.
2018 Phase 1;Phase 2 EUCTR2018-000192-33-GB United Kingdom;United States;
RAAV2/5-hNAGLU
UniQure Biopharma B.V.
2013 Phase 1/Phase 2 NCT03300453 France;
RAAV2/5-hNaGlu
Institut Pasteur
- Phase 1;Phase 2 EUCTR2012-000856-33-FR France;
RAAV2/6 Left ZFN Vector
Sangamo Therapeutics, Inc.
2018 Phase 1;Phase 2 EUCTR2018-000206-28-GB United Kingdom;United States;
2018 Phase 1;Phase 2 EUCTR2018-000192-33-GB United Kingdom;United States;
RAAV2/6 Right ZFN Vector
Sangamo Therapeutics, Inc.
2018 Phase 1;Phase 2 EUCTR2018-000206-28-GB United Kingdom;United States;
2018 Phase 1;Phase 2 EUCTR2018-000192-33-GB United Kingdom;United States;
RAAV9.CMV.hNAGLU
Abeona Therapeutics Europe SL.
2021 Phase 1;Phase 2 EUCTR2019-002936-97-FR France;Germany;United States;
2019 Phase 1;Phase 2 EUCTR2014-001411-39-GB France;Germany;Italy;Netherlands;Spain;United Kingdom;United States;
2019 Phase 1;Phase 2 EUCTR2014-001411-39-DE France;Germany;Spain;United Kingdom;United States;
- Phase 1;Phase 2 EUCTR2019-002936-97-DE France;Germany;Spain;United States;
Abeona Therapeutics Inc
2018 Phase 1;Phase 2 EUCTR2014-001411-39-ES France;Germany;Italy;Netherlands;Spain;United Kingdom;United States;
RECOMBINANT HUMAN ACID SPHINGOMYELINASE (rhASM)
Genzyme Corporation
2015 Phase 2 EUCTR2014-003198-40-IT Brazil;Chile;France;Germany;Italy;United Kingdom;United States;
2015 Phase 2 EUCTR2014-003198-40-GB Brazil;France;Germany;Italy;United Kingdom;United States;
2015 Phase 2 EUCTR2014-003198-40-DE Brazil;France;Germany;Italy;United Kingdom;United States;
- Phase 2 EUCTR2014-003198-40-FR Brazil;Chile;France;Germany;Italy;United Kingdom;United States;
RECOMBINANT HUMAN ARYLSULFATASE A
Shire Human Genetic Therapies, Inc.
2021 Phase 2 EUCTR2018-003291-12-GR Argentina;Belgium;Brazil;Canada;France;Germany;Greece;Israel;Italy;Japan;Netherlands;Spain;United Kingdom;United States;
2020 Phase 2 EUCTR2018-003291-12-DE Belgium;Brazil;Canada;France;Germany;Israel;Japan;Mexico;Netherlands;Spain;United Kingdom;United States;
2019 Phase 2 EUCTR2018-003291-12-NL Argentina;Belgium;Brazil;Canada;France;Germany;Greece;Israel;Italy;Japan;Netherlands;Spain;United Kingdom;United States;
2019 Phase 2 EUCTR2018-003291-12-GB Belgium;Brazil;Canada;France;Germany;Israel;Japan;Mexico;Netherlands;Spain;United Kingdom;United States;
2019 Phase 2 EUCTR2018-003291-12-FR Belgium;Brazil;Canada;Denmark;France;Germany;Israel;Japan;Mexico;Netherlands;Spain;United Kingdom;United States;
2019 Phase 2 EUCTR2018-003291-12-ES Belgium;Brazil;Canada;Denmark;France;Germany;Israel;Japan;Mexico;Netherlands;Spain;United Kingdom;United States;
2019 Phase 2 EUCTR2018-003291-12-BE Belgium;Brazil;Canada;France;Germany;Israel;Japan;Mexico;Netherlands;Spain;United Kingdom;United States;
RECOMBINANT HUMAN BETA GLUCURONIDASE
Ultragenyx Pharmaceutical Inc.
2016 Phase 3 EUCTR2015-001875-32-PT Brazil;Mexico;Portugal;United States;
2016 Phase 2 EUCTR2015-000104-26-PT Portugal;Spain;United States;
2016 Phase 2 EUCTR2015-000104-26-ES Portugal;Spain;United States;
2014 Phase 1;Phase 2 EUCTR2013-001152-35-ES Spain;United Kingdom;
2013 Phase 1;Phase 2 EUCTR2013-001152-35-GB Spain;United Kingdom;
- - EUCTR2015-001875-32-Outside-EU/EEA United States;
- - EUCTR2015-000104-26-Outside-EU/EEA United States;
- - EUCTR2014-005638-71-Outside-EU/EEA United States;
RECOMBINANT HUMAN TRIPEPTIDYL PEPTIDASE-1 (RHTPP1)
BioMarin Pharmaceutical Inc.
2015 Phase 2 EUCTR2014-003480-37-GB Germany;Italy;United Kingdom;United States;
2015 Phase 2 EUCTR2014-003480-37-DE Germany;Italy;United Kingdom;United States;
2015 - EUCTR2014-003480-37-IT Germany;Italy;Japan;United Kingdom;United States;
REPLAGAL ®
Shire Human Genetic Therapies, Inc.
2021 Phase 3 EUCTR2018-004689-32-SE Argentina;Australia;Belgium;Bosnia and Herzegovina;Brazil;Canada;Croatia;Finland;Germany;Greece;Hungary;Italy;Latvia;Poland;Portugal;Romania;Slovenia;Spain;Sweden;Ukraine;
2021 Phase 3 EUCTR2018-004689-32-PT Argentina;Australia;Belgium;Bosnia and Herzegovina;Brazil;Canada;Croatia;Finland;Germany;Greece;Hungary;Italy;Latvia;Poland;Portugal;Romania;Slovenia;Spain;Sweden;Ukraine;
2021 Phase 3 EUCTR2018-004689-32-PL Argentina;Australia;Belgium;Bosnia and Herzegovina;Brazil;Canada;Croatia;Finland;Germany;Greece;Hungary;Italy;Latvia;Poland;Portugal;Romania;Slovenia;Spain;Sweden;Ukraine;
2021 Phase 3 EUCTR2018-004689-32-GR Argentina;Australia;Belgium;Bosnia and Herzegovina;Brazil;Canada;Croatia;Finland;Germany;Greece;Hungary;Italy;Latvia;Poland;Portugal;Romania;Slovenia;Spain;Sweden;Ukraine;
2021 Phase 3 EUCTR2018-004689-32-FI Argentina;Australia;Belgium;Bosnia and Herzegovina;Brazil;Canada;Croatia;Finland;Germany;Greece;Hungary;Italy;Latvia;Poland;Portugal;Romania;Slovenia;Spain;Sweden;Ukraine;
RHGUS
Ultragenyx Pharmaceutical Inc.
2016 Phase 2 EUCTR2015-000104-26-PT Portugal;Spain;United States;
2016 Phase 2 EUCTR2015-000104-26-ES Portugal;Spain;United States;
2014 Phase 1;Phase 2 EUCTR2013-001152-35-ES Spain;United Kingdom;
2013 Phase 1;Phase 2 EUCTR2013-001152-35-GB Spain;United Kingdom;
- - EUCTR2015-001875-32-Outside-EU/EEA United States;
- - EUCTR2015-000104-26-Outside-EU/EEA United States;
- - EUCTR2014-005638-71-Outside-EU/EEA United States;
RHNAGLU-IGF2
Allievex Corporation
2018 Phase 2 EUCTR2017-003083-13-GB Colombia;Germany;Spain;Taiwan;Turkey;United Kingdom;United States;
2016 Phase 1;Phase 2 EUCTR2015-001985-25-DE Australia;Colombia;Germany;Spain;Taiwan;Turkey;United Kingdom;
2015 Phase 1;Phase 2 EUCTR2015-001985-25-GB Australia;Colombia;Germany;Spain;Taiwan;Turkey;United Kingdom;
- Phase 2 EUCTR2017-003083-13-DE Colombia;Germany;Spain;Taiwan;Turkey;United Kingdom;United States;
BioMarin Pharmaceutical Inc.
2017 Phase 2 EUCTR2017-003083-13-ES Argentina;Australia;Colombia;Germany;Spain;Taiwan;Turkey;United Kingdom;United States;
2016 Phase 1;Phase 2 EUCTR2015-001985-25-ES Australia;Brazil;Colombia;Germany;Spain;Taiwan;Turkey;United Kingdom;
RP 103
HOSPICES CIVILS DE LYON
2010 - EUCTR2010-020098-18-FR France;
RP103
Horizon Pharma USA, Inc.
2014 Phase 3 EUCTR2012-002773-64-GB Belgium;France;Germany;Italy;Netherlands;United Kingdom;United States;
2013 Phase 3 NCT01733316 Belgium;France;Italy;Netherlands;United Kingdom;United States;
2013 Phase 3 EUCTR2012-002773-64-NL Belgium;France;Germany;Italy;Netherlands;United Kingdom;United States;
2012 Phase 3 NCT01744782 Brazil;United States;
2010 Phase 3 EUCTR2010-018365-34-NL France;Netherlands;United States;
2009 Phase 1/Phase 2 NCT00872729 United States;
Raptor Therapeutics Inc.
2014 Phase 3 EUCTR2012-002773-64-IT Belgium;France;Germany;Italy;Netherlands;United Kingdom;United States;
2014 Phase 3 EUCTR2012-002773-64-BE Belgium;France;Germany;Italy;Netherlands;United Kingdom;United States;
2013 Phase 3 EUCTR2012-002773-64-FR Belgium;France;Italy;Netherlands;United Kingdom;United States;
2010 Phase 3 EUCTR2009-017882-42-FR France;Netherlands;
2010 - EUCTR2009-017882-42-NL France;Netherlands;
- Phase 3 EUCTR2010-018365-34-FR France;Netherlands;
RVX000222
Resverlogix Corp
2019 Phase 1/Phase 2 NCT03228940 Canada;
Rebisufligene etisparvovec
Abeona Therapeutics Inc
2020 Phase 1;Phase 2 EUCTR2019-002979-34-ES Australia;France;Germany;Spain;United States;
2019 Phase 1;Phase 2 EUCTR2015-003904-21-FR Australia;Brazil;France;Germany;Spain;United Kingdom;United States;
Recombinant Adeno-associated virus 2/6 vector encoding the cDNA for human alpha galactosidase A
Sangamo Therapeutics, Inc.
2019 Phase 1;Phase 2 EUCTR2019-000667-24-GB United Kingdom;United States;
Recombinant Human Alpha-N-Acetylglucosaminidase (rhNAGLU)
Alexion Pharmaceuticals, Inc
2015 Phase 2 EUCTR2015-001983-20-GB United Kingdom;
Alexion Pharmaceuticals, Inc.
2016 Phase 2 EUCTR2015-000753-20-NL Netherlands;
2016 Phase 1 EUCTR2013-003400-39-ES Spain;United Kingdom;United States;
2014 Phase 1;Phase 2 EUCTR2013-003400-39-GB Spain;United Kingdom;United States;
Recombinant Human Arylsulfatase A (rhASA)
SHIRE HUMAN GENETIC THERAPIES, INC
2020 Phase 2 EUCTR2018-003291-12-IT Belgium;Brazil;Canada;France;Germany;Israel;Italy;Japan;Netherlands;Spain;United Kingdom;United States;
Shire Human Genetics Therapies Inc
2017 Phase 1;Phase 2 EUCTR2012-003775-20-IT Australia;Brazil;Czech Republic;Denmark;France;Germany;Italy;Japan;United Kingdom;
2016 Phase 1;Phase 2 EUCTR2012-003775-20-GB Australia;Brazil;Czech Republic;Denmark;France;Germany;Italy;Japan;United Kingdom;
2014 Phase 1;Phase 2 EUCTR2012-003775-20-CZ Australia;Brazil;Czech Republic;Denmark;France;Germany;Italy;Japan;United Kingdom;
2013 Phase 1;Phase 2 EUCTR2012-003775-20-DK Australia;Brazil;Czech Republic;Czechia;Denmark;France;Germany;Italy;Japan;United Kingdom;
2013 Phase 1;Phase 2 EUCTR2012-003775-20-DE Australia;Brazil;Czech Republic;Denmark;France;Germany;Italy;Japan;United Kingdom;
- Phase 1;Phase 2 EUCTR2012-003775-20-FR Argentina;Australia;Brazil;Czech Republic;Denmark;France;Germany;United Kingdom;
Recombinant Human Arylsulfatase A (rhASA,
Shire Human Genetics Therapies Inc
2012 Phase 1;Phase 2 EUCTR2011-002044-28-DE Australia;Brazil;Denmark;France;Germany;Japan;
2011 Phase 1;Phase 2 EUCTR2011-002044-28-DK Australia;Brazil;Denmark;France;Germany;Japan;
Recombinant Human Glucocerebrosidase (prGCD)
Protalix Biotherapeutics
2009 - EUCTR2008-005825-12-DE Germany;Italy;Spain;United Kingdom;
Recombinant alpha-galactosidase A
Assistance Publique - Hôpitaux de Paris
2005 Phase 4 NCT00487630 France;
Recombinant human Arylsulfatase A (rhASA)
Shire
2007 Phase 1/Phase 2 NCT00633139 Denmark;
Recombinant human Arysulfatase B, recombinant human N-acetylgalactosamine 4-sulfatase
BioMarin Pharmaceutical Inc.
2007 Phase 4 EUCTR2005-003512-30-FR France;Portugal;
Recombinant human N-acetylgalactosamine 4-sulfatase
BioMarin Pharmaceutical Inc.
- Phase 3 EUCTR2004-000642-21-IE Ireland;
Recombinant human N-acetylgalactosamine 6-sulfatase, BMN 110
BIOMARIN PHARMACEUTICAL INC.
2011 - EUCTR2010-020199-45-IT Argentina;Australia;Brazil;Canada;Colombia;Denmark;Germany;Italy;Japan;Korea, Democratic People's Republic of;Portugal;Qatar;Saudi Arabia;Spain;Switzerland;Taiwan;Turkey;United Kingdom;United States;
BioMarin Pharmaceutical Inc.
2012 Phase 3 EUCTR2010-020199-45-DE Argentina;Australia;Brazil;Canada;Colombia;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Portugal;Qatar;Saudi Arabia;Spain;Switzerland;Taiwan;Turkey;United Kingdom;United States;
2012 - EUCTR2010-020199-45-PT Argentina;Australia;Brazil;Canada;Colombia;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Portugal;Qatar;Saudi Arabia;Spain;Switzerland;Taiwan;Turkey;United Kingdom;United States;
Recombinant human N-acetylgalactosamine 6-sulfatase, BMN 110 drug substance
BioMarin Pharmaceutical Inc.
2009 - EUCTR2008-007365-23-GB United Kingdom;
Recombinant human N-acetylgalactosamine-6-sulfatase (rhGALNS)
BioMarin Pharmaceutical Inc.
2013 Phase 3 EUCTR2010-020199-45-ES Argentina;Australia;Brazil;Canada;Colombia;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Netherlands;Norway;Portugal;Qatar;Saudi Arabia;Spain;Taiwan;United Kingdom;United States;
2012 Phase 3 EUCTR2010-020199-45-DK Argentina;Australia;Brazil;Canada;Colombia;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Netherlands;Norway;Portugal;Qatar;Saudi Arabia;Spain;Taiwan;United Kingdom;United States;
2012 - EUCTR2011-005703-33-GB Germany;Italy;United Kingdom;United States;
2012 - EUCTR2011-005703-33-DE Germany;Italy;United Kingdom;United States;
2011 EUCTR2010-020199-45-GB Argentina;Australia;Brazil;Canada;Colombia;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Portugal;Qatar;Saudi Arabia;Spain;Switzerland;Taiwan;United Kingdom;United States;
Recombinant human N-acetylgalactosamine-6-sulfatase, BMN 110
BioMarin Pharmaceutical Inc.
2013 Phase 3 EUCTR2010-020199-45-ES Argentina;Australia;Brazil;Canada;Colombia;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Netherlands;Norway;Portugal;Qatar;Saudi Arabia;Spain;Taiwan;United Kingdom;United States;
2012 Phase 3 EUCTR2010-020199-45-DK Argentina;Australia;Brazil;Canada;Colombia;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Netherlands;Norway;Portugal;Qatar;Saudi Arabia;Spain;Taiwan;United Kingdom;United States;
Recombinant human N-acetylgalactosamine-6-sulfatase, BMN 110 drug substance
BIOMARIN PHARMACEUTICAL INC.
2012 Phase 2 EUCTR2011-003197-84-IT Italy;United Kingdom;United States;
2011 - EUCTR2010-020198-18-IT Denmark;Germany;Italy;Norway;Portugal;United Kingdom;
BioMarin Pharmaceutical Inc
2012 Phase 3 EUCTR2010-020198-18-NO Argentina;Australia;Brazil;Canada;Colombia;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Portugal;Qatar;Saudi Arabia;Switzerland;Taiwan;United Kingdom;United States;
2011 - EUCTR2010-020198-18-PT Argentina;Australia;Brazil;Canada;Colombia;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Portugal;Qatar;Saudi Arabia;Switzerland;Taiwan;United Kingdom;United States;
2011 - EUCTR2010-020198-18-DK Argentina;Australia;Brazil;Canada;Colombia;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Portugal;Qatar;Saudi Arabia;Switzerland;Taiwan;United Kingdom;United States;
2011 - EUCTR2010-020198-18-DE Argentina;Australia;Brazil;Canada;Colombia;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Portugal;Qatar;Saudi Arabia;Switzerland;Taiwan;United Kingdom;United States;
2010 - EUCTR2010-020198-18-GB Argentina;Australia;Brazil;Canada;Colombia;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Portugal;Qatar;Saudi Arabia;Spain;Switzerland;Taiwan;Turkey;United Kingdom;United States;
- Phase 3 EUCTR2010-020198-18-NL Denmark;France;Germany;Italy;Netherlands;Norway;Portugal;United Kingdom;
- Phase 3 EUCTR2010-020198-18-FR Denmark;France;Germany;Italy;Netherlands;Norway;Portugal;United Kingdom;
BioMarin Pharmaceutical Inc.
2012 - EUCTR2011-005703-33-GB Germany;Italy;United Kingdom;United States;
2012 - EUCTR2011-005703-33-DE Germany;Italy;United Kingdom;United States;
2012 - EUCTR2011-005682-20-GB Canada;Germany;United Kingdom;United States;
2012 - EUCTR2011-005682-20-DE Canada;Germany;United Kingdom;United States;
2011 Phase 2 EUCTR2011-003197-84-GB Italy;United Kingdom;United States;
2011 EUCTR2010-020199-45-GB Argentina;Australia;Brazil;Canada;Colombia;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Portugal;Qatar;Saudi Arabia;Spain;Switzerland;Taiwan;United Kingdom;United States;
2010 EUCTR2010-021048-16-GB United Kingdom;
Recombinant human Nacetylgalactosamine-
BioMarin Pharmaceutical Inc.
2012 Phase 3 EUCTR2010-020199-45-DE Argentina;Australia;Brazil;Canada;Colombia;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Portugal;Qatar;Saudi Arabia;Spain;Switzerland;Taiwan;Turkey;United Kingdom;United States;
2012 - EUCTR2010-020199-45-PT Argentina;Australia;Brazil;Canada;Colombia;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Portugal;Qatar;Saudi Arabia;Spain;Switzerland;Taiwan;Turkey;United Kingdom;United States;
Recombinant human alpha-galactosidase
Genzyme Europe BV
2008 - EUCTR2007-005668-28-GB Argentina;Brazil;Canada;Czech Republic;France;Germany;Netherlands;Norway;Poland;Portugal;United Kingdom;United States;
Recombinant human alpha-galactosidase abbreviated as r-h-alpha-GAL
Genzyme Europe BV
2009 Phase 3 EUCTR2007-005668-28-FR Czech Republic;France;Germany;Netherlands;Portugal;United Kingdom;
2008 - EUCTR2007-005668-28-PT Czech Republic;France;Germany;Netherlands;Portugal;United Kingdom;
2008 - EUCTR2007-005668-28-NL Czech Republic;France;Germany;Netherlands;Portugal;United Kingdom;
2008 - EUCTR2007-005668-28-DE Czech Republic;France;Germany;Netherlands;Portugal;United Kingdom;
2008 - EUCTR2007-005668-28-CZ Czech Republic;France;Germany;Netherlands;Portugal;United Kingdom;
Recombinant human alpha-mannosidase
CHIESI FARMACEUTICI S.P.A.
2018 Phase 2 EUCTR2016-001988-36-IT Austria;Denmark;France;Germany;Italy;
Chiesi Farmaceutici S.p.A.
2017 Phase 2 EUCTR2016-001988-36-FR Austria;Denmark;France;Germany;Italy;
2017 Phase 2 EUCTR2016-001988-36-DE Austria;Denmark;Germany;
2017 Phase 2 EUCTR2016-001988-36-AT Austria;Denmark;Germany;
2016 Phase 2 EUCTR2016-001988-36-DK Austria;Denmark;Germany;Italy;
2013 Phase 3 EUCTR2013-000321-31-DK Denmark;Poland;
- Phase 3 EUCTR2013-000321-31-PL Denmark;Poland;
Zymenex A/S
2013 Phase 3 EUCTR2012-000979-17-ES Belgium;Denmark;Germany;Spain;Sweden;
2013 - EUCTR2012-000979-17-SE Belgium;Denmark;Germany;Spain;Sweden;
2013 - EUCTR2012-000979-17-BE Belgium;Denmark;Germany;Spain;Sweden;
2012 Phase 2 EUCTR2011-004355-40-DK Belgium;Denmark;Germany;Spain;United Kingdom;
2012 - EUCTR2012-000979-17-DK Belgium;Denmark;Germany;Spain;Sweden;
2012 - EUCTR2012-000979-17-DE Belgium;Denmark;Germany;Spain;Sweden;
2012 - EUCTR2011-004355-40-GB Belgium;Denmark;Germany;Spain;United Kingdom;
2012 - EUCTR2011-004355-40-DE Belgium;Denmark;Germany;Spain;United Kingdom;
2012 - EUCTR2011-004355-40-BE Belgium;Denmark;Germany;Spain;United Kingdom;
2010 - EUCTR2010-022085-26-DK Denmark;
2010 - EUCTR2010-022084-36-DK Denmark;
Recombinant human arylsulfatase A
Shire
2012 Phase 1/Phase 2 NCT01510028 Argentina;Australia;Brazil;Denmark;France;Germany;Japan;
Shire Human Genetic Therapies Inc
2010 Phase 2 EUCTR2008-000084-41-BE Belgium;Denmark;France;Italy;
2009 - EUCTR2008-000084-41-FR Belgium;Denmark;France;Italy;
2008 Phase 2 EUCTR2008-000084-41-DK Belgium;Denmark;France;Italy;
Shire Pharmaceutical Ireland Limited
2008 - EUCTR2007-006345-40-DK Denmark;
Shire Pharmaceuticals Ireland Limited
2008 Phase 2 EUCTR2007-007165-20-DK Denmark;
Recombinant human beta-glucuronidase
Hamazaki Takashi
2020 Phase 3 JPRN-jRCT2051190085 Japan;
Ultragenyx Pharmaceutical Inc.
2016 Phase 2 EUCTR2015-000104-26-PT Portugal;Spain;United States;
2016 Phase 2 EUCTR2015-000104-26-ES Portugal;Spain;United States;
2014 Phase 1;Phase 2 EUCTR2013-001152-35-ES Spain;United Kingdom;
2013 Phase 1;Phase 2 EUCTR2013-001152-35-GB Spain;United Kingdom;
- - EUCTR2015-001875-32-Outside-EU/EEA United States;
- - EUCTR2014-005638-71-Outside-EU/EEA United States;
Recombinant human beta-glucuronidase (rhGUS)
Ultragenyx Pharmaceutical Inc.
2016 Phase 3 EUCTR2015-001875-32-PT Brazil;Mexico;Portugal;United States;
Recombinant human betaglucuronidase
Ultragenyx Pharmaceutical Inc.
- - EUCTR2015-000104-26-Outside-EU/EEA United States;
Recombinant human derived macrophage-targeted beta-Glucocerebrosidase
Genzyme Europe B.V.
2010 - EUCTR2008-005223-28-DE Argentina;Australia;Brazil;Canada;Czech Republic;Egypt;France;Germany;Italy;Netherlands;Russian Federation;Spain;Turkey;United Kingdom;United States;
2009 Phase 3 EUCTR2008-005223-28-GB Argentina;Australia;Brazil;Canada;Czech Republic;Egypt;France;Germany;Italy;Netherlands;Russian Federation;Spain;Turkey;United Kingdom;United States;
2009 Phase 3 EUCTR2008-005223-28-FR Czech Republic;France;Germany;Italy;Netherlands;Spain;United Kingdom;
2009 - EUCTR2008-005223-28-NL Czech Republic;France;Germany;Italy;Netherlands;Spain;United Kingdom;
Recombinant human derived macrophage-targeted beta-glucocerebrosidase
Genzyme Europe B.V.
2009 - EUCTR2008-005223-28-CZ Czech Republic;France;Germany;Italy;Netherlands;Spain;United Kingdom;
Recombinant human derived macrophage-targeted ß-Glucocerebrosidase
Genzyme Europe B.V.
2010 - EUCTR2008-005223-28-DE Argentina;Australia;Brazil;Canada;Czech Republic;Egypt;France;Germany;Italy;Netherlands;Russian Federation;Spain;Turkey;United Kingdom;United States;
2009 Phase 3 EUCTR2008-005223-28-GB Argentina;Australia;Brazil;Canada;Czech Republic;Egypt;France;Germany;Italy;Netherlands;Russian Federation;Spain;Turkey;United Kingdom;United States;
2009 Phase 3 EUCTR2008-005223-28-FR Czech Republic;France;Germany;Italy;Netherlands;Spain;United Kingdom;
2009 - EUCTR2008-005223-28-NL Czech Republic;France;Germany;Italy;Netherlands;Spain;United Kingdom;
Recombinant human derived macrophage-targeted ß-glucocerebrosidase
Genzyme Europe B.V.
2009 - EUCTR2008-005223-28-CZ Czech Republic;France;Germany;Italy;Netherlands;Spain;United Kingdom;
Recombinant human glucerebrosidase
Protalix Biotherapeutics
2012 - EUCTR2011-003685-32-ES Canada;Chile;Israel;Italy;Mexico;Serbia;South Africa;Spain;
2011 - EUCTR2011-003676-37-ES Israel;South Africa;Spain;
Recombinant human glucocerebrosidase
Protalix Biotherapeutics
2009 - EUCTR2008-005826-35-ES Italy;Spain;United Kingdom;
2009 - EUCTR2008-005825-12-ES Germany;Italy;Spain;United Kingdom;
2008 Phase 3 EUCTR2007-000498-42-GB Italy;Spain;United Kingdom;
2008 - EUCTR2007-000498-42-ES Italy;Spain;United Kingdom;
Recombinant human glucocerebrosidase (prGCD)
Protalix Biotherapeutics
2009 Phase 3 EUCTR2008-005826-35-GB Italy;Spain;United Kingdom;
2009 Phase 3 EUCTR2008-005825-12-GB Germany;Italy;Spain;United Kingdom;
Recombinant human heparan N-sulfatase
SHIRE HUMAN GENETIC THERAPIES, INC
2015 Phase 2 EUCTR2014-003960-20-IT France;Germany;Italy;Netherlands;Spain;United Kingdom;United States;
Recombinant human heparan N-sulfatase (rhHNS)
Shire
2010 Phase 1/Phase 2 NCT01155778 Netherlands;United Kingdom;
Shire Human Genetic Therapies, Inc
2014 Phase 2 EUCTR2013-003450-24-NL Argentina;France;Germany;Italy;Netherlands;Spain;United Kingdom;United States;
2014 Phase 2 EUCTR2013-003450-24-IT France;Germany;Italy;Netherlands;Spain;United Kingdom;
2014 Phase 2 EUCTR2013-003450-24-GB Argentina;Brazil;France;Germany;Italy;Netherlands;Spain;United Kingdom;United States;
2014 Phase 2 EUCTR2013-003450-24-DE Argentina;Brazil;France;Germany;Italy;Netherlands;Spain;United Kingdom;United States;
2011 Phase 1;Phase 2 EUCTR2010-021348-16-NL Netherlands;United Kingdom;
2011 Phase 1;Phase 2 EUCTR2010-021348-16-GB Netherlands;United Kingdom;
- Phase 2 EUCTR2013-003450-24-FR Argentina;Brazil;France;Germany;Italy;Netherlands;Spain;United Kingdom;United States;
Shire Human Genetic Therapies, Inc.
2015 Phase 2 EUCTR2014-003960-20-NL France;Germany;Italy;Netherlands;Spain;United Kingdom;United States;
2015 Phase 2 EUCTR2014-003960-20-GB France;Germany;Italy;Netherlands;Spain;United Kingdom;United States;
2015 Phase 2 EUCTR2014-003960-20-DE France;Germany;Italy;Netherlands;Spain;United Kingdom;United States;
Shire Human Genetic Therapies, Inc. (Shire HGT)
2010 Phase 1;Phase 2 EUCTR2009-015984-15-GB United Kingdom;
Shire human Genetic Therapies, Inc.
2015 Phase 2 EUCTR2014-003960-20-ES Argentina;France;Germany;Italy;Netherlands;Spain;United Kingdom;United States;
- Phase 2 EUCTR2014-003960-20-FR Argentina;France;Germany;Italy;Netherlands;Spain;United Kingdom;United States;
Recombinant human heparan N-sulfatase [rhHNS]
Shire
2014 Phase 2 NCT02060526 Argentina;France;Germany;Italy;Netherlands;Spain;United Kingdom;United States;
Recombinant human heparan Nsulfatase (rhHNS)
SHIRE HUMAN GENETIC THERAPIES, INC
2015 Phase 2 EUCTR2014-003960-20-IT France;Germany;Italy;Netherlands;Spain;United Kingdom;United States;
Recombinant human lysosomal acid lipase (rhLAL)
Alexion Pharmaceuticals Inc.
2013 Phase 2;Phase 3 EUCTR2011-000032-28-IE Egypt;France;Germany;Ireland;Italy;Saudi Arabia;Taiwan;Turkey;United Kingdom;United States;
SYNAGEVA BIOPHARMA CORP.
2012 - EUCTR2011-000032-28-IT Germany;Ireland;Italy;Taiwan;United Kingdom;United States;
Synageva Biopharma Corp.
2013 - EUCTR2011-000032-28-DE France;Germany;Italy;Taiwan;United Kingdom;United States;
2011 Phase 1;Phase 2;Phase 3 EUCTR2011-000032-28-GB France;Germany;Ireland;Italy;Taiwan;United Kingdom;United States;
2011 - EUCTR2011-002880-42-GB France;Germany;United Kingdom;United States;
2011 - EUCTR2010-024068-16-GB Czech Republic;United Kingdom;
2011 - EUCTR2010-024068-16-CZ Czech Republic;United Kingdom;United States;
- Phase 2 EUCTR2011-002880-42-FR France;Germany;United Kingdom;United States;
- Phase 1;Phase 2 EUCTR2011-000032-28-FR France;Germany;Ireland;Italy;United Kingdom;United States;
Recombinant human lysosomal acid lipase (rhLAL), esterase, cholesterol (human gene LIPA)
Synageva Biopharma Corp.
2011 Phase 2 EUCTR2011-001513-13-GB Czech Republic;France;United Kingdom;United States;
Recombinant human lysosomal acid lipase (rhLAL), esterase, cholesterol (human gene LIPA), lysosomal acid lipase (human gene LIPA)
Synageva Biopharma Corp.
2011 Phase 2 EUCTR2011-001513-13-CZ Czech Republic;France;United Kingdom;United States;
Recombinant human lysosomal alpha-mannosidase
Zymenex A/S
2015 - EUCTR2014-003950-15-DK Denmark;
2013 Phase 3 EUCTR2013-000336-97-FR Denmark;France;
Recombinant human tripeptidyl peptidase-1
BioMarin Pharmaceutical Inc
2017 Phase 2 EUCTR2015-000891-85-GB Germany;Italy;United Kingdom;United States;
BioMarin Pharmaceutical Inc.
2016 Phase 2 EUCTR2015-000891-85-DE Germany;Italy;United Kingdom;
2014 Phase 1;Phase 2 EUCTR2012-005430-11-IT Germany;Italy;United Kingdom;
2013 Phase 1;Phase 2 EUCTR2012-005430-11-GB Germany;Italy;United Kingdom;United States;
2013 Phase 1;Phase 2 EUCTR2012-005430-11-DE Germany;United Kingdom;
Recombinant self-complementary adeno-associated viral vector serotype 9 containing the human CLN6 gene
Amicus Therapeutics, Inc.
2021 Phase 1;Phase 2 EUCTR2021-000706-21-DE Germany;United States;
Reduced Intensity Conditioning
Duke University
2008 Phase 1 NCT00744692 United States;
Reminder Product
Amicus Therapeutics, Inc.
2010 Phase 3 EUCTR2009-013459-31-BE Belgium;Denmark;France;Germany;Italy;Netherlands;Spain;United Kingdom;
2010 - EUCTR2009-013459-31-NL Belgium;Denmark;France;Germany;Italy;Netherlands;Spain;United Kingdom;
- - EUCTR2009-013459-31-DE Belgium;Denmark;France;Germany;Italy;Netherlands;Spain;United Kingdom;
Reminder capsules
Amicus Therapeutics, Inc.
2009 Phase 2 EUCTR2007-001838-13-GB Australia;Brazil;United Kingdom;United States;
Replagal
Amicus Therapeutics, Inc.
2012 - EUCTR2010-022709-16-GB Belgium;United Kingdom;
2012 - EUCTR2010-022636-37-DE Argentina;Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Hungary;Israel;Italy;Japan;Mexico;Poland;Russian Federation;Slovakia;Switzerland;Taiwan;Turkey;United Kingdom;United States;
2011 Phase 3 EUCTR2010-022636-37-GR Argentina;Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Hungary;Israel;Italy;Japan;Mexico;Poland;Russian Federation;Slovakia;Switzerland;Taiwan;Turkey;United Kingdom;United States;
2011 Phase 2 EUCTR2010-022709-16-BE Australia;Belgium;Canada;France;Netherlands;Switzerland;United Kingdom;
2011 - EUCTR2010-022636-37-GB Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;United Kingdom;United States;
2011 - EUCTR2010-022636-37-DK Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;United Kingdom;United States;
2011 - EUCTR2010-022636-37-BE Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;United Kingdom;United States;
2011 - EUCTR2010-022636-37-AT Australia;Austria;Belgium;Brazil;Denmark;France;Germany;Greece;Italy;Japan;United Kingdom;United States;
Baylor Research Institute
2003 Phase 2 NCT00068107 United States;
National Institute of Neurological Disorders and Stroke (NINDS)
2004 Phase 2 NCT00075244 United States;
2003 Phase 2 NCT00071877 United States;
SANOFI-AVENTIS RECHERCHE E DEVELOPPEMENT
2022 Phase 3 EUCTR2021-002320-20-IT Austria;Canada;China;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Turkey;United Kingdom;United States;
Sanofi Aventis Groupe (SAG)
2019 Phase 4 EUCTR2019-000064-21-GB Austria;Canada;Czech Republic;Denmark;France;Germany;Italy;Norway;Spain;Turkey;United Kingdom;
- Phase 4 EUCTR2019-000064-21-NO Austria;Canada;Czech Republic;Denmark;France;Germany;Italy;Norway;Spain;Turkey;United Kingdom;
- Phase 4 EUCTR2019-000064-21-CZ Austria;Canada;Czech Republic;Denmark;France;Germany;Italy;Norway;Spain;Turkey;United Kingdom;
Shire
2022 Phase 4 NCT05067868 India;
2010 Phase 3 NCT01124643 Australia;Czech Republic;Czechia;Finland;Paraguay;Poland;Slovenia;United Kingdom;United States;
2008 Phase 3 NCT00864851 Australia;Czech Republic;Czechia;Finland;Paraguay;Poland;Slovenia;United Kingdom;United States;
Shire Human Genetic Therapies (HGT), Inc.
2012 Phase 3;Phase 4 EUCTR2009-015985-75-SI Australia;Czech Republic;Finland;Paraguay;Poland;Slovenia;United Kingdom;United States;
2011 Phase 3;Phase 4 EUCTR2009-015985-75-GB Australia;Czech Republic;Finland;Paraguay;Poland;Slovenia;United Kingdom;United States;
2011 - EUCTR2009-015985-75-FI Australia;Czech Republic;Finland;Paraguay;Poland;Slovenia;United Kingdom;United States;
2010 - EUCTR2009-015985-75-PL Australia;Czech Republic;Finland;Paraguay;Poland;Slovenia;United Kingdom;United States;
2010 - EUCTR2009-015985-75-CZ Australia;Czech Republic;Finland;Paraguay;Poland;Slovenia;United Kingdom;United States;
Shire Human Genetic Therapies, Inc. (Shire HGT)
2011 - EUCTR2007-005543-22-SI Australia;Czech Republic;Finland;Paraguay;Slovenia;United Kingdom;United States;
2010 Phase 3;Phase 4 EUCTR2007-005543-22-GB Australia;Czech Republic;Finland;Paraguay;Slovenia;United Kingdom;United States;
2010 Phase 3;Phase 4 EUCTR2007-005543-22-FI Australia;Czech Republic;Finland;Paraguay;Slovenia;United Kingdom;United States;
2008 - EUCTR2007-005543-22-CZ Australia;Czech Republic;Finland;Paraguay;Slovenia;United Kingdom;United States;
TKT Inc
2004 - EUCTR2004-000772-14-CZ Czech Republic;
sanofi-aventis recherche et développement
- Phase 3 EUCTR2021-002320-20-ES Austria;Canada;China;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Turkey;United Kingdom;United States;
Replagal (Agalsidase Alfa)
National Institute of Neurological Disorders and Stroke (NINDS)
2004 Phase 4 NCT00097890 United States;
Replagal (agalsidase alfa)
Shire
2011 Phase 2 NCT01363492 United States;
Replagal agalsidase alfa
National Institute of Neurological Disorders and Stroke (NINDS)
2003 Phase 1 NCT00357786 United States;
Rh Growth Hormone
National Human Genome Research Institute (NHGRI)
2014 Phase 1/Phase 2 NCT02124070 United States;
RhARSA
Assistance Publique - Hôpitaux de Paris
2008 Phase 2 NCT01303146 France;
RhASA
Shire Human Genetic Therapies Inc
2010 Phase 2 EUCTR2008-000084-41-BE Belgium;Denmark;France;Italy;
2009 - EUCTR2008-000084-41-FR Belgium;Denmark;France;Italy;
2008 Phase 2 EUCTR2008-000084-41-DK Belgium;Denmark;France;Italy;
Shire Pharmaceutical Ireland Limited
2008 - EUCTR2007-006345-40-DK Denmark;
Shire Pharmaceuticals Ireland Limited
2008 Phase 2 EUCTR2007-007165-20-DK Denmark;
2006 - EUCTR2006-005341-11-DK Denmark;
RhASA - Dose Level 1
Shire
2007 Phase 1 NCT00418561 Denmark;
RhASA - Dose Level 2
Shire
2007 Phase 1 NCT00418561 Denmark;
RhASA - Dose Level 3
Shire
2007 Phase 1 NCT00418561 Denmark;
RhASM
Genzyme, a Sanofi Company
2006 Phase 1 NCT00410566 United States;
RhHNS-
Shire
2011 Phase 1/Phase 2 NCT01299727 Netherlands;United Kingdom;
RhIDU (recombinant human-Alpha-L-Iduronidase)
Genzyme, a Sanofi Company
2000 Phase 3 NCT00912925 Canada;Germany;United States;
RhNAGLU radiolabelled
Alexion Pharmaceuticals, Inc.
2016 Phase 2 EUCTR2015-000753-20-NL Netherlands;
Rimiducid
Bellicum Pharmaceuticals
2017 - NCT03639844 United States;
Rituximab
National Human Genome Research Institute (NHGRI)
2019 Phase 1/Phase 2 NCT03952637 United States;
Rivogenlecleucel
Bellicum Pharmaceuticals
2017 - NCT03639844 United States;
SAF-301
SANFILIPPO Therapeutics SAS
- - EUCTR2010-019962-10-FR France;
SAR402671, GZ402671 o GZ/SAR402671
SANOFI-AVENTIS RECHERCHE E DEVELOPPEMENT
2022 Phase 3 EUCTR2021-002320-20-IT Austria;Canada;China;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Turkey;United Kingdom;United States;
2021 Phase 3 EUCTR2021-002350-90-IT Argentina;Austria;Brazil;Bulgaria;Canada;China;Denmark;Finland;France;Germany;Greece;Italy;Mexico;Netherlands;Norway;Poland;Romania;Russian Federation;Turkey;Ukraine;United Kingdom;
SAR402671, GZ402671 or GZ / SAR402671
Sanofi-aventis recherche & développement
- Phase 3 EUCTR2021-005402-10-FR Argentina;Brazil;Canada;China;Egypt;France;Germany;Hungary;Japan;Taiwan;Turkey;United Kingdom;United States;
- Phase 3 EUCTR2021-005402-10-DE Argentina;Brazil;Canada;China;Egypt;France;Germany;Hungary;Japan;Taiwan;Turkey;United Kingdom;United States;
SAR402671, GZ402671 or GZ/SAR402671
Genzyme Corporation
2015 Phase 2 EUCTR2014-004995-49-PL Czech Republic;France;Poland;Russian Federation;United Kingdom;United States;
2015 Phase 2 EUCTR2014-004995-49-GB Czech Republic;France;Poland;Russian Federation;United Kingdom;United States;
2015 Phase 2 EUCTR2014-004995-49-FR Czech Republic;France;Poland;Russian Federation;United Kingdom;United States;
Sanofi-Aventis Recherche & Développement
2021 Phase 3 EUCTR2021-002350-90-GR Argentina;Austria;Brazil;Bulgaria;Canada;China;Denmark;Finland;France;Germany;Greece;Italy;Mexico;Netherlands;Norway;Poland;Romania;Russian Federation;Turkey;Ukraine;United Kingdom;
sanofi-aventis recherche et développement
- Phase 3 EUCTR2021-002320-20-ES Austria;Canada;China;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Turkey;United Kingdom;United States;
SB-318
Sangamo Therapeutics
2020 - NCT04628871 United States;
2017 Phase 1/Phase 2 NCT02702115 United States;
SB-47171
Sangamo Therapeutics, Inc.
2018 Phase 1;Phase 2 EUCTR2018-000206-28-GB United Kingdom;United States;
2018 Phase 1;Phase 2 EUCTR2018-000192-33-GB United Kingdom;United States;
SB-47898
Sangamo Therapeutics, Inc.
2018 Phase 1;Phase 2 EUCTR2018-000206-28-GB United Kingdom;United States;
2018 Phase 1;Phase 2 EUCTR2018-000192-33-GB United Kingdom;United States;
SB-913
Sangamo Therapeutics
2020 - NCT04628871 United States;
2017 Phase 1/Phase 2 NCT03041324 United States;
SB-FIX
Sangamo Therapeutics
2020 - NCT04628871 United States;
SB-IDUA
Sangamo Therapeutics, Inc.
2018 Phase 1;Phase 2 EUCTR2018-000206-28-GB United Kingdom;United States;
SBC-102
Alexion Pharmaceuitcals Inc
2014 Phase 2 EUCTR2011-004287-30-HR Australia;Belgium;Brazil;Canada;Croatia;Denmark;Germany;Italy;Japan;Mexico;Netherlands;Russian Federation;Spain;Turkey;United Kingdom;United States;
2014 Phase 2 EUCTR2011-004287-30-DE Australia;Belgium;Brazil;Canada;Croatia;Denmark;Germany;Italy;Japan;Mexico;Netherlands;Russian Federation;Spain;Turkey;United Kingdom;United States;
Alexion Pharmaceuticals
2011 Phase 2/Phase 3 NCT01473875 France;United Kingdom;
Alexion Pharmaceuticals Inc
2015 Phase 2 EUCTR2014-000533-22-FI Finland;Italy;United Kingdom;United States;
2015 Phase 2 EUCTR2011-004287-30-NL Australia;Belgium;Brazil;Canada;Croatia;Denmark;Germany;Italy;Mexico;Netherlands;Russian Federation;Spain;Turkey;United Kingdom;United States;
2015 Phase 2 EUCTR2011-004287-30-GB Australia;Belgium;Brazil;Canada;Croatia;Denmark;Germany;Italy;Japan;Mexico;Netherlands;Poland;Russian Federation;Spain;Turkey;United Kingdom;United States;
2014 Phase 2 EUCTR2014-000533-22-GB Finland;Italy;United Kingdom;United States;
Alexion Pharmaceuticals inc
2014 Phase 2 EUCTR2011-004287-30-DK Australia;Belgium;Brazil;Canada;Croatia;Denmark;Germany;Italy;Japan;Mexico;Netherlands;Poland;Russian Federation;Spain;Turkey;United Kingdom;United States;
Alexion Pharmaceuticals, Inc.
2015 Phase 3 EUCTR2011-002750-31-CZ Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Japan;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;United States;
2013 Phase 3 EUCTR2011-002750-31-GB Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Japan;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;United States;
2013 Phase 3 EUCTR2011-002750-31-DE Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Japan;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;United States;
- Phase 3 EUCTR2011-002750-31-PL Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Japan;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;United States;
SYNAGEVA BIOPHARMA CORP.
2012 - EUCTR2011-000032-28-IT Germany;Ireland;Italy;Taiwan;United Kingdom;United States;
Synageva BioPharma Corp.
2014 Phase 2 EUCTR2014-000533-22-IT Finland;Italy;United Kingdom;United States;
2014 Phase 2 EUCTR2011-004287-30-IT Australia;Belgium;Brazil;Canada;Croatia;Denmark;Germany;Italy;Japan;Mexico;Netherlands;Poland;Russian Federation;Spain;Turkey;United Kingdom;United States;
2014 Phase 2 EUCTR2011-004287-30-ES Australia;Belgium;Brazil;Canada;Croatia;Denmark;Germany;Italy;Japan;Mexico;Netherlands;Poland;Russian Federation;Spain;Turkey;United Kingdom;United States;
2014 Phase 2 EUCTR2011-004287-30-BE Australia;Belgium;Brazil;Canada;Croatia;Denmark;Germany;Italy;Japan;Mexico;Netherlands;Russian Federation;Spain;Turkey;United Kingdom;United States;
Synageva BioPharma Corporation
2013 Phase 3 EUCTR2011-002750-31-IT Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;
2013 Phase 3 EUCTR2011-002750-31-HR Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;
2013 Phase 3 EUCTR2011-002750-31-GR Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;
2013 Phase 3 EUCTR2011-002750-31-ES Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;
- Phase 3 EUCTR2011-002750-31-FR Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;
Synageva Biopharma Corp.
2011 Phase 2 EUCTR2011-001513-13-GB Czech Republic;France;United Kingdom;United States;
2011 Phase 2 EUCTR2011-001513-13-CZ Czech Republic;France;United Kingdom;United States;
2011 Phase 1;Phase 2;Phase 3 EUCTR2011-000032-28-GB France;Germany;Ireland;Italy;Taiwan;United Kingdom;United States;
2011 - EUCTR2011-002880-42-GB France;Germany;United Kingdom;United States;
2011 - EUCTR2010-024068-16-GB Czech Republic;United Kingdom;
2011 - EUCTR2010-024068-16-CZ Czech Republic;United Kingdom;United States;
- Phase 2 EUCTR2011-002880-42-FR France;Germany;United Kingdom;United States;
- Phase 1;Phase 2 EUCTR2011-000032-28-FR France;Germany;Ireland;Italy;United Kingdom;United States;
SBC-102, recombinant human lysosomal acid lipase
Alexion Pharmaceuticals Inc.
2013 Phase 2;Phase 3 EUCTR2011-000032-28-IE Egypt;France;Germany;Ireland;Italy;Saudi Arabia;Taiwan;Turkey;United Kingdom;United States;
Synageva Biopharma Corp.
2013 - EUCTR2011-000032-28-DE France;Germany;Italy;Taiwan;United Kingdom;United States;
SBC-103
Alexion Pharmaceuticals
2015 Phase 1/Phase 2 NCT02618512 United Kingdom;
2015 Phase 1/Phase 2 NCT02324049 Spain;United Kingdom;United States;
Alexion Pharmaceuticals, Inc
2015 Phase 2 EUCTR2015-001983-20-GB United Kingdom;
Alexion Pharmaceuticals, Inc.
2016 Phase 1 EUCTR2013-003400-39-ES Spain;United Kingdom;United States;
2014 Phase 1;Phase 2 EUCTR2013-003400-39-GB Spain;United Kingdom;United States;
SHP611
SHIRE HUMAN GENETIC THERAPIES, INC
2020 Phase 2 EUCTR2018-003291-12-IT Belgium;Brazil;Canada;France;Germany;Israel;Italy;Japan;Netherlands;Spain;United Kingdom;United States;
Shire
2019 Phase 2 NCT03771898 Belgium;Brazil;Canada;Denmark;France;Germany;Israel;Japan;Mexico;Netherlands;Spain;United Kingdom;United States;
Shire Human Genetic Therapies, Inc.
2021 Phase 2 EUCTR2018-003291-12-GR Argentina;Belgium;Brazil;Canada;France;Germany;Greece;Israel;Italy;Japan;Netherlands;Spain;United Kingdom;United States;
2020 Phase 2 EUCTR2018-003291-12-DE Belgium;Brazil;Canada;France;Germany;Israel;Japan;Mexico;Netherlands;Spain;United Kingdom;United States;
2019 Phase 2 EUCTR2018-003291-12-NL Argentina;Belgium;Brazil;Canada;France;Germany;Greece;Israel;Italy;Japan;Netherlands;Spain;United Kingdom;United States;
2019 Phase 2 EUCTR2018-003291-12-GB Belgium;Brazil;Canada;France;Germany;Israel;Japan;Mexico;Netherlands;Spain;United Kingdom;United States;
2019 Phase 2 EUCTR2018-003291-12-FR Belgium;Brazil;Canada;Denmark;France;Germany;Israel;Japan;Mexico;Netherlands;Spain;United Kingdom;United States;
2019 Phase 2 EUCTR2018-003291-12-ES Belgium;Brazil;Canada;Denmark;France;Germany;Israel;Japan;Mexico;Netherlands;Spain;United Kingdom;United States;
2019 Phase 2 EUCTR2018-003291-12-BE Belgium;Brazil;Canada;France;Germany;Israel;Japan;Mexico;Netherlands;Spain;United Kingdom;United States;
Shire Human Genetics Therapies Inc
2017 Phase 1;Phase 2 EUCTR2012-003775-20-IT Australia;Brazil;Czech Republic;Denmark;France;Germany;Italy;Japan;United Kingdom;
2016 Phase 1;Phase 2 EUCTR2012-003775-20-GB Australia;Brazil;Czech Republic;Denmark;France;Germany;Italy;Japan;United Kingdom;
2014 Phase 1;Phase 2 EUCTR2012-003775-20-CZ Australia;Brazil;Czech Republic;Denmark;France;Germany;Italy;Japan;United Kingdom;
2013 Phase 1;Phase 2 EUCTR2012-003775-20-DK Australia;Brazil;Czech Republic;Czechia;Denmark;France;Germany;Italy;Japan;United Kingdom;
2013 Phase 1;Phase 2 EUCTR2012-003775-20-DE Australia;Brazil;Czech Republic;Denmark;France;Germany;Italy;Japan;United Kingdom;
SHP675
Shire Human Genetic Therapies, Inc.
2021 Phase 3 EUCTR2018-004689-32-SE Argentina;Australia;Belgium;Bosnia and Herzegovina;Brazil;Canada;Croatia;Finland;Germany;Greece;Hungary;Italy;Latvia;Poland;Portugal;Romania;Slovenia;Spain;Sweden;Ukraine;
2021 Phase 3 EUCTR2018-004689-32-PT Argentina;Australia;Belgium;Bosnia and Herzegovina;Brazil;Canada;Croatia;Finland;Germany;Greece;Hungary;Italy;Latvia;Poland;Portugal;Romania;Slovenia;Spain;Sweden;Ukraine;
2021 Phase 3 EUCTR2018-004689-32-PL Argentina;Australia;Belgium;Bosnia and Herzegovina;Brazil;Canada;Croatia;Finland;Germany;Greece;Hungary;Italy;Latvia;Poland;Portugal;Romania;Slovenia;Spain;Sweden;Ukraine;
2021 Phase 3 EUCTR2018-004689-32-GR Argentina;Australia;Belgium;Bosnia and Herzegovina;Brazil;Canada;Croatia;Finland;Germany;Greece;Hungary;Italy;Latvia;Poland;Portugal;Romania;Slovenia;Spain;Sweden;Ukraine;
2021 Phase 3 EUCTR2018-004689-32-FI Argentina;Australia;Belgium;Bosnia and Herzegovina;Brazil;Canada;Croatia;Finland;Germany;Greece;Hungary;Italy;Latvia;Poland;Portugal;Romania;Slovenia;Spain;Sweden;Ukraine;
SOBI003
Swedish Orphan Biovitrum
2019 Phase 1/Phase 2 NCT03811028 Turkey;United States;
2018 Phase 1/Phase 2 NCT03423186 Germany;Turkey;United States;
Swedish Orphan Biovitrum AB (publ)
2019 Phase 1 EUCTR2017-002806-10-NL Germany;Netherlands;Turkey;United States;
- Phase 1 EUCTR2017-002806-10-DE Germany;Netherlands;Turkey;United States;
ST-920
Sangamo Therapeutics
2021 - NCT05039866 United States;
2019 Phase 1/Phase 2 NCT04046224 United Kingdom;United States;
Sangamo Therapeutics, Inc.
2019 Phase 1;Phase 2 EUCTR2019-000667-24-GB United Kingdom;United States;
SUB195712
IntraBio Ltd
2019 Phase 2 EUCTR2018-004406-25-GB Germany;Spain;United Kingdom;United States;
2019 Phase 2 EUCTR2018-004331-71-GB Germany;Slovakia;Spain;United Kingdom;United States;
Saline Solution for Injection
Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
2017 Phase 1/Phase 2 NCT03153319 United States;
ScAAV9.CB.CLN6
Amicus Therapeutics, Inc.
2021 Phase 1;Phase 2 EUCTR2021-000706-21-DE Germany;United States;
ScAAV9.U1A.SGSH
Abeona Therapeutics Inc
2019 Phase 1;Phase 2 EUCTR2018-000504-42-ES Australia;Spain;United States;
2016 Phase 1;Phase 2 EUCTR2015-003904-21-ES Australia;Spain;United States;
ScAAV9.U1A.hSGSH
Abeona Therapeutics Inc
2020 Phase 1;Phase 2 EUCTR2019-002979-34-ES Australia;France;Germany;Spain;United States;
2019 Phase 1;Phase 2 EUCTR2015-003904-21-FR Australia;Brazil;France;Germany;Spain;United Kingdom;United States;
Sebelipase alfa
Alexion Pharmaceuticals
2015 - NCT02376751 -
2011 Phase 2 NCT01488097 Canada;Czech Republic;Czechia;France;United Kingdom;United States;
2011 Phase 1/Phase 2 NCT01307098 Czech Republic;Czechia;France;United Kingdom;United States;
Alexion Pharmaceuticals Inc.
2013 Phase 2;Phase 3 EUCTR2011-000032-28-IE Egypt;France;Germany;Ireland;Italy;Saudi Arabia;Taiwan;Turkey;United Kingdom;United States;
Alexion Pharmaceuticals, Inc.
2015 Phase 3 EUCTR2011-002750-31-CZ Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Japan;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;United States;
2013 Phase 3 EUCTR2011-002750-31-GB Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Japan;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;United States;
2013 Phase 3 EUCTR2011-002750-31-DE Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Japan;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;United States;
- Phase 3 EUCTR2011-002750-31-PL Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Japan;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;United States;
Synageva BioPharma Corporation
2013 Phase 3 EUCTR2011-002750-31-IT Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;
2013 Phase 3 EUCTR2011-002750-31-HR Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;
2013 Phase 3 EUCTR2011-002750-31-GR Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;
2013 Phase 3 EUCTR2011-002750-31-ES Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;
- Phase 3 EUCTR2011-002750-31-FR Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;
Synageva Biopharma Corp.
2013 - EUCTR2011-000032-28-DE France;Germany;Italy;Taiwan;United Kingdom;United States;
2011 Phase 2 EUCTR2011-001513-13-CZ Czech Republic;France;United Kingdom;United States;
Sebelipase alfa (SBC-102)
Alexion Pharmaceuticals
2011 Phase 2/Phase 3 NCT01371825 Egypt;France;Germany;India;Ireland;Italy;Saudi Arabia;Taiwan;Turkey;United Kingdom;United States;
Sirolimus
National Human Genome Research Institute (NHGRI)
2019 Phase 1/Phase 2 NCT03952637 United States;
Prevail Therapeutics
2021 Phase 1/Phase 2 NCT04411654 United Kingdom;United States;
Skeletal survey
National Human Genome Research Institute (NHGRI)
2019 Phase 1/Phase 2 NCT03952637 United States;
Skin biopsy
National Human Genome Research Institute (NHGRI)
2019 Phase 1/Phase 2 NCT03952637 United States;
Slit Lamp assessment and intra-ocular pressure measurement
Université de Montréal
2014 - NCT02023086 Canada;
Somatropin (DNA origin)
Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
2008 Phase 2/Phase 3 NCT00748969 United States;
Speech and modified barium swallow study
National Human Genome Research Institute (NHGRI)
2019 Phase 1/Phase 2 NCT03952637 United States;
Ss-mercaptoethylamine Hydrochloride & 2-aminoethanethiol Hydrochloride
Recordati Rare Diseases
2020 Phase 3 EUCTR2018-002984-24-IT Belgium;France;Germany;Italy;United Kingdom;
2020 Phase 3 EUCTR2018-002984-24-DE France;Germany;United Kingdom;
- Phase 3 EUCTR2018-002984-24-BE Belgium;France;Germany;Italy;United Kingdom;
Stem Cell Transplant
Masonic Cancer Center, University of Minnesota
2004 Phase 2 NCT00176891 United States;
1999 Phase 2 NCT00176917 United States;
1995 Phase 2/Phase 3 NCT00176904 United States;
Stem Cell Transplantation
Masonic Cancer Center, University of Minnesota
2014 Phase 2 NCT02171104 United States;
2008 Phase 2 NCT00668564 United States;
Surgery to implant human CNS stem cells (HuCNS-SC)
StemCells, Inc.
2006 Phase 1 NCT00337636 United States;
TBD
BioMarin Pharmaceutical Inc.
2015 - EUCTR2014-003480-37-IT Germany;Italy;Japan;United Kingdom;United States;
2014 Phase 1;Phase 2 EUCTR2012-005430-11-IT Germany;Italy;United Kingdom;
2013 Phase 1;Phase 2 EUCTR2012-005430-11-DE Germany;United Kingdom;
TSHA-101
Dr. Anupam Sehgal
2021 Phase 1/Phase 2 NCT04798235 Canada;
Taliglucerase alfa
Pfizer
2011 Phase 3 NCT01422187 -
2011 Phase 3 NCT01411228 Israel;Paraguay;South Africa;
2010 Phase 4 NCT01132690 Israel;Paraguay;South Africa;United States;
2008 Phase 3 NCT00712348 Australia;Canada;Israel;Serbia;Spain;United Kingdom;United States;
2008 Phase 3 NCT00705939 Australia;Canada;Chile;Israel;South Africa;Spain;United Kingdom;United States;
Protalix Biotherapeutics
2009 Phase 3 EUCTR2008-005826-35-GB Italy;Spain;United Kingdom;
2009 Phase 3 EUCTR2008-005825-12-GB Germany;Italy;Spain;United Kingdom;
2009 - EUCTR2008-005825-12-DE Germany;Italy;Spain;United Kingdom;
Taliglucerase alfa - Recombinant human glucocerebrosidase
Protalix Biotherapeutics
- - EUCTR2008-005826-35-Outside-EU/EEA Australia;Canada;Chile;Israel;South Africa;Spain;United Kingdom;United States;
- - EUCTR2008-005825-12-Outside-EU/EEA Canada;Israel;United Kingdom;United States;
- - EUCTR2007-000498-42-Outside-EU/EEA Israel;United States;
Temperature sensitivity measurement with Advanced Thermal Stimulation
Universitaire Ziekenhuizen Leuven
2014 Phase 0 NCT02533076 Belgium;
Thiotepa--escalated dose
University of Florida
2018 Phase 1/Phase 2 NCT03513328 United States;
Thiotepa--single daily dose
University of Florida
2018 Phase 1/Phase 2 NCT03513328 United States;
To be added after EV code is available
Passage Bio, Inc.
- Phase 1;Phase 2 EUCTR2020-005229-95-NL Brazil;Canada;Israel;Netherlands;United Kingdom;United States;
Total body Irradiation
Masonic Cancer Center, University of Minnesota
2006 Phase 2 NCT00383448 United States;
Trappsol Cyclo
CTD Holdings, Inc.
2016 Phase 1;Phase 2 EUCTR2015-005761-23-GB Italy;Sweden;United Kingdom;
Cyclo Therapeutics, Inc.
2017 Phase 1;Phase 2 EUCTR2015-005761-23-SE Italy;Sweden;United Kingdom;
Trappsol® Cyclo™
Cyclo Therapeutics, Inc.
2022 Phase 3 EUCTR2020-003136-25-PL Australia;France;Germany;Israel;Italy;Poland;United Kingdom;United States;
2022 Phase 3 EUCTR2020-003136-25-DE Australia;France;Germany;Israel;Italy;Poland;United Kingdom;United States;
2021 Phase 3 EUCTR2020-003136-25-FR Australia;France;Israel;United Kingdom;United States;
- Phase 3 EUCTR2020-003136-25-IT Australia;France;Germany;Israel;Italy;Poland;Spain;United Kingdom;United States;
Treosulfan
Tokai University School of Medicine
2013 Phase 1 JPRN-UMIN000009758 Japan;
Triapin& triapin Mite
Salford Royal NHS Foundation Trust
2009 Phase 4 EUCTR2007-007482-21-GB United Kingdom;
Tropicamide
Université de Montréal
2014 - NCT02023086 Canada;
UX003
Ultragenyx Pharmaceutical Inc
2015 Phase 3 NCT02432144 Brazil;Mexico;Portugal;United States;
2015 Phase 2 NCT02418455 Portugal;Spain;United States;
2014 Phase 3 NCT02230566 United States;
Ultragenyx Pharmaceutical Inc.
2016 Phase 3 EUCTR2015-001875-32-PT Brazil;Mexico;Portugal;United States;
2016 Phase 2 EUCTR2015-000104-26-PT Portugal;Spain;United States;
2016 Phase 2 EUCTR2015-000104-26-ES Portugal;Spain;United States;
2014 Phase 1;Phase 2 EUCTR2013-001152-35-ES Spain;United Kingdom;
2013 Phase 1;Phase 2 EUCTR2013-001152-35-GB Spain;United Kingdom;
- - EUCTR2015-001875-32-Outside-EU/EEA United States;
- - EUCTR2015-000104-26-Outside-EU/EEA United States;
- - EUCTR2014-005638-71-Outside-EU/EEA United States;
Unrelated Umbilical Cord Blood Transplant
Duke University
2008 Phase 1 NCT00744692 United States;
Urine collection
Baylor Research Institute
2018 - NCT04002531 United States;
VPRIV - 400 U - POLVERE PER SOLUZIONE PER INFUSIONE - USO ENDOVENOSO - FLACONCINO(VETRO) 1 FLACONCINO
SHIRE HUMAN GENETIC THERAPIES, INC
2018 Phase 4 EUCTR2015-001578-17-IT Germany;Israel;Italy;Spain;United Kingdom;United States;
VPRIV 400 Units powder for solution of infusion
Shire Human Genetic Therapies, Inc.
2016 Phase 4 EUCTR2015-001578-17-GB Canada;Germany;Israel;Spain;United Kingdom;United States;
2016 Phase 4 EUCTR2015-001578-17-DE Germany;India;Israel;Italy;Spain;Turkey;United Kingdom;United States;
2016 - EUCTR2015-001578-17-ES Canada;Germany;Israel;Italy;Spain;United Kingdom;United States;
VPRIV ®,
Shire
2007 Phase 3 NCT00430625 Argentina;Israel;Paraguay;Russian Federation;Tunisia;United States;
VPRIV®
Shire
2008 Phase 3 NCT00635427 Argentina;India;Israel;Korea, Republic of;Paraguay;Poland;Russian Federation;Spain;Tunisia;United Kingdom;United States;
VTS-270
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
2020 Phase 1/Phase 2 NCT03887533 United States;
Mandos LLC
2019 Phase 2/Phase 3 NCT03879655 Costa Rica;
Vtesse, Inc., a Mallinckrodt Pharmaceuticals Company
2020 Phase 2 NCT03687476 -
Vtesse, LLC, a Mallinckrodt Pharmaceuticals Company
2013 Phase 1 NCT01747135 United States;
Vector viral adenoasociado de serotipo 9 que contiene el gen de la sulfamidasa humana
Laboratorios del Dr. Esteve, S.A.
2016 Phase 1;Phase 2 EUCTR2015-000359-26-ES Spain;
Velaglucerase alfa
Baylor Research Institute
2015 Phase 4 NCT02528617 United States;
SHIRE HUMAN GENETIC THERAPIES, INC
2018 Phase 4 EUCTR2015-001578-17-IT Germany;Israel;Italy;Spain;United Kingdom;United States;
Shire
2016 Phase 4 NCT02574286 Israel;Spain;United Kingdom;United States;
2013 Phase 3 NCT01842841 Japan;
2012 Phase 3 NCT01614574 Japan;
2012 Phase 1/Phase 2 NCT01685216 Egypt;India;Tunisia;
2009 - NCT00954460 United States;
2008 Phase 3 NCT00553631 Argentina;India;Israel;Paraguay;Russian Federation;Spain;Tunisia;United Kingdom;United States;
Shire Human Genetic Therapies Inc
- Phase 2;Phase 3 EUCTR2006-006304-11-DE Germany;Italy;Spain;United Kingdom;
Shire Human Genetic Therapies, Inc.
2008 - EUCTR2008-001965-27-ES Spain;United Kingdom;
2007 - EUCTR2007-002840-21-GB Italy;Spain;United Kingdom;
Takeda
2021 Phase 4 NCT04718779 United States;
Velaglucerase alfa (VPRIV)
Shire
2021 - NCT04429984 India;
Velaglucerasi alfa
SHIRE HUMAN GENETIC THERAPIES, INC
2018 Phase 4 EUCTR2015-001578-17-IT Germany;Israel;Italy;Spain;United Kingdom;United States;
Velmanase Alfa (e.g. Lamazym)
Chiesi Farmaceutici S.p.A.
2016 Phase 2 NCT02998879 Austria;Denmark;France;Germany;Italy;
Velmanase alfa
Chiesi Farmaceutici S.p.A.
2015 Phase 3 EUCTR2013-000336-97-DK Denmark;France;
Venglustat
GENZYME CORPORATION
2020 Phase 3 EUCTR2019-002375-34-IT Argentina;Austria;Brazil;Czech Republic;Czechia;France;Germany;Italy;Japan;Portugal;Russian Federation;Spain;Turkey;United Kingdom;United States;
Genzyme Corporation
2021 Phase 3 EUCTR2019-002375-34-AT Argentina;Austria;Brazil;Czech Republic;Czechia;France;Germany;Italy;Japan;Portugal;Russian Federation;Spain;Turkey;United Kingdom;United States;
2020 Phase 3 EUCTR2019-002375-34-PT Argentina;Austria;Brazil;Czech Republic;Czechia;France;Germany;Italy;Japan;Portugal;Russian Federation;Spain;Turkey;United Kingdom;United States;
2020 Phase 3 EUCTR2019-002375-34-GB Argentina;Austria;Brazil;Czech Republic;France;Germany;Italy;Japan;Portugal;Russian Federation;Spain;Turkey;United Kingdom;United States;
2020 Phase 3 EUCTR2019-002375-34-DE Argentina;Austria;Brazil;Czech Republic;France;Germany;Italy;Japan;Portugal;Russian Federation;Spain;Turkey;United Kingdom;United States;
2020 Phase 3 EUCTR2019-002375-34-CZ Argentina;Austria;Brazil;Czech Republic;France;Germany;Italy;Japan;Portugal;Russian Federation;Spain;Turkey;United Kingdom;United States;
2016 Phase 2 EUCTR2014-002550-39-DE Germany;Japan;United Kingdom;United States;
- Phase 3 EUCTR2019-002375-34-FR Argentina;Austria;Brazil;Czech Republic;France;Germany;Italy;Japan;Portugal;Russian Federation;Spain;Turkey;United Kingdom;United States;
Genzyme, a Sanofi Company
2022 Phase 3 NCT05222906 -
Tanaka Tomoyuki
2020 Phase 3 JPRN-jRCT2021200023 Argentina;Austria;Brazil;Czechia;France;Germany;Italy;Japan;Portugal;Russian Federation;Spain;Turkey;United Kingdom;United States;
Venglustat (GZ402671)
Genzyme, a Sanofi Company
2022 Phase 3 NCT05206773 United States;
2017 Phase 2 NCT02843035 Germany;Japan;United Kingdom;United States;
Venglustat GZ402671
Genzyme, a Sanofi Company
2020 Phase 3 NCT04221451 Argentina;Austria;Brazil;Czechia;France;Germany;Italy;Japan;Portugal;Russian Federation;Spain;Turkey;United Kingdom;United States;
Venglustat malate
Genzyme Corporation
2021 Phase 2;Phase 3 EUCTR2020-003120-17-HU Argentina;Brazil;Canada;China;Egypt;France;Germany;Hungary;Japan;Sweden;Taiwan;Turkey;United States;
2021 Phase 2;Phase 3 EUCTR2020-003120-17-FR Argentina;Brazil;Canada;China;Egypt;France;Germany;Hungary;Japan;Sweden;Taiwan;Turkey;United States;
2021 Phase 2;Phase 3 EUCTR2020-003120-17-DE Argentina;Brazil;Canada;China;Egypt;France;Germany;Hungary;Japan;Sweden;Taiwan;Turkey;United States;
- Phase 2;Phase 3 EUCTR2020-003120-17-SE Argentina;Brazil;Canada;China;Egypt;France;Germany;Hungary;Japan;Sweden;Taiwan;Turkey;United States;
Sanofi-Aventis Recherche & Développement
2021 Phase 3 EUCTR2021-002350-90-GR Argentina;Austria;Brazil;Bulgaria;Canada;China;Denmark;Finland;France;Germany;Greece;Italy;Mexico;Netherlands;Norway;Poland;Romania;Russian Federation;Turkey;Ukraine;United Kingdom;
Sanofi-aventis recherche & développement
- Phase 3 EUCTR2021-005402-10-FR Argentina;Brazil;Canada;China;Egypt;France;Germany;Hungary;Japan;Taiwan;Turkey;United Kingdom;United States;
- Phase 3 EUCTR2021-005402-10-DE Argentina;Brazil;Canada;China;Egypt;France;Germany;Hungary;Japan;Taiwan;Turkey;United Kingdom;United States;
sanofi-aventis recherche et développement
- Phase 3 EUCTR2021-002320-20-ES Austria;Canada;China;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Turkey;United Kingdom;United States;
Venglustat malato
SANOFI-AVENTIS RECHERCHE E DEVELOPPEMENT
2022 Phase 3 EUCTR2021-002320-20-IT Austria;Canada;China;Czechia;Denmark;France;Germany;Greece;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Spain;Taiwan;Turkey;United Kingdom;United States;
2021 Phase 3 EUCTR2021-002350-90-IT Argentina;Austria;Brazil;Bulgaria;Canada;China;Denmark;Finland;France;Germany;Greece;Italy;Mexico;Netherlands;Norway;Poland;Romania;Russian Federation;Turkey;Ukraine;United Kingdom;
Vestronidase alfa
Hamazaki Takashi
2020 Phase 3 JPRN-jRCT2051190085 Japan;
Vimizim® (elosulfase alfa)
BioMarin Pharmaceutical
2014 - NCT02294877 Australia;Austria;Belgium;Canada;Czechia;Denmark;France;Germany;Ireland;Italy;Malaysia;Netherlands;Poland;Portugal;Puerto Rico;Taiwan;United Kingdom;United States;
Visual field testing
Université de Montréal
2014 - NCT02023086 Canada;
Vitamin D
Shire
2016 Phase 4 NCT02574286 Israel;Spain;United Kingdom;United States;
Vorinostat
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
2014 Phase 1/Phase 2 NCT02124083 United States;
Warfarin
The Cooper Health System
2007 - NCT00683189 United States;
Water
Amicus Therapeutics
2011 Phase 1 NCT01853852 Australia;
Whole body Protein turnover
University of Texas Southwestern Medical Center
2006 - NCT00286689 United States;
ZAVESCA 84CPS
Actelion Registration Ltd
2006 - EUCTR2005-001651-37-IT Czech Republic;Germany;Hungary;Italy;Spain;United Kingdom;
ZESTORIC
Salford Royal NHS Foundation Trust
2009 Phase 4 EUCTR2007-007482-21-GB United Kingdom;
Zavesca
Actelion Pharmaceuticals Ltd
2007 - EUCTR2005-001651-37-HU Czech Republic;Germany;Hungary;Italy;Spain;United Kingdom;
2006 - EUCTR2005-001651-37-ES Czech Republic;Germany;Hungary;Italy;Spain;United Kingdom;
2006 - EUCTR2005-001651-37-CZ Czech Republic;Germany;Hungary;Italy;Spain;United Kingdom;
2005 Phase 4 EUCTR2005-001651-37-GB Czech Republic;Germany;Hungary;Italy;Spain;United Kingdom;
Orphazyme ApS
2015 Phase 1 EUCTR2014-005194-37-ES Germany;Spain;Switzerland;
2015 Phase 1 EUCTR2014-005194-37-DE Germany;Spain;Switzerland;
Zavesca (Miglustat)
Children's Research Institute
2004 Phase 3 NCT00672022 United States;
Zestoretic 10,20
Salford Royal NHS Foundation Trust
2009 Phase 4 EUCTR2007-007482-21-GB United Kingdom;
Zestril
Salford Royal NHS Foundation Trust
2009 Phase 4 EUCTR2007-007482-21-GB United Kingdom;
[14C] AT1001
Amicus Therapeutics
2011 Phase 1 NCT01730482 -
sebelipase alpha
Alexion Pharmaceuitcals Inc
2014 Phase 2 EUCTR2011-004287-30-HR Australia;Belgium;Brazil;Canada;Croatia;Denmark;Germany;Italy;Japan;Mexico;Netherlands;Russian Federation;Spain;Turkey;United Kingdom;United States;
2014 Phase 2 EUCTR2011-004287-30-DE Australia;Belgium;Brazil;Canada;Croatia;Denmark;Germany;Italy;Japan;Mexico;Netherlands;Russian Federation;Spain;Turkey;United Kingdom;United States;
Alexion Pharmaceuticals Inc
2015 Phase 2 EUCTR2014-000533-22-FI Finland;Italy;United Kingdom;United States;
2015 Phase 2 EUCTR2011-004287-30-NL Australia;Belgium;Brazil;Canada;Croatia;Denmark;Germany;Italy;Mexico;Netherlands;Russian Federation;Spain;Turkey;United Kingdom;United States;
2015 Phase 2 EUCTR2011-004287-30-GB Australia;Belgium;Brazil;Canada;Croatia;Denmark;Germany;Italy;Japan;Mexico;Netherlands;Poland;Russian Federation;Spain;Turkey;United Kingdom;United States;
2014 Phase 2 EUCTR2014-000533-22-GB Finland;Italy;United Kingdom;United States;
Alexion Pharmaceuticals inc
2014 Phase 2 EUCTR2011-004287-30-DK Australia;Belgium;Brazil;Canada;Croatia;Denmark;Germany;Italy;Japan;Mexico;Netherlands;Poland;Russian Federation;Spain;Turkey;United Kingdom;United States;
Alexion Pharmaceuticals, Inc.
2015 Phase 3 EUCTR2011-002750-31-CZ Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Japan;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;United States;
2013 Phase 3 EUCTR2011-002750-31-GB Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Japan;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;United States;
2013 Phase 3 EUCTR2011-002750-31-DE Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Japan;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;United States;
- Phase 3 EUCTR2011-002750-31-PL Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Japan;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;United States;
Synageva BioPharma Corp.
2014 Phase 2 EUCTR2014-000533-22-IT Finland;Italy;United Kingdom;United States;
2014 Phase 2 EUCTR2011-004287-30-IT Australia;Belgium;Brazil;Canada;Croatia;Denmark;Germany;Italy;Japan;Mexico;Netherlands;Poland;Russian Federation;Spain;Turkey;United Kingdom;United States;
2014 Phase 2 EUCTR2011-004287-30-BE Australia;Belgium;Brazil;Canada;Croatia;Denmark;Germany;Italy;Japan;Mexico;Netherlands;Russian Federation;Spain;Turkey;United Kingdom;United States;
Synageva BioPharma Corporation
2013 Phase 3 EUCTR2011-002750-31-IT Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;
2013 Phase 3 EUCTR2011-002750-31-HR Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;
2013 Phase 3 EUCTR2011-002750-31-GR Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;
2013 Phase 3 EUCTR2011-002750-31-ES Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;
- Phase 3 EUCTR2011-002750-31-FR Argentina;Australia;Belgium;Brazil;Chile;Croatia;Cyprus;Czech Republic;Denmark;France;Germany;Greece;Israel;Italy;Mexico;Netherlands;Poland;Romania;Russian Federation;South Africa;Spain;Sweden;Taiwan;Turkey;Ukraine;United Kingdom;