256. 筋型糖原病 Disease details / Clinical trials / Drug dev / DR info

1-deoxynojirimycin hydrochloride   
   Amicus Therapeutics, Inc.
      2011   -   EUCTR2011-002154-32-GB   Canada;France;United Kingdom;United States;
      2009   -   EUCTR2008-002302-18-DE   Germany;United Kingdom;
      2008   -   EUCTR2008-002302-18-GB   Germany;United Kingdom;
A-glucosidasi acida umana ricombinante coniugata con multiple copie di bis-mannosio-6- fosfato-tetra-mannosio glicano sintetico (NEOGAA)   
   GENZYME CORPORATION
      2016   Phase 3   EUCTR2016-000942-77-IT   Austria;Belgium;Czech Republic;Denmark;Germany;Italy;Netherlands;Poland;Spain;Sweden;United Kingdom;
AAV2/8LSPhGAA   
   Asklepios Biopharmaceutical, Inc.
      2018   Phase 1/Phase 2   NCT03533673   United States;
ALGLUCOSIDASE ALFA (MYOZYME)   
   Genzyme, a Sanofi Company
      2018   Phase 4   NCT03687333   China;
ALGLUCOSIDASI ALFA UMANA RICOMBINANTE   
   AMICUS THERAPEUTICS, INC.
      2019   Phase 3   EUCTR2018-000755-40-IT   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Democratic People's Republic of;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
AT2220   
   Amicus Therapeutics, Inc.
      2011   -   EUCTR2011-002154-32-GB   Canada;France;United Kingdom;United States;
      2009   -   EUCTR2008-002302-18-DE   Germany;United Kingdom;
      2008   -   EUCTR2008-002302-18-GB   Germany;United Kingdom;
      -   Phase 2   EUCTR2013-002257-30-GB   Netherlands;United Kingdom;United States;
AT2221   
   AMICUS THERAPEUTICS, INC.
      2020   Phase 3   EUCTR2019-000954-67-IT   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Democratic People's Republic of;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
      2019   Phase 3   EUCTR2018-000755-40-IT   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Democratic People's Republic of;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
   Amicus Therapeutics
      2019   Phase 3   NCT04138277   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;New Zealand;Poland;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
      2019   -   NCT04327973   Italy;Taiwan;United States;
      2019   -   NCT03865836   -
      2018   Phase 3   NCT03729362   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;New Zealand;Poland;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
      2016   Phase 1/Phase 2   NCT02675465   Australia;Germany;Netherlands;New Zealand;United Kingdom;United States;
   Amicus Therapeutics, Inc.
      2020   Phase 3   EUCTR2019-000954-67-SI   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
      2020   Phase 3   EUCTR2019-000954-67-GR   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
      2020   Phase 3   EUCTR2019-000954-67-GB   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
      2020   Phase 3   EUCTR2019-000954-67-FR   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
      2020   Phase 3   EUCTR2019-000954-67-ES   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
      2020   Phase 3   EUCTR2019-000954-67-DK   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
      2020   Phase 3   EUCTR2019-000954-67-DE   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
      2020   Phase 3   EUCTR2019-000954-67-BE   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
      2019   Phase 3   EUCTR2018-000755-40-SK   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
      2019   Phase 3   EUCTR2018-000755-40-SI   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
      2019   Phase 3   EUCTR2018-000755-40-SE   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
      2019   Phase 3   EUCTR2018-000755-40-NL   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
      2019   Phase 3   EUCTR2018-000755-40-HU   Australia;Austria;Belgium;Bosnia and Herzegovina;Brazil;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Israel;Italy;Japan;Korea, Republic of;Netherlands;New Zealand;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
      2019   Phase 3   EUCTR2018-000755-40-GR   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
      2019   Phase 3   EUCTR2018-000755-40-GB   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
      2019   Phase 3   EUCTR2018-000755-40-ES   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
      2019   Phase 3   EUCTR2018-000755-40-DK   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
      2019   Phase 3   EUCTR2018-000755-40-DE   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
      2019   Phase 3   EUCTR2018-000755-40-BG   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
      2019   Phase 3   EUCTR2018-000755-40-BE   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
      2019   Phase 3   EUCTR2018-000755-40-AT   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
      2016   Phase 1;Phase 2   EUCTR2015-004798-34-NL   Australia;Germany;Netherlands;New Zealand;United Kingdom;United States;
      2016   Phase 1;Phase 2   EUCTR2015-004798-34-GB   Australia;Germany;Netherlands;United Kingdom;United States;
      2016   Phase 1;Phase 2   EUCTR2015-004798-34-DE   Australia;Germany;Netherlands;United Kingdom;United States;
AT2221 65 mg Formulated Capsules   
   Amicus Therapeutics, Inc.
      2020   Phase 3   EUCTR2019-000954-67-AT   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
AT845   
   Audentes Therapeutics, Inc.
      2020   Phase 1;Phase 2   EUCTR2019-003595-38-GB   Germany;Italy;United Kingdom;
ATB200   
   AMICUS THERAPEUTICS, INC.
      2020   Phase 3   EUCTR2019-000954-67-IT   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Democratic People's Republic of;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
      2019   Phase 3   EUCTR2018-000755-40-IT   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Democratic People's Republic of;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
   Amicus Therapeutics
      2019   Phase 3   NCT04138277   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;New Zealand;Poland;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
      2019   -   NCT04327973   Italy;Taiwan;United States;
      2019   -   NCT03865836   -
      2018   Phase 3   NCT03729362   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;New Zealand;Poland;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
      2016   Phase 1/Phase 2   NCT02675465   Australia;Germany;Netherlands;New Zealand;United Kingdom;United States;
   Amicus Therapeutics, Inc.
      2020   Phase 3   EUCTR2019-000954-67-SI   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
      2020   Phase 3   EUCTR2019-000954-67-GR   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
      2020   Phase 3   EUCTR2019-000954-67-GB   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
      2020   Phase 3   EUCTR2019-000954-67-FR   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
      2020   Phase 3   EUCTR2019-000954-67-ES   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
      2020   Phase 3   EUCTR2019-000954-67-DK   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
      2020   Phase 3   EUCTR2019-000954-67-DE   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
      2020   Phase 3   EUCTR2019-000954-67-BE   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
      2020   Phase 3   EUCTR2019-000954-67-AT   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
      2019   Phase 3   EUCTR2018-000755-40-SK   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
      2019   Phase 3   EUCTR2018-000755-40-SI   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
      2019   Phase 3   EUCTR2018-000755-40-SE   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
      2019   Phase 3   EUCTR2018-000755-40-NL   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
      2019   Phase 3   EUCTR2018-000755-40-HU   Australia;Austria;Belgium;Bosnia and Herzegovina;Brazil;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Israel;Italy;Japan;Korea, Republic of;Netherlands;New Zealand;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
      2019   Phase 3   EUCTR2018-000755-40-GR   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
      2019   Phase 3   EUCTR2018-000755-40-GB   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
      2019   Phase 3   EUCTR2018-000755-40-ES   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
      2019   Phase 3   EUCTR2018-000755-40-DK   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
      2019   Phase 3   EUCTR2018-000755-40-DE   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
      2019   Phase 3   EUCTR2018-000755-40-BG   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
      2019   Phase 3   EUCTR2018-000755-40-BE   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
      2019   Phase 3   EUCTR2018-000755-40-AT   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
      2016   Phase 1;Phase 2   EUCTR2015-004798-34-NL   Australia;Germany;Netherlands;New Zealand;United Kingdom;United States;
      2016   Phase 1;Phase 2   EUCTR2015-004798-34-GB   Australia;Germany;Netherlands;United Kingdom;United States;
      2016   Phase 1;Phase 2   EUCTR2015-004798-34-DE   Australia;Germany;Netherlands;United Kingdom;United States;
Acetaminophen   
   University of Florida
      2017   Phase 1   NCT02240407   United States;
Albuterol   
   Duke University
      2013   Phase 1/Phase 2   NCT01885936   United States;
      2012   Phase 1   NCT01859624   United States;
Aldurazyme (laronidase)   
   University of California, San Francisco
      2021   Phase 1   NCT04532047   United States;
Alglucosidase Alfa (Genetical Recombination)   
   Amicus Therapeutics, Inc./CMIC Co., Ltd.
      2019   Phase 3   JPRN-JapicCTI-194887   Japan, Asia except Japan, North America, South America, Europe, Oceania;
Alglucosidase alfa   
   Amicus Therapeutics
      2018   Phase 3   NCT03729362   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;New Zealand;Poland;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
   Erasmus MC Sophia
      2009   -   EUCTR2008-008293-31-NL   Netherlands;
   GENZYME
      2004   -   EUCTR2004-002168-59-IT   Italy;
   Genzyme Europe B.V.
      2007   -   EUCTR2006-003644-31-DE   France;Germany;Netherlands;
   Genzyme Europe BV
      2007   Phase 4   EUCTR2006-003644-31-FR   France;Germany;Netherlands;
      2007   -   EUCTR2006-003644-31-NL   France;Germany;Netherlands;
      2007   -   EUCTR2005-002759-42-DE   Germany;
      2005   Phase 2   EUCTR2005-002829-31-GB   Belgium;United Kingdom;
      2005   -   EUCTR2005-001629-27-DE   Germany;Italy;
      -   Phase 2   EUCTR2005-002829-31-BE   Belgium;United Kingdom;
   Genzyme, a Sanofi Company
      2015   -   NCT01710813   Belgium;Czech Republic;Czechia;Germany;Italy;Taiwan;United States;
      2014   Phase 4   NCT01410890   Bulgaria;Germany;India;Russian Federation;Ukraine;United Kingdom;United States;
      2012   Phase 4   NCT01597596   Germany;Taiwan;United States;
      2012   Phase 4   NCT01526785   United States;
      2012   -   NCT00566878   United States;
      2008   Phase 4   NCT00486889   United States;
      2007   Phase 4   NCT00483379   Australia;Canada;United States;
      2007   Phase 4   NCT00455195   Australia;Canada;France;Netherlands;United States;
      2005   Phase 3   NCT00158600   France;Netherlands;United States;
      2003   Phase 2   NCT00051935   United States;
      2003   -   NCT00074919   United States;
   Shanghai Children's Medical Center Affiliated to Shanghai Jiao Tong University School of Medicine
      2018   Phase 4 study   ChiCTR1800018514   China;
Alglucosidase alfa (GZ419829)   
   Sanofi
      2016   Phase 3   NCT02782741   Argentina;Australia;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Czechia;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States;
Alglucosidase alfa (recombinant human acid alpha-glucosidase [rhGAA])   
   Genzyme, a Sanofi Company
      2007   -   NCT00520143   United States;
Alglucosidase alfa GZ419829   
   Genzyme, a Sanofi Company
      2017   Phase 2   NCT03019406   France;Japan;Taiwan;United Kingdom;United States;
Alglucosidase alfa GZ419829 (Myozyme)   
   Sanofi
      2021   -   NCT04848779   Belgium;Germany;Italy;Taiwan;
Alglucosidase alpha   
   Genzyme Europe B.V.
      2007   -   EUCTR2006-003644-31-DE   France;Germany;Netherlands;
   Genzyme Europe BV
      2007   Phase 4   EUCTR2006-003644-31-FR   France;Germany;Netherlands;
      2007   -   EUCTR2006-003644-31-NL   France;Germany;Netherlands;
      2007   -   EUCTR2005-002759-42-DE   Germany;
      2005   Phase 2   EUCTR2005-002829-31-GB   Belgium;United Kingdom;
      -   Phase 2   EUCTR2005-002829-31-BE   Belgium;United Kingdom;
Avalglucosidase Alfa (NeoGAA)   
   Genzyme Corporation
      2017   Phase 2   EUCTR2016-003475-21-GB   France;Germany;Japan;Taiwan;United Kingdom;United States;
Avalglucosidase alfa   
   Sanofi
      2021   Phase 3   NCT04910776   Belgium;Germany;Italy;Netherlands;Taiwan;United Kingdom;
Avalglucosidase alfa (GZ402666)   
   Genzyme, a Sanofi Company
      2022   Phase 4   NCT05164055   -
Avalglucosidase alfa GZ402666   
   Genzyme, a Sanofi Company
      2017   Phase 2   NCT03019406   France;Japan;Taiwan;United Kingdom;United States;
Avalglucosidase alfa(GZ402666)   
   Sanofi
      2016   Phase 3   NCT02782741   Argentina;Australia;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Czechia;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States;
Avalglucosidasi alfa   
   SANOFI-AVENTIS RECHERCHE E DEVELOPPEMENT
      2021   Phase 3   EUCTR2020-004686-39-IT   Belgium;China;France;Germany;Italy;Netherlands;Taiwan;United Kingdom;United States;
BMN 701   
   BioMarin Pharmaceutical
      2014   Phase 3   NCT01924845   Belgium;France;Germany;Italy;Netherlands;Portugal;United Kingdom;United States;
      2011   Phase 2   NCT01435772   Australia;France;Germany;New Zealand;United Kingdom;United States;
      2011   Phase 1/Phase 2   NCT01230801   Australia;France;Germany;United Kingdom;United States;
   BioMarin Pharmaceutical Inc
      2016   Phase 3   EUCTR2013-001768-48-AT   Austria;Belgium;France;Germany;Italy;Japan;Netherlands;Portugal;Switzerland;United Kingdom;United States;
      2015   Phase 3   EUCTR2013-001768-48-PT   Argentina;Austria;Belgium;Colombia;France;Germany;Italy;Japan;Netherlands;Portugal;United Kingdom;United States;
      2014   Phase 3   EUCTR2013-001768-48-IT   Argentina;Austria;Belgium;Colombia;France;Germany;Italy;Japan;Netherlands;Portugal;United Kingdom;United States;
      2014   Phase 3   EUCTR2013-001768-48-DE   Argentina;Austria;Belgium;Colombia;France;Germany;Italy;Japan;Netherlands;Portugal;United Kingdom;United States;
      2014   Phase 3   EUCTR2013-001768-48-BE   Argentina;Austria;Belgium;Colombia;France;Germany;Italy;Japan;Netherlands;Portugal;United Kingdom;United States;
      2013   Phase 3   EUCTR2013-001768-48-GB   Austria;Belgium;Colombia;Denmark;France;Germany;Italy;Japan;Netherlands;Portugal;United Kingdom;United States;
      2013   Phase 2   EUCTR2011-001805-28-DE   Australia;Germany;New Zealand;United Kingdom;United States;
      2012   Phase 2   EUCTR2011-001805-28-GB   Australia;France;Germany;United Kingdom;United States;
      2012   -   EUCTR2010-023561-22-DE   France;Germany;Netherlands;United Kingdom;United States;
      2011   Phase 1;Phase 2   EUCTR2010-023561-22-GB   Germany;United Kingdom;United States;
Beta-hydroxybuturate esters   
   Rigshospitalet, Denmark
      2019   -   NCT03945370   Denmark;
Blood draw for optional genetic exome sequencing   
   LMU Klinikum
      2022   -   NCT05272969   Germany;
Bortezomib   
   National Taiwan University Hospital
      2015   Phase 4   NCT02525172   Taiwan;
CYCLOPHOSPHAMIDE MONOHYDRATE   
   Genzyme Corporation Inc
      -   -   EUCTR2015-000583-34-Outside-EU/EEA   Israel;United States;
Calcium   
   LMU Klinikum
      2022   -   NCT05272969   Germany;
Cipaglucosidase alfa   
   Amicus Therapeutics, Inc.
      2020   Phase 3   EUCTR2019-000954-67-SI   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
      2020   Phase 3   EUCTR2019-000954-67-GR   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
      2020   Phase 3   EUCTR2019-000954-67-GB   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
      2020   Phase 3   EUCTR2019-000954-67-FR   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
      2020   Phase 3   EUCTR2019-000954-67-DK   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
      2020   Phase 3   EUCTR2019-000954-67-DE   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
      2020   Phase 3   EUCTR2019-000954-67-BE   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
      2020   Phase 3   EUCTR2019-000954-67-AT   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
      2019   Phase 3   EUCTR2018-000755-40-SI   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
      2019   Phase 3   EUCTR2018-000755-40-SE   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
      2019   Phase 3   EUCTR2018-000755-40-NL   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
      2019   Phase 3   EUCTR2018-000755-40-GR   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
      2019   Phase 3   EUCTR2018-000755-40-DK   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
      2019   Phase 3   EUCTR2018-000755-40-DE   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
      2019   Phase 3   EUCTR2018-000755-40-BE   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
      2019   Phase 3   EUCTR2018-000755-40-AT   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
      2016   Phase 1;Phase 2   EUCTR2015-004798-34-GB   Australia;Germany;Netherlands;United Kingdom;United States;
      2016   Phase 1;Phase 2   EUCTR2015-004798-34-DE   Australia;Germany;Netherlands;United Kingdom;United States;
Cipaglucosidase alfa (ATB200)   
   Amicus Therapeutics
      2022   Phase 3   NCT04808505   United States;
Cipaglucosidasi alfa   
   AMICUS THERAPEUTICS, INC.
      2020   Phase 3   EUCTR2019-000954-67-IT   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Democratic People's Republic of;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
Clenbuterol   
   Duke University
      2023   Phase 2   NCT04094948   United States;
   Dwight Koeberl, M.D., Ph.D.
      2013   Phase 1/Phase 2   NCT01942590   United States;
Creatine   
   LMU Klinikum
      2022   -   NCT05272969   Germany;
Deprakine   
   John Vissing
      2014   Phase 2   EUCTR2014-001637-88-DK   Denmark;
Diphenhydramine   
   University of Florida
      2017   Phase 1   NCT02240407   United States;
Duvoglustat   
   Amicus Therapeutics
      2011   Phase 2   NCT01380743   Canada;France;United Kingdom;United States;
      2008   Phase 2   NCT00688597   Australia;Canada;France;Germany;Netherlands;United Kingdom;United States;
Duvoglustat HCl   
   Amicus Therapeutics, Inc.
      -   Phase 2   EUCTR2013-002257-30-GB   Netherlands;United Kingdom;United States;
Duvoglustat hydrochloride   
   Amicus Therapeutics, Inc.
      2011   -   EUCTR2011-002154-32-GB   Canada;France;United Kingdom;United States;
      -   Phase 2   EUCTR2013-002257-30-GB   Netherlands;United Kingdom;United States;
Enzyme Replacement Agent   
   Fundació Institut de Recerca de l'Hospital de la Santa Creu i Sant Pau
      2017   -   NCT03045042   Spain;
Epilim Chrono 200 Controlled Released tablets   
   University College London
      2014   Phase 2   EUCTR2012-002933-12-GB   Denmark;United Kingdom;United States;
Epilim Chrono 300 Controlled Released tablets   
   University College London
      2014   Phase 2   EUCTR2012-002933-12-GB   Denmark;United Kingdom;United States;
Epilim Chrono 500 Controlled Released tablets   
   University College London
      2014   Phase 2   EUCTR2012-002933-12-GB   Denmark;United Kingdom;United States;
Filgrastim   
   Serhat Gumrukcu, MD PhD
      2022   -   NCT04476550   United States;
Folic (or folinic) acid   
   GlaxoSmithKline
      2019   Phase 3   NCT03980483   Argentina;Canada;China;Czechia;Hungary;India;Italy;Latvia;Lithuania;Malaysia;Mexico;Poland;Russian Federation;Serbia;South Africa;Spain;Ukraine;United Kingdom;United States;
GILT-rhGAA   
   BioMarin Pharmaceutical Inc
      2016   Phase 3   EUCTR2013-001768-48-AT   Austria;Belgium;France;Germany;Italy;Japan;Netherlands;Portugal;Switzerland;United Kingdom;United States;
      2015   Phase 3   EUCTR2013-001768-48-PT   Argentina;Austria;Belgium;Colombia;France;Germany;Italy;Japan;Netherlands;Portugal;United Kingdom;United States;
      2014   Phase 3   EUCTR2013-001768-48-IT   Argentina;Austria;Belgium;Colombia;France;Germany;Italy;Japan;Netherlands;Portugal;United Kingdom;United States;
      2014   Phase 3   EUCTR2013-001768-48-DE   Argentina;Austria;Belgium;Colombia;France;Germany;Italy;Japan;Netherlands;Portugal;United Kingdom;United States;
      2014   Phase 3   EUCTR2013-001768-48-BE   Argentina;Austria;Belgium;Colombia;France;Germany;Italy;Japan;Netherlands;Portugal;United Kingdom;United States;
      2013   Phase 3   EUCTR2013-001768-48-GB   Austria;Belgium;Colombia;Denmark;France;Germany;Italy;Japan;Netherlands;Portugal;United Kingdom;United States;
      2013   Phase 2   EUCTR2011-001805-28-DE   Australia;Germany;New Zealand;United Kingdom;United States;
      2012   Phase 2   EUCTR2011-001805-28-GB   Australia;France;Germany;United Kingdom;United States;
      2012   -   EUCTR2010-023561-22-DE   France;Germany;Netherlands;United Kingdom;United States;
      2011   Phase 1;Phase 2   EUCTR2010-023561-22-GB   Germany;United Kingdom;United States;
GSK3196165 (Otilimab)   
   GlaxoSmithKline
      2019   Phase 3   NCT03980483   Argentina;Canada;China;Czechia;Hungary;India;Italy;Latvia;Lithuania;Malaysia;Mexico;Poland;Russian Federation;Serbia;South Africa;Spain;Ukraine;United Kingdom;United States;
GZ402666   
   GENZYME CORPORATION
      2016   Phase 3   EUCTR2016-000942-77-IT   Austria;Belgium;Czech Republic;Denmark;Germany;Italy;Netherlands;Poland;Spain;Sweden;United Kingdom;
   Genzyme Corporation
      2019   Phase 3   EUCTR2016-000942-77-HU   Argentina;Australia;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States;
      2018   Phase 3   EUCTR2016-000942-77-PT   Argentina;Australia;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Czechia;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States;
      2018   Phase 3   EUCTR2016-000942-77-BG   Argentina;Australia;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Czechia;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States;
      2017   Phase 3   EUCTR2016-000942-77-NL   Argentina;Australia;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Czechia;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States;
      2017   Phase 3   EUCTR2016-000942-77-AT   Argentina;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Czechia;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States;
      2017   Phase 2   EUCTR2016-003475-21-GB   France;Germany;Japan;Taiwan;United Kingdom;United States;
      2016   Phase 3   EUCTR2016-000942-77-SE   Argentina;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States;
      2016   Phase 3   EUCTR2016-000942-77-GB   Argentina;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States;
      2016   Phase 3   EUCTR2016-000942-77-ES   Argentina;Austria;Belgium;Brazil;Canada;Colombia;Czech Republic;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States;
      2016   Phase 3   EUCTR2016-000942-77-DK   Argentina;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States;
      2016   Phase 3   EUCTR2016-000942-77-DE   Argentina;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Czechia;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States;
      2016   Phase 3   EUCTR2016-000942-77-CZ   Argentina;Australia;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States;
      2016   Phase 3   EUCTR2016-000942-77-BE   Argentina;Austria;Belgium;Brazil;Canada;Colombia;Czech Republic;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States;
      2014   Phase 2   EUCTR2013-003321-28-NL   Belgium;Denmark;France;Germany;Italy;Netherlands;United Kingdom;United States;
      2014   Phase 2   EUCTR2013-003321-28-DK   Belgium;Denmark;France;Germany;Italy;Netherlands;United Kingdom;United States;
      2014   Phase 2   EUCTR2013-003321-28-DE   Belgium;Denmark;France;Germany;Italy;Netherlands;United Kingdom;United States;
      2014   Phase 2   EUCTR2013-003321-28-BE   Belgium;Denmark;France;Germany;Italy;Netherlands;United Kingdom;United States;
      2014   -   EUCTR2013-003321-28-FR   Belgium;Denmark;France;Germany;Italy;Netherlands;United Kingdom;United States;
      -   Phase 3   EUCTR2016-000942-77-PL   Argentina;Australia;Austria;Belgium;Bulgaria;Canada;Colombia;Czech Republic;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States;
      -   Phase 2   EUCTR2016-003475-21-FR   France;Germany;Japan;Taiwan;United Kingdom;United States;
      -   Phase 2   EUCTR2016-003475-21-DE   France;Germany;Japan;Taiwan;United Kingdom;United States;
   Genzyme, a Sanofi Company
      2014   Phase 2/Phase 3   NCT02032524   Belgium;Denmark;France;Germany;Netherlands;United Kingdom;United States;
      2013   Phase 1   NCT01898364   Belgium;Denmark;France;Germany;Italy;Netherlands;United Kingdom;United States;
   SANOFI-AVENTIS RECHERCHE E DEVELOPPEMENT
      2021   Phase 3   EUCTR2020-004686-39-IT   Belgium;China;France;Germany;Italy;Netherlands;Taiwan;United Kingdom;United States;
   Sanofi-Aventis Recherche & Développement
      2021   Phase 3   EUCTR2020-004686-39-NL   Belgium;China;France;Germany;Italy;Netherlands;Taiwan;United Kingdom;United States;
      -   Phase 4   EUCTR2021-002590-26-FR   France;
      -   Phase 3   EUCTR2020-004686-39-DE   Belgium;China;France;Germany;Italy;Netherlands;Taiwan;United Kingdom;United States;
      -   Phase 3   EUCTR2020-004686-39-BE   Belgium;China;France;Germany;Italy;Netherlands;Taiwan;United Kingdom;United States;
GZ419829   
   Sanofi(China) Investment Co Ltd, Shanghai Branch
      -   Phase 4   EUCTR2021-004047-25-Outside-EU/EEA   China;
Genetic test: ACE polymorphism   
   LMU Klinikum
      2022   -   NCT05272969   Germany;
Genetic test: ACTN3 polymorphism   
   LMU Klinikum
      2022   -   NCT05272969   Germany;
Glucosidase alfa   
   Klinikum der Universitaet Muenchen
      2016   -   NCT02824068   Argentina;Brazil;Germany;Italy;Taiwan;
Glycosade   
   John Mitchell
      2013   -   NCT02054832   Canada;
Intravenous immune globulin   
   National Taiwan University Hospital
      2015   Phase 4   NCT02525172   Taiwan;
Ketocal 4:1 liquid Nutricia (intervention)   
   Rigshospitalet, Denmark
      2019   -   NCT04044508   Denmark;United Kingdom;
LMX 4 Topical Cream   
   University of Florida
      2017   Phase 1   NCT02240407   United States;
Lidocaine   
   University of Florida
      2017   Phase 1   NCT02240407   United States;
Low carbohydrate ketogenic diet   
   IRCCS Eugenio Medea
      2019   -   NCT04292938   Italy;
Lumizyme®   
   Genzyme Corporation Inc
      -   -   EUCTR2015-000512-18-Outside-EU/EEA   United States;
METHOTREXATE SODIUM   
   Genzyme Corporation Inc
      -   -   EUCTR2015-000584-14-Outside-EU/EEA   United States;
      -   -   EUCTR2015-000583-34-Outside-EU/EEA   Israel;United States;
MYOZYME®   
   GENZYME CORPORATION
      2016   Phase 3   EUCTR2016-000942-77-IT   Austria;Belgium;Czech Republic;Denmark;Germany;Italy;Netherlands;Poland;Spain;Sweden;United Kingdom;
   Genzyme Corporation
      2019   Phase 3   EUCTR2016-000942-77-HU   Argentina;Australia;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States;
      2018   Phase 3   EUCTR2016-000942-77-PT   Argentina;Australia;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Czechia;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States;
      2018   Phase 3   EUCTR2016-000942-77-BG   Argentina;Australia;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Czechia;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States;
      2017   Phase 3   EUCTR2016-000942-77-NL   Argentina;Australia;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Czechia;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States;
      2017   Phase 3   EUCTR2016-000942-77-AT   Argentina;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Czechia;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States;
      2017   Phase 2   EUCTR2016-003475-21-GB   France;Germany;Japan;Taiwan;United Kingdom;United States;
      2016   Phase 3   EUCTR2016-000942-77-SE   Argentina;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States;
      2016   Phase 3   EUCTR2016-000942-77-GB   Argentina;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States;
      2016   Phase 3   EUCTR2016-000942-77-ES   Argentina;Austria;Belgium;Brazil;Canada;Colombia;Czech Republic;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States;
      2016   Phase 3   EUCTR2016-000942-77-DK   Argentina;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States;
      2016   Phase 3   EUCTR2016-000942-77-DE   Argentina;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Czechia;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States;
      2016   Phase 3   EUCTR2016-000942-77-CZ   Argentina;Australia;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States;
      2016   Phase 3   EUCTR2016-000942-77-BE   Argentina;Austria;Belgium;Brazil;Canada;Colombia;Czech Republic;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States;
      -   Phase 3   EUCTR2016-000942-77-PL   Argentina;Australia;Austria;Belgium;Bulgaria;Canada;Colombia;Czech Republic;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States;
      -   Phase 2   EUCTR2016-003475-21-FR   France;Germany;Japan;Taiwan;United Kingdom;United States;
      -   Phase 2   EUCTR2016-003475-21-DE   France;Germany;Japan;Taiwan;United Kingdom;United States;
   Sanofi(China) Investment Co Ltd, Shanghai Branch
      -   Phase 4   EUCTR2021-004047-25-Outside-EU/EEA   China;
Magnesium   
   LMU Klinikum
      2022   -   NCT05272969   Germany;
Methotrexate   
   Genzyme, a Sanofi Company
      2009   Phase 4   NCT00701129   Israel;United States;
   GlaxoSmithKline
      2019   Phase 3   NCT03980483   Argentina;Canada;China;Czechia;Hungary;India;Italy;Latvia;Lithuania;Malaysia;Mexico;Poland;Russian Federation;Serbia;South Africa;Spain;Ukraine;United Kingdom;United States;
   National Taiwan University Hospital
      2015   Phase 4   NCT02525172   Taiwan;
Miglustat   
   Amicus Therapeutics
      2020   Phase 3   NCT03911505   Canada;Japan;Taiwan;United States;
   UNIVERSITA` DEGLI STUDI DI NAPOLI FEDERICO II
      2011   -   EUCTR2010-024647-32-IT   Italy;
   University of Florida
      2008   -   NCT01451879   United States;
Miglustat (AT2221)   
   Amicus Therapeutics
      2022   Phase 3   NCT04808505   United States;
   Amicus Therapeutics, Inc./CMIC Co., Ltd.
      2019   Phase 3   JPRN-JapicCTI-194887   Japan, Asia except Japan, North America, South America, Europe, Oceania;
Myozyme   
   AMICUS THERAPEUTICS, INC.
      2019   Phase 3   EUCTR2018-000755-40-IT   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Democratic People's Republic of;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
   Amicus Therapeutics, Inc.
      2019   Phase 3   EUCTR2018-000755-40-SK   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
      2019   Phase 3   EUCTR2018-000755-40-SI   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
      2019   Phase 3   EUCTR2018-000755-40-SE   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
      2019   Phase 3   EUCTR2018-000755-40-NL   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
      2019   Phase 3   EUCTR2018-000755-40-HU   Australia;Austria;Belgium;Bosnia and Herzegovina;Brazil;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Israel;Italy;Japan;Korea, Republic of;Netherlands;New Zealand;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
      2019   Phase 3   EUCTR2018-000755-40-GR   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
      2019   Phase 3   EUCTR2018-000755-40-GB   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
      2019   Phase 3   EUCTR2018-000755-40-ES   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
      2019   Phase 3   EUCTR2018-000755-40-DK   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
      2019   Phase 3   EUCTR2018-000755-40-DE   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
      2019   Phase 3   EUCTR2018-000755-40-BG   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
      2019   Phase 3   EUCTR2018-000755-40-BE   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
      2019   Phase 3   EUCTR2018-000755-40-AT   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
      -   Phase 2   EUCTR2013-002257-30-GB   Netherlands;United Kingdom;United States;
   GENZYME
      2005   -   EUCTR2005-001629-27-IT   Germany;Italy;
   Genzyme Corporation
      2017   Phase 4   EUCTR2010-022231-11-GB   Bulgaria;Germany;India;Russian Federation;Ukraine;United Kingdom;United States;
      2012   Phase 1;Phase 4   EUCTR2010-022231-11-DE   Bulgaria;China;Germany;India;Poland;Russian Federation;Ukraine;United Kingdom;United States;
      -   Phase 4   EUCTR2021-005552-11-Outside-EU/EEA   United States;
   Genzyme Corporation, a Sanofi company
      2013   -   EUCTR2011-005595-42-DE   Canada;Germany;India;Russian Federation;Taiwan;Turkey;United States;
   Genzyme Europe B.V.
      2012   -   EUCTR2010-020611-36-NL   Denmark;Germany;Netherlands;United Kingdom;United States;
      2011   -   EUCTR2010-020611-36-GB   Germany;Netherlands;United Kingdom;United States;
      2011   -   EUCTR2010-020611-36-DE   Germany;Netherlands;United Kingdom;United States;
      2007   -   EUCTR2006-003644-31-DE   France;Germany;Netherlands;
   Genzyme Europe BV
      2007   Phase 4   EUCTR2006-003644-31-FR   France;Germany;Netherlands;
      2007   -   EUCTR2006-003644-31-NL   France;Germany;Netherlands;
      2007   -   EUCTR2005-002759-42-DE   Germany;
      2005   Phase 2   EUCTR2005-002829-31-GB   Belgium;United Kingdom;
      2005   -   EUCTR2005-001629-27-DE   Germany;Italy;
      -   Phase 2   EUCTR2005-002829-31-BE   Belgium;United Kingdom;
   Genzyme, a Sanofi Company
      2005   Phase 3   NCT00268944   France;
      2005   Phase 2/Phase 3   NCT00125879   France;Germany;Israel;Italy;Netherlands;Taiwan;United States;
      2005   Phase 2   NCT00250939   Netherlands;
      2004   -   NCT00074932   United States;
      2003   Phase 2/Phase 3   NCT00059280   France;Israel;Taiwan;United Kingdom;United States;
      2003   Phase 2   NCT00765414   United States;
      2003   Phase 2   NCT00763932   France;South Africa;United States;
      2003   Phase 1/Phase 2   NCT00053573   France;Israel;United Kingdom;United States;
   Valerion Therapeutics, LLC
      2017   Phase 1;Phase 2   EUCTR2016-004578-16-GB   United Kingdom;United States;
Myozyme 50 mg powder for concentrate for solution for infusion   
   Genzyme Corporation Inc
      -   -   EUCTR2015-000582-31-Outside-EU/EEA   Australia;Canada;United States;
Myozyme®   
   Genzyme Corporation
      2019   Phase 3   EUCTR2016-000942-77-HU   Argentina;Australia;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States;
      2018   Phase 3   EUCTR2016-000942-77-PT   Argentina;Australia;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Czechia;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States;
      2018   Phase 3   EUCTR2016-000942-77-BG   Argentina;Australia;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Czechia;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States;
      2017   Phase 3   EUCTR2016-000942-77-NL   Argentina;Australia;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Czechia;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States;
      2017   Phase 3   EUCTR2016-000942-77-AT   Argentina;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Czechia;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States;
      2017   Phase 2   EUCTR2016-003475-21-GB   France;Germany;Japan;Taiwan;United Kingdom;United States;
      2016   Phase 3   EUCTR2016-000942-77-SE   Argentina;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States;
      2016   Phase 3   EUCTR2016-000942-77-GB   Argentina;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States;
      2016   Phase 3   EUCTR2016-000942-77-ES   Argentina;Austria;Belgium;Brazil;Canada;Colombia;Czech Republic;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States;
      2016   Phase 3   EUCTR2016-000942-77-DK   Argentina;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States;
      2016   Phase 3   EUCTR2016-000942-77-DE   Argentina;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Czechia;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States;
      2016   Phase 3   EUCTR2016-000942-77-CZ   Argentina;Australia;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States;
      2016   Phase 3   EUCTR2016-000942-77-BE   Argentina;Austria;Belgium;Brazil;Canada;Colombia;Czech Republic;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States;
      -   Phase 3   EUCTR2016-000942-77-PL   Argentina;Australia;Austria;Belgium;Bulgaria;Canada;Colombia;Czech Republic;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States;
      -   Phase 2   EUCTR2016-003475-21-FR   France;Germany;Japan;Taiwan;United Kingdom;United States;
      -   Phase 2   EUCTR2016-003475-21-DE   France;Germany;Japan;Taiwan;United Kingdom;United States;
   Genzyme Corporation Inc
      -   -   EUCTR2015-000584-14-Outside-EU/EEA   United States;
      -   -   EUCTR2015-000583-34-Outside-EU/EEA   Israel;United States;
Myozyme® (alglucosidase alfa)   
   Genzyme, a Sanofi Company
      2008   Phase 4   NCT00701701   Canada;Israel;United States;
NEO-GAA   
   Genzyme Corporation
      2017   Phase 3   EUCTR2016-000942-77-NL   Argentina;Australia;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Czechia;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States;
NeoGAA   
   Genzyme Corporation
      2014   Phase 2   EUCTR2013-003321-28-NL   Belgium;Denmark;France;Germany;Italy;Netherlands;United Kingdom;United States;
      2014   -   EUCTR2013-003321-28-FR   Belgium;Denmark;France;Germany;Italy;Netherlands;United Kingdom;United States;
NeoGAA or avalglucosidase alfa   
   Genzyme Corporation
      2018   Phase 3   EUCTR2016-000942-77-PT   Argentina;Australia;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Czechia;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States;
      2017   Phase 3   EUCTR2016-000942-77-AT   Argentina;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Czechia;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States;
      2016   Phase 3   EUCTR2016-000942-77-GB   Argentina;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States;
      2016   Phase 3   EUCTR2016-000942-77-DE   Argentina;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Czechia;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States;
      -   Phase 3   EUCTR2016-000942-77-PL   Argentina;Australia;Austria;Belgium;Bulgaria;Canada;Colombia;Czech Republic;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States;
Non applicabile   
   AMICUS THERAPEUTICS, INC.
      2019   Phase 3   EUCTR2018-000755-40-IT   Argentina;Australia;Austria;Belgium;Bosnia and Herzegovina;Bulgaria;Canada;Denmark;France;Germany;Greece;Hungary;Italy;Japan;Korea, Democratic People's Republic of;Korea, Republic of;Netherlands;Poland;Romania;Slovakia;Slovenia;Spain;Sweden;Taiwan;United Kingdom;United States;
Phosphate   
   LMU Klinikum
      2022   -   NCT05272969   Germany;
Plecebo   
   Amicus Therapeutics, Inc./CMIC Co., Ltd.
      2019   Phase 3   JPRN-JapicCTI-194887   Japan, Asia except Japan, North America, South America, Europe, Oceania;
Pyridostigmine Bromide   
   University of Florida
      2015   Early Phase 1   NCT02357225   United States;
RAAV1-CMV-GAA (study agent) Administration   
   University of Florida
      2010   Phase 1/Phase 2   NCT00976352   United States;
RECOMBINANT HUMAN ?-GLUCOSIDASE CONJUGATED WITH SYNTHETIC BISMANNOSE-6-PHOSPHATE-MAN6 GLYCAN   
   Genzyme Corporation
      2014   Phase 2   EUCTR2013-003321-28-NL   Belgium;Denmark;France;Germany;Italy;Netherlands;United Kingdom;United States;
      2014   Phase 2   EUCTR2013-003321-28-DK   Belgium;Denmark;France;Germany;Italy;Netherlands;United Kingdom;United States;
      2014   Phase 2   EUCTR2013-003321-28-DE   Belgium;Denmark;France;Germany;Italy;Netherlands;United Kingdom;United States;
      2014   Phase 2   EUCTR2013-003321-28-BE   Belgium;Denmark;France;Germany;Italy;Netherlands;United Kingdom;United States;
      2014   -   EUCTR2013-003321-28-FR   Belgium;Denmark;France;Germany;Italy;Netherlands;United Kingdom;United States;
   Sanofi-Aventis Recherche & Développement
      2021   Phase 3   EUCTR2020-004686-39-NL   Belgium;China;France;Germany;Italy;Netherlands;Taiwan;United Kingdom;United States;
      -   Phase 4   EUCTR2021-002590-26-FR   France;
      -   Phase 3   EUCTR2020-004686-39-DE   Belgium;China;France;Germany;Italy;Netherlands;Taiwan;United Kingdom;United States;
      -   Phase 3   EUCTR2020-004686-39-BE   Belgium;China;France;Germany;Italy;Netherlands;Taiwan;United Kingdom;United States;
RECOMBINANT HUMAN ALFA-GLUCOSIDASE CONJUGATED WITH SYNTHETIC BISMANNOSE-6-PHOSPHATE-MAN6 GLYCAN   
   SANOFI-AVENTIS RECHERCHE E DEVELOPPEMENT
      2021   Phase 3   EUCTR2020-004686-39-IT   Belgium;China;France;Germany;Italy;Netherlands;Taiwan;United Kingdom;United States;
Rapamycin   
   University of Florida
      2017   Phase 1   NCT02240407   United States;
Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase   
   University of Florida
      2017   Phase 1   NCT02240407   United States;
Recombinant human a-glucosidase conjugated with multiple copies of synthetic bis-mannose-6-phosphate-tetra-mannose glycan (NEOGAA)   
   Genzyme Corporation
      2019   Phase 3   EUCTR2016-000942-77-HU   Argentina;Australia;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States;
      2018   Phase 3   EUCTR2016-000942-77-PT   Argentina;Australia;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Czechia;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States;
      2018   Phase 3   EUCTR2016-000942-77-BG   Argentina;Australia;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Czechia;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States;
      2017   Phase 3   EUCTR2016-000942-77-NL   Argentina;Australia;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Czechia;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States;
      2017   Phase 3   EUCTR2016-000942-77-AT   Argentina;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Czechia;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States;
      2016   Phase 3   EUCTR2016-000942-77-SE   Argentina;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States;
      2016   Phase 3   EUCTR2016-000942-77-GB   Argentina;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States;
      2016   Phase 3   EUCTR2016-000942-77-ES   Argentina;Austria;Belgium;Brazil;Canada;Colombia;Czech Republic;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States;
      2016   Phase 3   EUCTR2016-000942-77-DK   Argentina;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States;
      2016   Phase 3   EUCTR2016-000942-77-DE   Argentina;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Czechia;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States;
      2016   Phase 3   EUCTR2016-000942-77-CZ   Argentina;Australia;Austria;Belgium;Brazil;Bulgaria;Canada;Colombia;Czech Republic;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States;
      2016   Phase 3   EUCTR2016-000942-77-BE   Argentina;Austria;Belgium;Brazil;Canada;Colombia;Czech Republic;Denmark;France;Germany;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States;
      -   Phase 3   EUCTR2016-000942-77-PL   Argentina;Australia;Austria;Belgium;Bulgaria;Canada;Colombia;Czech Republic;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Mexico;Netherlands;Poland;Portugal;Russian Federation;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States;
      -   Phase 2   EUCTR2016-003475-21-FR   France;Germany;Japan;Taiwan;United Kingdom;United States;
      -   Phase 2   EUCTR2016-003475-21-DE   France;Germany;Japan;Taiwan;United Kingdom;United States;
Recombinant human a-glucosidase conjugated with multiple copies of synthetic bis-mannose-6-phosphate-tetra-mannose glycan Avalglucosidase Alfa (NeoGAA)   
   Genzyme Corporation
      2017   Phase 2   EUCTR2016-003475-21-GB   France;Germany;Japan;Taiwan;United Kingdom;United States;
Recombinant human acid alfa-glucosidase (ATB200)   
   Amicus Therapeutics, Inc./CMIC Co., Ltd.
      2019   Phase 3   JPRN-JapicCTI-194887   Japan, Asia except Japan, North America, South America, Europe, Oceania;
Recombinant human acid alfa-glucosidase (rhGAA)   
   GENZYME
      2004   -   EUCTR2004-002168-59-IT   Italy;
Recombinant human acid alpha-glucosidase (rhGAA)   
   Genzyme, a Sanofi Company
      2001   Phase 2   NCT00025896   United States;
Reveglucosidase alfa   
   BioMarin Pharmaceutical Inc
      2013   Phase 3   EUCTR2013-001768-48-GB   Austria;Belgium;Colombia;Denmark;France;Germany;Italy;Japan;Netherlands;Portugal;United Kingdom;United States;
Reveglucosidase alpha   
   BioMarin Pharmaceutical Inc
      2016   Phase 3   EUCTR2013-001768-48-AT   Austria;Belgium;France;Germany;Italy;Japan;Netherlands;Portugal;Switzerland;United Kingdom;United States;
      2014   Phase 3   EUCTR2013-001768-48-DE   Argentina;Austria;Belgium;Colombia;France;Germany;Italy;Japan;Netherlands;Portugal;United Kingdom;United States;
      2014   Phase 3   EUCTR2013-001768-48-BE   Argentina;Austria;Belgium;Colombia;France;Germany;Italy;Japan;Netherlands;Portugal;United Kingdom;United States;
RhGAA   
   Amicus Therapeutics
      2011   Phase 2   NCT01380743   Canada;France;United Kingdom;United States;
   Genzyme Corporation
      2017   Phase 4   EUCTR2010-022231-11-GB   Bulgaria;Germany;India;Russian Federation;Ukraine;United Kingdom;United States;
      2012   Phase 1;Phase 4   EUCTR2010-022231-11-DE   Bulgaria;China;Germany;India;Poland;Russian Federation;Ukraine;United Kingdom;United States;
   Genzyme Corporation Inc
      -   -   EUCTR2015-000512-18-Outside-EU/EEA   United States;
   Genzyme Corporation, a Sanofi company
      2013   -   EUCTR2011-005595-42-DE   Canada;Germany;India;Russian Federation;Taiwan;Turkey;United States;
   Genzyme Europe B.V.
      2012   -   EUCTR2010-020611-36-NL   Denmark;Germany;Netherlands;United Kingdom;United States;
      2011   -   EUCTR2010-020611-36-GB   Germany;Netherlands;United Kingdom;United States;
      2011   -   EUCTR2010-020611-36-DE   Germany;Netherlands;United Kingdom;United States;
   Valerion Therapeutics, LLC
      2017   Phase 1;Phase 2   EUCTR2016-004578-16-GB   United Kingdom;United States;
      2017   Phase 1/Phase 2   NCT02898753   United Kingdom;United States;
RhGAA, acid alpha glucosidase   
   Genzyme Europe B.V.
      2007   -   EUCTR2006-003644-31-DE   France;Germany;Netherlands;
   Genzyme Europe BV
      2007   Phase 4   EUCTR2006-003644-31-FR   France;Germany;Netherlands;
      2007   -   EUCTR2006-003644-31-NL   France;Germany;Netherlands;
      2005   Phase 2   EUCTR2005-002829-31-GB   Belgium;United Kingdom;
      -   Phase 2   EUCTR2005-002829-31-BE   Belgium;United Kingdom;
Rituxan   
   University of Florida
      2017   Phase 1   NCT02240407   United States;
Rituximab   
   Genzyme, a Sanofi Company
      2009   Phase 4   NCT00701129   Israel;United States;
   National Taiwan University Hospital
      2015   Phase 4   NCT02525172   Taiwan;
   University of Florida
      2008   -   NCT01451879   United States;
SODIUM VALPROATE   
   John Vissing
      2014   Phase 2   EUCTR2014-001637-88-DK   Denmark;
SPK-3006   
   Spark Therapeutics
      2021   Phase 1;Phase 2   EUCTR2019-001283-30-NL   France;Germany;Italy;Netherlands;United Kingdom;United States;
      2021   Phase 1;Phase 2   EUCTR2019-001283-30-DK   Denmark;France;Germany;Italy;Netherlands;Spain;United Kingdom;United States;
      2020   Phase 1;Phase 2   EUCTR2019-001283-30-FR   France;Germany;Italy;Netherlands;United Kingdom;United States;
      2020   Phase 1;Phase 2   EUCTR2019-001283-30-DE   Denmark;France;Germany;Italy;Netherlands;Spain;United Kingdom;United States;
Salbutamol   
   National Taiwan University Hospital
      2013   Phase 4   NCT02405598   Taiwan;
Sodium Valproate   
   University College, London
      2015   Phase 2   NCT03112889   Denmark;United Kingdom;
Sodium valproate   
   University College London
      2014   Phase 2   EUCTR2012-002933-12-GB   Denmark;United Kingdom;United States;
Ss-hydroxybuturate esters   
   Rigshospitalet, Denmark
      2019   -   NCT03945370   Denmark;
Tofacitinib   
   GlaxoSmithKline
      2019   Phase 3   NCT03980483   Argentina;Canada;China;Czechia;Hungary;India;Italy;Latvia;Lithuania;Malaysia;Mexico;Poland;Russian Federation;Serbia;South Africa;Spain;Ukraine;United Kingdom;United States;
Triheptanoin   
   Institut National de la Santé Et de la Recherche Médicale, France
      2016   Phase 2   NCT02919631   -
   Rigshospitalet, Denmark
      2018   Phase 2   NCT03642860   Denmark;
      2015   Phase 2   NCT02432768   Denmark;
UX007   
   Copenhagen Neuromuscular Center
      2018   Phase 2   EUCTR2017-004153-17-DK   Denmark;
   Neuromuscular Research Unit, Rigshospitalet
      2015   Phase 2   EUCTR2014-003644-12-DK   Denmark;
VAL-1221   
   Valerion Therapeutics, LLC
      2017   Phase 1;Phase 2   EUCTR2016-004578-16-GB   United Kingdom;United States;
      2017   Phase 1/Phase 2   NCT02898753   United Kingdom;United States;
Valproic acid   
   University College London
      2014   Phase 2   EUCTR2012-002933-12-GB   Denmark;United Kingdom;United States;
Vitamin D   
   LMU Klinikum
      2022   -   NCT05272969   Germany;
Zavesca® Prescription   
   University of Florida
      2016   Phase 1   NCT02185651   United States;