19. ライソゾーム病 Disease details / Clinical trials / Drug dev / DR 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;