3. 脊髄性筋萎縮症 Disease details / Clinical trials / Drug dev / DR info

臨床試験数 : 237 / 薬物数 : 123 - (DrugBank : 29) / 標的遺伝子数 : 51 - 標的パスウェイ数 : 75

薬物ごとの開発者（Primary Sponsor）、臨床試験情報（抜粋）です。
0000   
   BIOGEN IDEC RESEARCH LIMITED
      -   Phase 3   EUCTR2015-001870-16-IT   Australia;Belgium;Canada;France;Germany;Hong Kong;Italy;Japan;Korea, Republic of;Spain;Sweden;Taiwan;Turkey;United Kingdom;United States;
2011-2012 seasonal flu vaccine Intramuscular   
   Children's Hospital Medical Center, Cincinnati
      2011   Phase 4   NCT01422200   United States;
2011-2012 seasonal flu vaccine Subcutaneous   
   Children's Hospital Medical Center, Cincinnati
      2011   Phase 4   NCT01422200   United States;
3,4-Diaminopyridine Phosphate   
   CATALYST PHARMACEUTICALS INC.
      2018   Phase 2   EUCTR2017-004600-22-IT   Italy;
4-cholesten-3-one, oxime   
   TROPHOS SA
      2011   Phase 2   EUCTR2010-020386-24-GB   Belgium;France;Germany;Italy;Netherlands;United Kingdom;
      2010   Phase 2   EUCTR2010-020386-24-DE   Belgium;Germany;Italy;Netherlands;United Kingdom;
      2010   Phase 2   EUCTR2010-020386-24-BE   Belgium;Germany;Italy;Netherlands;United Kingdom;
      2010   -   EUCTR2010-020386-24-NL   Belgium;Germany;Italy;Netherlands;United Kingdom;
      -   Phase 2   EUCTR2010-020386-24-FR   Belgium;France;Germany;Italy;Netherlands;United Kingdom;
4-cholesten-3-one,oxime   
   TROPHOS
      2007   -   EUCTR2006-006845-14-FR   France;
AVXS-101   
   AVEXIS, INC.
      2020   Phase 3   EUCTR2019-002611-26-IT   Australia;Belgium;Canada;France;Italy;Japan;Korea, Republic of;Spain;Taiwan;United Kingdom;United States;
   AveXis, Inc.
      2020   Phase 3   EUCTR2019-002611-26-GB   Australia;Belgium;Canada;France;Italy;Japan;Korea, Republic of;Spain;Taiwan;United Kingdom;United States;
      2020   Phase 3   EUCTR2019-002611-26-FR   Australia;Belgium;Canada;France;Italy;Japan;Korea, Republic of;Spain;Taiwan;United Kingdom;United States;
      2020   Phase 3   EUCTR2019-002611-26-ES   Australia;Belgium;Canada;France;Italy;Japan;Korea, Republic of;Spain;Taiwan;United Kingdom;United States;
   Novartis Gene Therapies
      2014   Phase 1   NCT02122952   United States;
   Novartis Gene Therapies, Inc.
      -   Phase 3   EUCTR2019-002611-26-BE   Australia;Belgium;Canada;France;Italy;Japan;Spain;Taiwan;United Kingdom;United States;
AVXS-101 (previously known as scAAV9.CB.SMN)   
   AVEXIS, INC.
      2018   Phase 3   EUCTR2017-000266-29-IT   Belgium;France;Germany;Italy;Netherlands;Spain;Sweden;United Kingdom;
   AveXis, Inc.
      2019   Phase 3   EUCTR2017-004087-35-NL   Australia;Belgium;Canada;Germany;Israel;Italy;Japan;Korea, Democratic People's Republic of;Netherlands;Spain;Taiwan;United Kingdom;United States;
      2019   Phase 3   EUCTR2017-004087-35-BE   Australia;Belgium;Canada;Germany;Israel;Italy;Japan;Korea, Democratic People's Republic of;Netherlands;Spain;Taiwan;United Kingdom;United States;
      2019   Phase 3   EUCTR2017-000266-29-NL   Belgium;France;Germany;Italy;Netherlands;Spain;Sweden;United Kingdom;
      2019   Phase 3   EUCTR2017-000266-29-BE   Belgium;France;Italy;Netherlands;Spain;Sweden;United Kingdom;
      2018   Phase 3   EUCTR2017-004087-35-IT   Australia;Belgium;Canada;Germany;Israel;Italy;Japan;Korea, Democratic People's Republic of;Netherlands;Spain;Taiwan;United Kingdom;United States;
      2018   Phase 3   EUCTR2017-004087-35-ES   Australia;Belgium;Canada;Germany;Israel;Italy;Japan;Korea, Democratic People's Republic of;Netherlands;Spain;Taiwan;United Kingdom;United States;
      2018   Phase 3   EUCTR2017-000266-29-GB   Belgium;France;Italy;Netherlands;Spain;Sweden;United Kingdom;
      2018   Phase 3   EUCTR2017-000266-29-ES   Belgium;France;Germany;Italy;Netherlands;Spain;Sweden;United Kingdom;
      2018   Phase 3   EUCTR2017-000266-29-DE   Belgium;France;Germany;Italy;Netherlands;Spain;Sweden;United Kingdom;
      -   Phase 3   EUCTR2017-004087-35-DE   Australia;Belgium;Canada;Germany;Israel;Italy;Japan;Korea, Democratic People's Republic of;Netherlands;Spain;Taiwan;United Kingdom;United States;
      -   Phase 3   EUCTR2017-000266-29-SE   Belgium;France;Germany;Italy;Netherlands;Spain;Sweden;United Kingdom;
      -   Phase 3   EUCTR2017-000266-29-FR   Belgium;France;Germany;Italy;Netherlands;Spain;Sweden;United Kingdom;
   Novartis Gene Therapies, Inc.
      2018   Phase 3   EUCTR2017-004087-35-GB   Australia;Belgium;Canada;Germany;Israel;Italy;Japan;Korea, Democratic People's Republic of;Netherlands;Spain;Taiwan;United Kingdom;United States;
Adipose derived mesenchymal stem cell   
   Tehran University of Medical Sciences
      2015   Phase 1/Phase 2   NCT02855112   Iran, Islamic Republic of;
Air stacking manuever   
   Centro Universitário Augusto Motta
      2011   -   NCT01522079   -
Amifampridina fosfato   
   CATALYST PHARMACEUTICALS INC.
      2018   Phase 2   EUCTR2018-000160-28-IT   Italy;
      2018   Phase 2   EUCTR2017-004600-22-IT   Italy;
Amifampridine Phosphate   
   Catalyst Pharmaceuticals, Inc.
      2019   Phase 2   NCT03781479   Italy;
Amifampridine Phosphate 10 MG Oral Tablet   
   Catalyst Pharmaceuticals, Inc.
      2019   Phase 2   NCT03819660   Italy;
Apitegromab   
   Scholar Rock, Inc.
      2019   Phase 2   EUCTR2018-004383-65-NL   Germany;Italy;Netherlands;Spain;United States;
      -   Phase 3   EUCTR2021-005314-34-NL   Belgium;France;Germany;Italy;Netherlands;Poland;Spain;United Kingdom;United States;
      -   Phase 3   EUCTR2021-005314-34-BE   Belgium;France;Germany;Italy;Netherlands;Poland;Spain;United Kingdom;United States;
Biocarn   
   University of Utah
      2009   -   EUCTR2008-003915-11-DE   Germany;
Blood draw for optional genetic exome sequencing   
   LMU Klinikum
      2022   -   NCT05272969   Germany;
Branaplam   
   Novartis Pharma Services AG
      2014   Phase 1;Phase 2   EUCTR2014-002053-19-BE   Belgium;Bulgaria;Czech Republic;Denmark;Germany;Hungary;Italy;Netherlands;Poland;Russian Federation;United States;
      -   Phase 1;Phase 2   EUCTR2014-002053-19-PL   Belgium;Bulgaria;Czech Republic;Denmark;Germany;Hungary;Italy;Netherlands;Poland;Russian Federation;United States;
   Novartis Pharmaceuticals
      2015   Phase 1/Phase 2   NCT02268552   Belgium;Bulgaria;Czechia;Denmark;Germany;Hungary;Italy;Netherlands;Poland;Russian Federation;
Calcium   
   LMU Klinikum
      2022   -   NCT05272969   Germany;
Celecoxib   
   Hugh McMillan
      2019   Phase 2   NCT02876094   Canada;
Creatine   
   LMU Klinikum
      2022   -   NCT05272969   Germany;
Evrysdi   
   F. HOFFMANN - LA ROCHE LTD.
      2022   Phase 2;Phase 3   EUCTR2021-003417-19-IT   Belgium;Italy;Netherlands;Poland;United Kingdom;United States;
   F.Hoffmann-La Roche Ltd
      2022   Phase 2;Phase 3   EUCTR2021-003417-19-PL   Belgium;Italy;Netherlands;Poland;United Kingdom;United States;
      -   Phase 2;Phase 3   EUCTR2021-003417-19-NL   Belgium;Italy;Netherlands;Poland;United Kingdom;United States;
      -   Phase 2;Phase 3   EUCTR2021-003417-19-BE   Belgium;Italy;Netherlands;Poland;United Kingdom;United States;
FIRDAPSE - 10 MG - COMPRESSE - USO ORALE - BLISTER(ALU/PVC/PVDC) 100 X 1 COMPRESSE   
   CATALYST PHARMACEUTICALS INC.
      2018   Phase 2   EUCTR2018-000160-28-IT   Italy;
      2018   Phase 2   EUCTR2017-004600-22-IT   Italy;
GH   
   Klinik Neuropädiatrie und Muskelkrankheiten
      2006   -   EUCTR2005-002822-78-DE   Germany;
GYM329   
   F.Hoffmann-La Roche Ltd
      2022   Phase 2;Phase 3   EUCTR2021-003417-19-PL   Belgium;Italy;Netherlands;Poland;United Kingdom;United States;
      -   Phase 2;Phase 3   EUCTR2021-003417-19-NL   Belgium;Italy;Netherlands;Poland;United Kingdom;United States;
      -   Phase 2;Phase 3   EUCTR2021-003417-19-BE   Belgium;Italy;Netherlands;Poland;United Kingdom;United States;
Genetic test: ACE polymorphism   
   LMU Klinikum
      2022   -   NCT05272969   Germany;
Genetic test: ACTN3 polymorphism   
   LMU Klinikum
      2022   -   NCT05272969   Germany;
HUMAN ANTI-PROMYOSTATIN MONOCLONAL ANTIBODY   
   Scholar Rock, Inc.
      2019   Phase 2   EUCTR2018-004383-65-NL   Germany;Italy;Netherlands;Spain;United States;
      2019   Phase 2   EUCTR2018-004383-65-ES   Canada;Germany;Italy;Netherlands;Spain;United States;
      2019   Phase 2   EUCTR2018-004383-65-DE   Germany;Italy;Netherlands;Spain;United States;
Hydroxyurea   
   Kaohsiung Medical University Chung-Ho Memorial Hospital
      2007   Phase 2/Phase 3   NCT00485511   Taiwan;
   Stanford University
      2004   Phase 1/Phase 2   NCT00568802   United States;
      2004   Phase 1/Phase 2   NCT00568698   United States;
ISIS 396443   
   BIOGEN IDEC RESEARCH LIMITED
      -   Phase 3   EUCTR2015-001870-16-IT   Australia;Belgium;Canada;France;Germany;Hong Kong;Italy;Japan;Korea, Republic of;Spain;Sweden;Taiwan;Turkey;United Kingdom;United States;
   Biogen Idec Research Limited
      2016   Phase 3   EUCTR2015-001870-16-SE   Australia;Belgium;Canada;France;Germany;Hong Kong;Italy;Japan;Korea, Republic of;Spain;Sweden;Turkey;United Kingdom;United States;
      2016   Phase 3   EUCTR2015-001870-16-GB   Australia;Belgium;Canada;France;Germany;Hong Kong;Italy;Japan;Korea, Republic of;Spain;Sweden;Turkey;United Kingdom;United States;
      2016   Phase 3   EUCTR2015-001870-16-DE   Australia;Belgium;Canada;France;Germany;Hong Kong;Italy;Japan;Korea, Republic of;Spain;Sweden;Turkey;United Kingdom;United States;
   Ionis Pharmaceuticals, Inc.
      2016   -   EUCTR2015-001870-16-ES   Australia;Belgium;Canada;France;Germany;Hong Kong;Italy;Japan;Korea, Republic of;Spain;Sweden;Taiwan;Turkey;United Kingdom;United States;
      2015   Phase 3   EUCTR2014-001947-18-SE   Canada;France;Germany;Hong Kong;Italy;Japan;Korea, Republic of;Spain;Sweden;United Kingdom;United States;
      2015   Phase 3   EUCTR2014-001947-18-DE   Canada;France;Germany;Hong Kong;Italy;Japan;Korea, Republic of;Spain;Sweden;United Kingdom;United States;
      2015   Phase 3   EUCTR2013-004422-29-DE   Australia;Belgium;Canada;France;Germany;Hong Kong;Italy;Japan;Korea, Republic of;Spain;Sweden;Taiwan;Turkey;United Kingdom;United States;
      2015   Phase 3   EUCTR2013-004422-29-BE   Australia;Belgium;Canada;France;Germany;Hong Kong;Italy;Japan;Korea, Republic of;Spain;Sweden;Taiwan;United Kingdom;United States;
      2014   Phase 3   EUCTR2013-004422-29-SE   Australia;Belgium;Canada;France;Germany;Hong Kong;Italy;Japan;Korea, Republic of;Spain;Sweden;Taiwan;Turkey;United Kingdom;United States;
      2014   Phase 3   EUCTR2013-004422-29-GB   Australia;Belgium;Canada;France;Germany;Hong Kong;Italy;Japan;Korea, Republic of;Spain;Sweden;Taiwan;Turkey;United Kingdom;United States;
      -   Phase 3   EUCTR2015-001870-16-FR   Australia;Belgium;Canada;France;Germany;Hong Kong;Italy;Japan;Korea, Republic of;Spain;Sweden;Taiwan;Turkey;United Kingdom;United States;
      -   Phase 2   EUCTR2017-000621-12-Outside-EU/EEA   Canada;United States;
      -   Phase 2   EUCTR2017-000327-27-Outside-EU/EEA   United States;
   Isis Pharmaceuticals, Inc.
      2015   Phase 3   EUCTR2014-001947-18-IT   Canada;France;Germany;Hong Kong;Italy;Japan;Korea, Republic of;Spain;Sweden;United Kingdom;United States;
      2015   Phase 3   EUCTR2014-001947-18-ES   Canada;France;Germany;Hong Kong;Italy;Japan;Korea, Republic of;Spain;Sweden;United Kingdom;United States;
      2015   Phase 3   EUCTR2013-004422-29-ES   Australia;Belgium;Canada;France;Germany;Hong Kong;Italy;Japan;Korea, Republic of;Spain;Sweden;Taiwan;United Kingdom;United States;
      2014   Phase 3   EUCTR2013-004422-29-IT   Australia;Belgium;Canada;France;Germany;Hong Kong;Italy;Japan;Korea, Republic of;Spain;Sweden;Taiwan;United Kingdom;United States;
      -   Phase 3   EUCTR2014-001947-18-GB   Canada;France;Germany;Hong Kong;Italy;Japan;Korea, Republic of;Spain;Sweden;United Kingdom;United States;
      -   Phase 3   EUCTR2014-001947-18-FR   Canada;France;Germany;Hong Kong;Italy;Japan;Korea, Republic of;Spain;Sweden;United Kingdom;United States;
ISIS 396443 (BIIB058)   
   Biogen Idec Research Limited
      2016   Phase 3   EUCTR2015-001870-16-BE   Australia;Belgium;Canada;France;Germany;Hong Kong;Italy;Japan;Korea, Republic of;Spain;Sweden;Turkey;United Kingdom;United States;
      2015   Phase 2   EUCTR2014-003657-33-GB   Germany;United Kingdom;United States;
      2015   Phase 2   EUCTR2014-003657-33-DE   Germany;United Kingdom;United States;
      2015   Phase 2   EUCTR2014-002098-12-GB   Australia;Germany;Italy;Qatar;Taiwan;Turkey;United Kingdom;United States;
      2015   Phase 2   EUCTR2014-002098-12-DE   Australia;Canada;Germany;Italy;Qatar;Taiwan;Turkey;United Kingdom;United States;
      2015   -   EUCTR2014-002098-12-IT   Argentina;Australia;Germany;Israel;Italy;Qatar;Taiwan;Turkey;United Kingdom;United States;
ISIS 396443, BIIB058   
   BIOGEN IDEC RESEARCH LIMITED
      2020   Phase 2;Phase 3   EUCTR2019-002663-10-IT   Argentina;Australia;Brazil;Canada;Chile;Colombia;Estonia;France;Germany;Greece;Hungary;Ireland;Israel;Italy;Korea, Republic of;Latvia;Lebanon;Mexico;Poland;Russian Federation;Saudi Arabia;Spain;Taiwan;Turkey;United Kingdom;United States;
   Biogen Idec Research Limited
      2021   Phase 3   EUCTR2020-004708-32-HU   Argentina;Australia;Brazil;Canada;Chile;Colombia;Estonia;France;Germany;Greece;Hungary;Ireland;Israel;Italy;Korea, Republic of;Latvia;Lebanon;Mexico;Poland;Russian Federation;Saudi Arabia;Spain;Taiwan;Turkey;United States;
      2021   Phase 3   EUCTR2020-004708-32-ES   Argentina;Australia;Brazil;Canada;Chile;Colombia;Estonia;France;Germany;Greece;Hungary;Ireland;Israel;Italy;Korea, Republic of;Latvia;Lebanon;Mexico;Poland;Russian Federation;Saudi Arabia;Spain;Taiwan;Turkey;United States;
      2021   Phase 2;Phase 3   EUCTR2019-002663-10-DE   Argentina;Australia;Brazil;Canada;Chile;Colombia;Estonia;France;Germany;Greece;Hungary;Ireland;Israel;Italy;Korea, Republic of;Latvia;Lebanon;Mexico;Poland;Russian Federation;Saudi Arabia;Spain;Taiwan;Turkey;United Kingdom;United States;
      2020   Phase 2;Phase 3   EUCTR2019-002663-10-LV   Argentina;Australia;Brazil;Canada;Chile;Colombia;Estonia;Hungary;Ireland;Italy;Korea, Republic of;Latvia;Lebanon;Mexico;Poland;Russian Federation;Saudi Arabia;Spain;United States;
      2020   Phase 2;Phase 3   EUCTR2019-002663-10-HU   Argentina;Australia;Brazil;Canada;Chile;Colombia;Estonia;Hungary;Ireland;Italy;Korea, Republic of;Latvia;Lebanon;Mexico;Poland;Russian Federation;Saudi Arabia;Spain;United States;
      2020   Phase 2;Phase 3   EUCTR2019-002663-10-GB   Argentina;Australia;Brazil;Canada;Chile;Colombia;Estonia;Germany;Greece;Hungary;Ireland;Israel;Italy;Korea, Republic of;Latvia;Lebanon;Mexico;Poland;Russian Federation;Saudi Arabia;Spain;Taiwan;Turkey;United Kingdom;United States;
      2020   Phase 2;Phase 3   EUCTR2019-002663-10-FR   Argentina;Australia;Brazil;Canada;Chile;Colombia;Estonia;France;Germany;Greece;Hungary;Ireland;Israel;Italy;Korea, Republic of;Latvia;Lebanon;Mexico;Poland;Russian Federation;Saudi Arabia;Spain;Taiwan;Turkey;United Kingdom;United States;
      2020   Phase 2;Phase 3   EUCTR2019-002663-10-ES   Argentina;Australia;Brazil;Canada;Chile;Colombia;Estonia;Greece;Hungary;Ireland;Italy;Korea, Republic of;Latvia;Lebanon;Mexico;Poland;Russian Federation;Saudi Arabia;Spain;United States;
      -   Phase 3   EUCTR2021-001294-23-DE   Australia;Belgium;Brazil;Canada;Germany;Japan;Poland;Spain;Switzerland;
      -   Phase 3   EUCTR2021-001294-23-BE   Australia;Belgium;Brazil;Canada;Germany;Japan;Poland;Spain;Switzerland;
      -   Phase 3   EUCTR2020-004708-32-PL   Australia;Brazil;Canada;Chile;China;Colombia;Estonia;France;Germany;Greece;Hungary;Ireland;Israel;Italy;Japan;Korea, Republic of;Latvia;Lebanon;Mexico;Netherlands;Poland;Russian Federation;Saudi Arabia;Spain;Taiwan;Turkey;United Kingdom;United States;
      -   Phase 3   EUCTR2020-004708-32-EE   Australia;Brazil;Canada;Chile;China;Colombia;Estonia;France;Germany;Greece;Hungary;Ireland;Israel;Italy;Japan;Korea, Republic of;Latvia;Lebanon;Mexico;Netherlands;Poland;Russian Federation;Saudi Arabia;Spain;Taiwan;Turkey;United Kingdom;United States;
      -   Phase 3   EUCTR2020-004708-32-DE   Australia;Brazil;Canada;Chile;China;Colombia;Estonia;France;Germany;Greece;Hungary;Ireland;Israel;Italy;Japan;Korea, Republic of;Latvia;Lebanon;Mexico;Netherlands;Poland;Russian Federation;Saudi Arabia;Spain;Taiwan;Turkey;United Kingdom;United States;
      -   Phase 2;Phase 3   EUCTR2019-002663-10-PL   Australia;Brazil;Canada;Chile;China;Colombia;Estonia;France;Germany;Greece;Hungary;Ireland;Israel;Italy;Japan;Korea, Republic of;Latvia;Lebanon;Mexico;Netherlands;Poland;Russian Federation;Saudi Arabia;Spain;Taiwan;Turkey;United Kingdom;United States;
      -   Phase 2;Phase 3   EUCTR2019-002663-10-NL   Australia;Brazil;Canada;Chile;China;Colombia;Estonia;France;Germany;Greece;Hungary;Ireland;Israel;Italy;Japan;Korea, Republic of;Latvia;Lebanon;Mexico;Netherlands;Poland;Russian Federation;Saudi Arabia;Spain;Taiwan;Turkey;United Kingdom;United States;
      -   Phase 2;Phase 3   EUCTR2019-002663-10-IE   Argentina;Australia;Brazil;Canada;Chile;China;Colombia;Estonia;France;Germany;Greece;Hungary;Ireland;Israel;Italy;Korea, Republic of;Latvia;Lebanon;Mexico;Netherlands;Poland;Russian Federation;Saudi Arabia;Spain;Taiwan;Turkey;United Kingdom;United States;
      -   Phase 2;Phase 3   EUCTR2019-002663-10-GR   Argentina;Australia;Brazil;Canada;Chile;Colombia;Estonia;France;Germany;Greece;Hungary;Ireland;Israel;Italy;Korea, Republic of;Latvia;Lebanon;Mexico;Poland;Russian Federation;Saudi Arabia;Spain;Taiwan;Turkey;United States;
Itraconazole   
   Hoffmann-La Roche
      2016   Phase 1   NCT02633709   Netherlands;
LMI070   
   NOVARTIS FARMA S.p.A.
      2015   Phase 1;Phase 2   EUCTR2014-002053-19-IT   Belgium;Bulgaria;Czech Republic;Denmark;European Union;Germany;Hungary;Italy;Netherlands;Poland;United States;
   Novartis Pharma Services AG
      2018   Phase 1;Phase 2   EUCTR2014-002053-19-HU   Belgium;Bulgaria;Czech Republic;Denmark;Germany;Hungary;Italy;Netherlands;Poland;Russian Federation;United States;
      2018   Phase 1;Phase 2   EUCTR2014-002053-19-CZ   Belgium;Bulgaria;Czech Republic;Denmark;Germany;Hungary;Italy;Netherlands;Poland;Russian Federation;United States;
      2018   Phase 1;Phase 2   EUCTR2014-002053-19-BG   Belgium;Bulgaria;Czech Republic;Denmark;Germany;Hungary;Italy;Netherlands;Poland;Russian Federation;United States;
      2015   Phase 1;Phase 2   EUCTR2014-002053-19-DE   Belgium;Bulgaria;Czech Republic;Denmark;Germany;Hungary;Italy;Netherlands;Poland;
      2014   Phase 1;Phase 2   EUCTR2014-002053-19-DK   Belgium;Bulgaria;Czech Republic;Denmark;Germany;Hungary;Italy;Netherlands;Poland;Russian Federation;United States;
      2014   Phase 1;Phase 2   EUCTR2014-002053-19-BE   Belgium;Bulgaria;Czech Republic;Denmark;Germany;Hungary;Italy;Netherlands;Poland;Russian Federation;United States;
      -   Phase 1;Phase 2   EUCTR2014-002053-19-PL   Belgium;Bulgaria;Czech Republic;Denmark;Germany;Hungary;Italy;Netherlands;Poland;Russian Federation;United States;
      -   Phase 1;Phase 2   EUCTR2014-002053-19-NL   Belgium;Denmark;Germany;Italy;Netherlands;United States;
Leuprolide   
   National Center for Research Resources (NCRR)
      1992   Phase 2   NCT00004771   -
Magnesium   
   LMU Klinikum
      2022   -   NCT05272969   Germany;
Mestinon   
   Universtiy Medical Center Utrecht
      2015   Phase 2   EUCTR2011-004369-34-NL   Netherlands;
Midazolam   
   Hoffmann-La Roche
      2019   Phase 1   NCT03988907   United States;
NUSINERSEN SODIUM   
   BIOGEN IDEC RESEARCH LIMITED
      2021   Phase 4   EUCTR2020-003492-18-IT   Germany;Israel;Italy;Spain;United States;
      2020   Phase 2;Phase 3   EUCTR2019-002663-10-IT   Argentina;Australia;Brazil;Canada;Chile;Colombia;Estonia;France;Germany;Greece;Hungary;Ireland;Israel;Italy;Korea, Republic of;Latvia;Lebanon;Mexico;Poland;Russian Federation;Saudi Arabia;Spain;Taiwan;Turkey;United Kingdom;United States;
   Biogen Idec Research Limited
      2021   Phase 4   EUCTR2020-003492-18-ES   Germany;Israel;Italy;Spain;United States;
      2021   Phase 4   EUCTR2020-003492-18-DE   Germany;Israel;Italy;Spain;United States;
      2021   Phase 3   EUCTR2020-004708-32-HU   Argentina;Australia;Brazil;Canada;Chile;Colombia;Estonia;France;Germany;Greece;Hungary;Ireland;Israel;Italy;Korea, Republic of;Latvia;Lebanon;Mexico;Poland;Russian Federation;Saudi Arabia;Spain;Taiwan;Turkey;United States;
      2021   Phase 3   EUCTR2020-004708-32-ES   Argentina;Australia;Brazil;Canada;Chile;Colombia;Estonia;France;Germany;Greece;Hungary;Ireland;Israel;Italy;Korea, Republic of;Latvia;Lebanon;Mexico;Poland;Russian Federation;Saudi Arabia;Spain;Taiwan;Turkey;United States;
      2021   Phase 2;Phase 3   EUCTR2019-002663-10-DE   Argentina;Australia;Brazil;Canada;Chile;Colombia;Estonia;France;Germany;Greece;Hungary;Ireland;Israel;Italy;Korea, Republic of;Latvia;Lebanon;Mexico;Poland;Russian Federation;Saudi Arabia;Spain;Taiwan;Turkey;United Kingdom;United States;
      2020   Phase 2;Phase 3   EUCTR2019-002663-10-LV   Argentina;Australia;Brazil;Canada;Chile;Colombia;Estonia;Hungary;Ireland;Italy;Korea, Republic of;Latvia;Lebanon;Mexico;Poland;Russian Federation;Saudi Arabia;Spain;United States;
      2020   Phase 2;Phase 3   EUCTR2019-002663-10-HU   Argentina;Australia;Brazil;Canada;Chile;Colombia;Estonia;Hungary;Ireland;Italy;Korea, Republic of;Latvia;Lebanon;Mexico;Poland;Russian Federation;Saudi Arabia;Spain;United States;
      2020   Phase 2;Phase 3   EUCTR2019-002663-10-GB   Argentina;Australia;Brazil;Canada;Chile;Colombia;Estonia;Germany;Greece;Hungary;Ireland;Israel;Italy;Korea, Republic of;Latvia;Lebanon;Mexico;Poland;Russian Federation;Saudi Arabia;Spain;Taiwan;Turkey;United Kingdom;United States;
      2020   Phase 2;Phase 3   EUCTR2019-002663-10-FR   Argentina;Australia;Brazil;Canada;Chile;Colombia;Estonia;France;Germany;Greece;Hungary;Ireland;Israel;Italy;Korea, Republic of;Latvia;Lebanon;Mexico;Poland;Russian Federation;Saudi Arabia;Spain;Taiwan;Turkey;United Kingdom;United States;
      2020   Phase 2;Phase 3   EUCTR2019-002663-10-ES   Argentina;Australia;Brazil;Canada;Chile;Colombia;Estonia;Greece;Hungary;Ireland;Italy;Korea, Republic of;Latvia;Lebanon;Mexico;Poland;Russian Federation;Saudi Arabia;Spain;United States;
      -   Phase 3   EUCTR2021-001294-23-DE   Australia;Belgium;Brazil;Canada;Germany;Japan;Poland;Spain;Switzerland;
      -   Phase 3   EUCTR2021-001294-23-BE   Australia;Belgium;Brazil;Canada;Germany;Japan;Poland;Spain;Switzerland;
      -   Phase 3   EUCTR2020-004708-32-PL   Australia;Brazil;Canada;Chile;China;Colombia;Estonia;France;Germany;Greece;Hungary;Ireland;Israel;Italy;Japan;Korea, Republic of;Latvia;Lebanon;Mexico;Netherlands;Poland;Russian Federation;Saudi Arabia;Spain;Taiwan;Turkey;United Kingdom;United States;
      -   Phase 3   EUCTR2020-004708-32-EE   Australia;Brazil;Canada;Chile;China;Colombia;Estonia;France;Germany;Greece;Hungary;Ireland;Israel;Italy;Japan;Korea, Republic of;Latvia;Lebanon;Mexico;Netherlands;Poland;Russian Federation;Saudi Arabia;Spain;Taiwan;Turkey;United Kingdom;United States;
      -   Phase 3   EUCTR2020-004708-32-DE   Australia;Brazil;Canada;Chile;China;Colombia;Estonia;France;Germany;Greece;Hungary;Ireland;Israel;Italy;Japan;Korea, Republic of;Latvia;Lebanon;Mexico;Netherlands;Poland;Russian Federation;Saudi Arabia;Spain;Taiwan;Turkey;United Kingdom;United States;
      -   Phase 2;Phase 3   EUCTR2019-002663-10-PL   Australia;Brazil;Canada;Chile;China;Colombia;Estonia;France;Germany;Greece;Hungary;Ireland;Israel;Italy;Japan;Korea, Republic of;Latvia;Lebanon;Mexico;Netherlands;Poland;Russian Federation;Saudi Arabia;Spain;Taiwan;Turkey;United Kingdom;United States;
      -   Phase 2;Phase 3   EUCTR2019-002663-10-NL   Australia;Brazil;Canada;Chile;China;Colombia;Estonia;France;Germany;Greece;Hungary;Ireland;Israel;Italy;Japan;Korea, Republic of;Latvia;Lebanon;Mexico;Netherlands;Poland;Russian Federation;Saudi Arabia;Spain;Taiwan;Turkey;United Kingdom;United States;
      -   Phase 2;Phase 3   EUCTR2019-002663-10-IE   Argentina;Australia;Brazil;Canada;Chile;China;Colombia;Estonia;France;Germany;Greece;Hungary;Ireland;Israel;Italy;Korea, Republic of;Latvia;Lebanon;Mexico;Netherlands;Poland;Russian Federation;Saudi Arabia;Spain;Taiwan;Turkey;United Kingdom;United States;
      -   Phase 2;Phase 3   EUCTR2019-002663-10-GR   Argentina;Australia;Brazil;Canada;Chile;Colombia;Estonia;France;Germany;Greece;Hungary;Ireland;Israel;Italy;Korea, Republic of;Latvia;Lebanon;Mexico;Poland;Russian Federation;Saudi Arabia;Spain;Taiwan;Turkey;United States;
Nerve Growth Factor   
   PLA General Hospital
      2010   -   ChiCTR-TRC-10001093   China;
Norditropin SimpleXx 15 mg/1.5 ml   
   Klinik Neuropädiatrie und Muskelkrankheiten
      2006   -   EUCTR2005-002822-78-DE   Germany;
Nursinersen   
   Biogen Idec Research Limited
      2020   Phase 2;Phase 3   EUCTR2019-002663-10-GB   Argentina;Australia;Brazil;Canada;Chile;Colombia;Estonia;Germany;Greece;Hungary;Ireland;Israel;Italy;Korea, Republic of;Latvia;Lebanon;Mexico;Poland;Russian Federation;Saudi Arabia;Spain;Taiwan;Turkey;United Kingdom;United States;
Nusinersen   
   Berger Zdenek
      2021   Phase 2-3   JPRN-jRCT2061200040   Colombia;Estonia;Ireland;Japan;Latvia;Spain;Taiwan;United States;
   Biogen
      2022   Phase 3   NCT05067790   United States;
      2021   Phase 4   NCT04488133   Germany;Israel;Italy;Spain;United States;
      2021   Phase 3   NCT04729907   Argentina;Australia;Brazil;Canada;Chile;Colombia;Estonia;France;Germany;Greece;Hungary;Ireland;Israel;Italy;Korea, Republic of;Latvia;Lebanon;Mexico;Poland;Russian Federation;Saudi Arabia;Spain;Taiwan;Turkey;United Kingdom;United States;
      2020   Phase 2/Phase 3   NCT04089566   Australia;Brazil;Canada;Chile;Colombia;Estonia;France;Germany;Greece;Hungary;Ireland;Israel;Italy;Japan;Korea, Republic of;Latvia;Lebanon;Mexico;Poland;Russian Federation;Saudi Arabia;Spain;Taiwan;United Kingdom;United States;
      2016   -   NCT02865109   Australia;Austria;Belgium;Canada;China;Colombia;Denmark;Finland;France;Germany;Greece;Ireland;Israel;Italy;Korea, Republic of;Mexico;Netherlands;New Zealand;Norway;Poland;Portugal;Slovenia;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States;
      2015   Phase 3   NCT02594124   Australia;Belgium;Canada;France;Germany;Hong Kong;Italy;Japan;Korea, Republic of;Spain;Sweden;Taiwan;Turkey;United Kingdom;United States;
      2015   Phase 2   NCT02462759   Germany;United States;
      2015   Phase 2   NCT02386553   Argentina;Australia;Canada;Germany;Israel;Italy;Qatar;Taiwan;Turkey;United Kingdom;United States;
      2014   Phase 3   NCT02292537   Canada;France;Germany;Hong Kong;Italy;Japan;Korea, Republic of;Spain;Sweden;United Kingdom;United States;
      2014   Phase 3   NCT02193074   Australia;Belgium;Canada;France;Germany;Hong Kong;Italy;Japan;Korea, Republic of;Spain;Sweden;Taiwan;Turkey;United Kingdom;United States;
      2014   Phase 1   NCT02052791   United States;
      2013   Phase 2   NCT01839656   Canada;United States;
      2013   Phase 1   NCT01780246   United States;
      2012   Phase 1/Phase 2   NCT01703988   United States;
      2011   Phase 1   NCT01494701   United States;
   Biogen Idec Research Limited
      2016   Phase 3   EUCTR2015-001870-16-SE   Australia;Belgium;Canada;France;Germany;Hong Kong;Italy;Japan;Korea, Republic of;Spain;Sweden;Turkey;United Kingdom;United States;
      2016   Phase 3   EUCTR2015-001870-16-GB   Australia;Belgium;Canada;France;Germany;Hong Kong;Italy;Japan;Korea, Republic of;Spain;Sweden;Turkey;United Kingdom;United States;
      2016   Phase 3   EUCTR2015-001870-16-DE   Australia;Belgium;Canada;France;Germany;Hong Kong;Italy;Japan;Korea, Republic of;Spain;Sweden;Turkey;United Kingdom;United States;
      2016   Phase 3   EUCTR2015-001870-16-BE   Australia;Belgium;Canada;France;Germany;Hong Kong;Italy;Japan;Korea, Republic of;Spain;Sweden;Turkey;United Kingdom;United States;
      2015   Phase 2   EUCTR2014-003657-33-DE   Germany;United Kingdom;United States;
      2015   Phase 2   EUCTR2014-002098-12-GB   Australia;Germany;Italy;Qatar;Taiwan;Turkey;United Kingdom;United States;
      2015   Phase 2   EUCTR2014-002098-12-DE   Australia;Canada;Germany;Italy;Qatar;Taiwan;Turkey;United Kingdom;United States;
   CHU de Reims
      2020   -   NCT04576494   France;
   Ionis Pharmaceuticals, Inc.
      -   Phase 2   EUCTR2017-000327-27-Outside-EU/EEA   United States;
   Northwell Health
      2019   -   NCT03878030   United States;
   Ohio State University
      2018   -   NCT04591678   United States;
   University Medical Centre Ljubljana
      2017   -   NCT04825119   Slovenia;
      2017   -   NCT04587492   Slovenia;
Nusinersen Injectable Product   
   Hospital Israelita Albert Einstein
      2020   -   NCT04404764   Brazil;
Nusinersen Treatments   
   NYU Langone Health
      2019   Early Phase 1   NCT04050852   United States;
OAV101   
   NOVARTIS PHARMA AG
      2021   Phase 3   EUCTR2020-005995-37-IT   Australia;Belgium;Canada;France;Germany;Italy;Portugal;Spain;Switzerland;Taiwan;United Kingdom;United States;
   Novartis Pharma AG
      2021   Phase 3   EUCTR2020-005995-37-PT   Australia;Belgium;Canada;France;Germany;Italy;Portugal;Spain;Switzerland;Taiwan;United Kingdom;United States;
      2021   Phase 3   EUCTR2020-005995-37-FR   Australia;Belgium;Canada;France;Germany;Italy;Portugal;Spain;Switzerland;Taiwan;United Kingdom;United States;
      2021   Phase 3   EUCTR2020-005995-37-BE   Australia;Belgium;Canada;France;Germany;Italy;Portugal;Spain;Switzerland;Taiwan;United Kingdom;United States;
      -   Phase 3   EUCTR2021-003474-31-DK   Brazil;China;Colombia;Denmark;Egypt;India;Italy;Malaysia;Mexico;Russian Federation;Saudi Arabia;Singapore;South Africa;Taiwan;Thailand;United Arab Emirates;United States;Vietnam;
Okrido 6mg/mL oral solution   
   Novartis Pharma AG
      -   Phase 3   EUCTR2021-003474-31-DK   Brazil;China;Colombia;Denmark;Egypt;India;Italy;Malaysia;Mexico;Russian Federation;Saudi Arabia;Singapore;South Africa;Taiwan;Thailand;United Arab Emirates;United States;Vietnam;
Olesoxime   
   Hoffmann-La Roche
      2016   Phase 2   NCT02628743   Belgium;France;Germany;Italy;Netherlands;Poland;United Kingdom;
      2010   Phase 2   NCT01302600   Belgium;France;Germany;Italy;Netherlands;Poland;United Kingdom;
   TROPHOS
      2010   -   EUCTR2010-020386-24-IT   Belgium;Germany;Italy;Netherlands;United Kingdom;
Onasemnogene Abeparvovec-xioi   
   Novartis Gene Therapies
      2020   Phase 4   NCT04042025   Australia;Belgium;Canada;France;Italy;Japan;Korea, Republic of;Spain;Taiwan;United Kingdom;United States;
      2019   Phase 3   NCT03837184   Japan;Korea, Republic of;Taiwan;
      2018   Phase 3   NCT03461289   Belgium;France;Germany;Italy;Netherlands;Spain;Sweden;United Kingdom;
      2017   Phase 3   NCT03306277   United States;
      2017   Phase 1   NCT03381729   United States;
      2017   -   NCT03421977   United States;
Onasemnogene abeparvovec   
   AveXis, Inc.
      2019   Phase 3   EUCTR2017-004087-35-NL   Australia;Belgium;Canada;Germany;Israel;Italy;Japan;Korea, Democratic People's Republic of;Netherlands;Spain;Taiwan;United Kingdom;United States;
      2019   Phase 3   EUCTR2017-004087-35-BE   Australia;Belgium;Canada;Germany;Israel;Italy;Japan;Korea, Democratic People's Republic of;Netherlands;Spain;Taiwan;United Kingdom;United States;
      2019   Phase 3   EUCTR2017-000266-29-NL   Belgium;France;Germany;Italy;Netherlands;Spain;Sweden;United Kingdom;
      2019   Phase 3   EUCTR2017-000266-29-BE   Belgium;France;Italy;Netherlands;Spain;Sweden;United Kingdom;
      2018   Phase 3   EUCTR2017-004087-35-IT   Australia;Belgium;Canada;Germany;Israel;Italy;Japan;Korea, Democratic People's Republic of;Netherlands;Spain;Taiwan;United Kingdom;United States;
      2018   Phase 3   EUCTR2017-004087-35-ES   Australia;Belgium;Canada;Germany;Israel;Italy;Japan;Korea, Democratic People's Republic of;Netherlands;Spain;Taiwan;United Kingdom;United States;
      2018   Phase 3   EUCTR2017-000266-29-GB   Belgium;France;Italy;Netherlands;Spain;Sweden;United Kingdom;
      2018   Phase 3   EUCTR2017-000266-29-ES   Belgium;France;Germany;Italy;Netherlands;Spain;Sweden;United Kingdom;
      2018   Phase 3   EUCTR2017-000266-29-DE   Belgium;France;Germany;Italy;Netherlands;Spain;Sweden;United Kingdom;
      -   Phase 3   EUCTR2017-004087-35-DE   Australia;Belgium;Canada;Germany;Israel;Italy;Japan;Korea, Democratic People's Republic of;Netherlands;Spain;Taiwan;United Kingdom;United States;
      -   Phase 3   EUCTR2017-000266-29-SE   Belgium;France;Germany;Italy;Netherlands;Spain;Sweden;United Kingdom;
      -   Phase 3   EUCTR2017-000266-29-FR   Belgium;France;Germany;Italy;Netherlands;Spain;Sweden;United Kingdom;
   Novartis Gene Therapies, Inc.
      2018   Phase 3   EUCTR2017-004087-35-GB   Australia;Belgium;Canada;Germany;Israel;Italy;Japan;Korea, Democratic People's Republic of;Netherlands;Spain;Taiwan;United Kingdom;United States;
Onasemnogene abeparvovec-xioi   
   Novartis Gene Therapies
      2018   Phase 3   NCT03505099   Australia;Belgium;Canada;Germany;Israel;Italy;Japan;Korea, Republic of;Spain;Taiwan;United Kingdom;United States;
Orfiril Saft   
   University of Utah
      2009   -   EUCTR2008-003915-11-DE   Germany;
Paracetamol 120Mg/5mL Oral Suspension   
   Mette Cathrine Oerngreen
      2019   Phase 4   NCT03648658   Denmark;
Phosphate   
   LMU Klinikum
      2022   -   NCT05272969   Germany;
Placebo   
   CHUGAI PHARMACEUTICAL CO., LTD.
      2018   Phase 2   JPRN-JapicCTI-173722   Japan, Asia except Japan, North America, South America, Europe;
Previously termed sc.AAV9.CB.SMN and AVXS-101   
   NOVARTIS PHARMA AG
      2021   Phase 3   EUCTR2020-005995-37-IT   Australia;Belgium;Canada;France;Germany;Italy;Portugal;Spain;Switzerland;Taiwan;United Kingdom;United States;
   Novartis Pharma AG
      2021   Phase 3   EUCTR2020-005995-37-PT   Australia;Belgium;Canada;France;Germany;Italy;Portugal;Spain;Switzerland;Taiwan;United Kingdom;United States;
      2021   Phase 3   EUCTR2020-005995-37-FR   Australia;Belgium;Canada;France;Germany;Italy;Portugal;Spain;Switzerland;Taiwan;United Kingdom;United States;
      2021   Phase 3   EUCTR2020-005995-37-BE   Australia;Belgium;Canada;France;Germany;Italy;Portugal;Spain;Switzerland;Taiwan;United Kingdom;United States;
      -   Phase 3   EUCTR2021-003474-31-DK   Brazil;China;Colombia;Denmark;Egypt;India;Italy;Malaysia;Mexico;Russian Federation;Saudi Arabia;Singapore;South Africa;Taiwan;Thailand;United Arab Emirates;United States;Vietnam;
Pyridostigmine   
   UMC Utrecht
      2015   Phase 2   NCT02941328   Netherlands;
Pyridostigmine Bromide   
   Centre Hospitalier Régional de la Citadelle
      2014   Phase 2   NCT02227823   Belgium;
Pyridostigmine bromide   
   Universtiy Medical Center Utrecht
      2015   Phase 2   EUCTR2011-004369-34-NL   Netherlands;
RO6885247   
   Hoffmann-La Roche
      2014   Phase 1   NCT02240355   Canada;Czech Republic;France;Italy;Netherlands;Sweden;Switzerland;Turkey;United Kingdom;United States;
RO6885247/F02   
   Roche Farma S.A., en nombre de F. Hoffmann-La Roche Ltd.
      2015   Phase 1   EUCTR2014-002246-41-ES   France;Italy;Netherlands;Spain;Sweden;Switzerland;Turkey;United Kingdom;United States;
RO6885247/F03   
   F. Hoffmann-La Roche Ltd.
      2014   Phase 1   EUCTR2014-002246-41-IT   Italy;Netherlands;Spain;Switzerland;United Kingdom;United States;
   Roche Farma S.A., en nombre de F. Hoffmann-La Roche Ltd.
      2015   Phase 1   EUCTR2014-002246-41-ES   France;Italy;Netherlands;Spain;Sweden;Switzerland;Turkey;United Kingdom;United States;
RO7034067   
   CHUGAI PHARMACEUTICAL CO., LTD.
      2018   Phase 2   JPRN-JapicCTI-183891   Japan, Asia except Japan, North America, South America, Europe;
      2018   Phase 2   JPRN-JapicCTI-173722   Japan, Asia except Japan, North America, South America, Europe;
   F. HOFFMANN - LA ROCHE LTD.
      2022   Phase 2;Phase 3   EUCTR2021-003417-19-IT   Belgium;Italy;Netherlands;Poland;United Kingdom;United States;
RO7034067 / F12   
   F. Hoffmann-La Roche Ltd
      2017   Phase 2   EUCTR2016-000778-40-DE   Belgium;Brazil;Canada;China;Croatia;France;Germany;Italy;Japan;Lebanon;Poland;Russian Federation;Saudi Arabia;Serbia;Spain;Switzerland;Turkey;Ukraine;United Kingdom;United States;
RO7034067 / F13   
   F. Hoffmann-La Roche Ltd
      2017   Phase 2   EUCTR2016-000778-40-DE   Belgium;Brazil;Canada;China;Croatia;France;Germany;Italy;Japan;Lebanon;Poland;Russian Federation;Saudi Arabia;Serbia;Spain;Switzerland;Turkey;Ukraine;United Kingdom;United States;
RO7034067/F06 + solvente (RO   
   F. HOFFMANN - LA ROCHE LTD.
      2017   Phase 2   EUCTR2016-004184-39-IT   Italy;Switzerland;United Kingdom;United States;
RO7034067/F06 with solvent (RO7034067/F08)   
   F. Hoffmann-La Roche Ltd
      2017   Phase 2   EUCTR2016-000778-40-FR   Belgium;France;Germany;Italy;
      2017   Phase 2   EUCTR2016-000778-40-DE   Belgium;Brazil;Canada;China;Croatia;France;Germany;Italy;Japan;Lebanon;Poland;Russian Federation;Saudi Arabia;Serbia;Spain;Switzerland;Turkey;Ukraine;United Kingdom;United States;
      2016   Phase 2   EUCTR2016-000778-40-IT   Belgium;France;Germany;Italy;
      2016   Phase 2   EUCTR2016-000750-35-IT   Australia;Belgium;Canada;France;Germany;Italy;Netherlands;Spain;Sweden;Switzerland;Turkey;United Kingdom;United States;
      -   Phase 2   EUCTR2016-000750-35-FR   Australia;Belgium;Canada;France;Germany;Italy;Netherlands;Spain;Sweden;Switzerland;Turkey;United Kingdom;United States;
RO7034067/F07 + solvente (RO   
   F. HOFFMANN - LA ROCHE LTD.
      2017   Phase 2   EUCTR2016-004184-39-IT   Italy;Switzerland;United Kingdom;United States;
RO7034067/F07 with solvent (RO7034067/F09)   
   F. Hoffmann-La Roche Ltd
      2017   Phase 2   EUCTR2016-000778-40-FR   Belgium;France;Germany;Italy;
      2017   Phase 2   EUCTR2016-000778-40-DE   Belgium;Brazil;Canada;China;Croatia;France;Germany;Italy;Japan;Lebanon;Poland;Russian Federation;Saudi Arabia;Serbia;Spain;Switzerland;Turkey;Ukraine;United Kingdom;United States;
      2016   Phase 2   EUCTR2016-000778-40-IT   Belgium;France;Germany;Italy;
      2016   Phase 2   EUCTR2016-000750-35-IT   Australia;Belgium;Canada;France;Germany;Italy;Netherlands;Spain;Sweden;Switzerland;Turkey;United Kingdom;United States;
      -   Phase 2   EUCTR2016-000750-35-FR   Australia;Belgium;Canada;France;Germany;Italy;Netherlands;Spain;Sweden;Switzerland;Turkey;United Kingdom;United States;
RO7034067/F12   
   F. HOFFMANN - LA ROCHE LTD.
      2019   Phase 2   EUCTR2018-002087-12-IT   Australia;Belgium;Brazil;China;Italy;Poland;Russian Federation;Saudi Arabia;United States;
   F. Hoffmann-La Roche Ltd
      2020   Phase 2   EUCTR2016-000750-35-GB   Belgium;Brazil;Bulgaria;Canada;China;Croatia;France;Germany;Italy;Japan;Poland;Russian Federation;Serbia;Spain;Turkey;United Kingdom;United States;
      2019   Phase 2   EUCTR2018-002087-12-PL   Australia;Belgium;Brazil;China;Italy;Poland;Russian Federation;Saudi Arabia;United States;
      2018   Phase 2   EUCTR2018-002087-12-BE   Australia;Belgium;Brazil;China;Italy;Poland;Russian Federation;Saudi Arabia;United States;
      2018   Phase 2   EUCTR2016-000778-40-PL   Belgium;Brazil;Canada;China;Croatia;France;Germany;Italy;Japan;Lebanon;Poland;Russian Federation;Saudi Arabia;Serbia;Spain;Switzerland;Turkey;Ukraine;United Kingdom;United States;
      2018   Phase 2   EUCTR2016-000778-40-HR   Belgium;Brazil;Canada;China;Croatia;France;Germany;Italy;Japan;Lebanon;Poland;Russian Federation;Saudi Arabia;Serbia;Spain;Switzerland;Turkey;Ukraine;United Kingdom;United States;
      2018   Phase 2   EUCTR2016-000778-40-ES   Belgium;Brazil;Canada;China;Croatia;France;Germany;Italy;Japan;Lebanon;Poland;Russian Federation;Saudi Arabia;Serbia;Spain;Switzerland;Turkey;Ukraine;United Kingdom;United States;
      2018   Phase 2   EUCTR2016-000750-35-HR   Argentina;Belgium;Brazil;Bulgaria;Canada;China;Croatia;France;Germany;Hungary;Italy;Japan;Mexico;Poland;Romania;Russian Federation;Serbia;Spain;Switzerland;Turkey;Ukraine;United Kingdom;United States;
      2018   Phase 2   EUCTR2016-000750-35-BG   Argentina;Belgium;Brazil;Bulgaria;Canada;China;Croatia;France;Germany;Hungary;Italy;Japan;Mexico;Poland;Romania;Russian Federation;Serbia;Spain;Switzerland;Turkey;Ukraine;United Kingdom;United States;
      2016   Phase 2   EUCTR2016-000778-40-BE   Belgium;Brazil;Canada;China;Croatia;France;Germany;Italy;Japan;Lebanon;Poland;Russian Federation;Saudi Arabia;Serbia;Spain;Switzerland;Turkey;Ukraine;United Kingdom;United States;
      2016   Phase 2   EUCTR2016-000750-35-DE   Argentina;Belgium;Brazil;Bulgaria;Canada;China;Croatia;France;Germany;Hungary;Italy;Japan;Mexico;Poland;Romania;Russian Federation;Serbia;Spain;Switzerland;Turkey;Ukraine;United Kingdom;United States;
      2016   Phase 2   EUCTR2016-000750-35-BE   Argentina;Belgium;Brazil;Bulgaria;Canada;China;Croatia;France;Germany;Hungary;Italy;Japan;Mexico;Poland;Romania;Russian Federation;Serbia;Spain;Switzerland;Turkey;Ukraine;United Kingdom;United States;
      -   Phase 2   EUCTR2016-000750-35-PL   Argentina;Belgium;Brazil;Bulgaria;Canada;China;Croatia;France;Germany;Hungary;Italy;Japan;Mexico;Poland;Romania;Russian Federation;Serbia;Spain;Switzerland;Turkey;Ukraine;United Kingdom;United States;
RO7034067/F13   
   F. HOFFMANN - LA ROCHE LTD.
      2019   Phase 2   EUCTR2018-002087-12-IT   Australia;Belgium;Brazil;China;Italy;Poland;Russian Federation;Saudi Arabia;United States;
   F. Hoffmann-La Roche Ltd
      2020   Phase 2   EUCTR2016-000750-35-GB   Belgium;Brazil;Bulgaria;Canada;China;Croatia;France;Germany;Italy;Japan;Poland;Russian Federation;Serbia;Spain;Turkey;United Kingdom;United States;
      2019   Phase 2   EUCTR2018-002087-12-PL   Australia;Belgium;Brazil;China;Italy;Poland;Russian Federation;Saudi Arabia;United States;
      2019   Phase 2   EUCTR2016-004184-39-NL   Belgium;France;Germany;Italy;Netherlands;Poland;Switzerland;United Kingdom;United States;
      2018   Phase 2   EUCTR2018-002087-12-BE   Australia;Belgium;Brazil;China;Italy;Poland;Russian Federation;Saudi Arabia;United States;
      2018   Phase 2   EUCTR2016-000778-40-PL   Belgium;Brazil;Canada;China;Croatia;France;Germany;Italy;Japan;Lebanon;Poland;Russian Federation;Saudi Arabia;Serbia;Spain;Switzerland;Turkey;Ukraine;United Kingdom;United States;
      2018   Phase 2   EUCTR2016-000778-40-HR   Belgium;Brazil;Canada;China;Croatia;France;Germany;Italy;Japan;Lebanon;Poland;Russian Federation;Saudi Arabia;Serbia;Spain;Switzerland;Turkey;Ukraine;United Kingdom;United States;
      2018   Phase 2   EUCTR2016-000778-40-ES   Belgium;Brazil;Canada;China;Croatia;France;Germany;Italy;Japan;Lebanon;Poland;Russian Federation;Saudi Arabia;Serbia;Spain;Switzerland;Turkey;Ukraine;United Kingdom;United States;
      2018   Phase 2   EUCTR2016-000750-35-HR   Argentina;Belgium;Brazil;Bulgaria;Canada;China;Croatia;France;Germany;Hungary;Italy;Japan;Mexico;Poland;Romania;Russian Federation;Serbia;Spain;Switzerland;Turkey;Ukraine;United Kingdom;United States;
      2018   Phase 2   EUCTR2016-000750-35-BG   Argentina;Belgium;Brazil;Bulgaria;Canada;China;Croatia;France;Germany;Hungary;Italy;Japan;Mexico;Poland;Romania;Russian Federation;Serbia;Spain;Switzerland;Turkey;Ukraine;United Kingdom;United States;
      2017   Phase 2   EUCTR2016-004184-39-GB   Belgium;France;Germany;Italy;Netherlands;Poland;Switzerland;United Kingdom;United States;
      2016   Phase 2   EUCTR2016-000778-40-BE   Belgium;Brazil;Canada;China;Croatia;France;Germany;Italy;Japan;Lebanon;Poland;Russian Federation;Saudi Arabia;Serbia;Spain;Switzerland;Turkey;Ukraine;United Kingdom;United States;
      2016   Phase 2   EUCTR2016-000750-35-DE   Argentina;Belgium;Brazil;Bulgaria;Canada;China;Croatia;France;Germany;Hungary;Italy;Japan;Mexico;Poland;Romania;Russian Federation;Serbia;Spain;Switzerland;Turkey;Ukraine;United Kingdom;United States;
      2016   Phase 2   EUCTR2016-000750-35-BE   Argentina;Belgium;Brazil;Bulgaria;Canada;China;Croatia;France;Germany;Hungary;Italy;Japan;Mexico;Poland;Romania;Russian Federation;Serbia;Spain;Switzerland;Turkey;Ukraine;United Kingdom;United States;
      -   Phase 2   EUCTR2016-004184-39-PL   Belgium;France;Germany;Italy;Netherlands;Poland;Switzerland;United Kingdom;United States;
      -   Phase 2   EUCTR2016-004184-39-FR   Belgium;France;Germany;Italy;Netherlands;Poland;Switzerland;United Kingdom;United States;
      -   Phase 2   EUCTR2016-004184-39-DE   Belgium;France;Germany;Italy;Netherlands;Poland;Switzerland;United Kingdom;United States;
      -   Phase 2   EUCTR2016-004184-39-BE   Belgium;France;Germany;Italy;Netherlands;Poland;Switzerland;United Kingdom;United States;
      -   Phase 2   EUCTR2016-000750-35-PL   Argentina;Belgium;Brazil;Bulgaria;Canada;China;Croatia;France;Germany;Hungary;Italy;Japan;Mexico;Poland;Romania;Russian Federation;Serbia;Spain;Switzerland;Turkey;Ukraine;United Kingdom;United States;
RO7090919   
   F. Hoffmann-La Roche Ltd
      2016   Phase 2;Phase 3   EUCTR2015-001589-25-NL   Belgium;France;Germany;Italy;Netherlands;Poland;United Kingdom;
      2015   Phase 2;Phase 3   EUCTR2015-001589-25-IT   Belgium;France;Germany;Italy;Netherlands;Poland;United Kingdom;
      2015   Phase 2;Phase 3   EUCTR2015-001589-25-GB   Belgium;France;Germany;Italy;Netherlands;Poland;United Kingdom;
      2015   Phase 2;Phase 3   EUCTR2015-001589-25-FR   Belgium;France;Germany;Italy;Netherlands;Poland;United Kingdom;
      2015   Phase 2;Phase 3   EUCTR2015-001589-25-BE   Belgium;France;Germany;Italy;Netherlands;Poland;United Kingdom;
      -   Phase 2;Phase 3   EUCTR2015-001589-25-PL   Belgium;France;Germany;Italy;Netherlands;Poland;United Kingdom;
      -   Phase 2;Phase 3   EUCTR2015-001589-25-DE   Belgium;France;Germany;Italy;Netherlands;Poland;United Kingdom;
RO7204239   
   Hoffmann-La Roche
      2022   Phase 2/Phase 3   NCT05115110   Belgium;Germany;Italy;Netherlands;Poland;United Kingdom;United States;
RO7204239/F01-01   
   F. HOFFMANN - LA ROCHE LTD.
      2022   Phase 2;Phase 3   EUCTR2021-003417-19-IT   Belgium;Italy;Netherlands;Poland;United Kingdom;United States;
   F.Hoffmann-La Roche Ltd
      2022   Phase 2;Phase 3   EUCTR2021-003417-19-PL   Belgium;Italy;Netherlands;Poland;United Kingdom;United States;
      -   Phase 2;Phase 3   EUCTR2021-003417-19-NL   Belgium;Italy;Netherlands;Poland;United Kingdom;United States;
      -   Phase 2;Phase 3   EUCTR2021-003417-19-BE   Belgium;Italy;Netherlands;Poland;United Kingdom;United States;
RVG 03820   
   Universtiy Medical Center Utrecht
      2015   Phase 2   EUCTR2011-004369-34-NL   Netherlands;
Reldesemtiv   
   Cytokinetics
      2016   Phase 2   NCT02644668   Canada;United States;
Riluzole   
   Assistance Publique - Hôpitaux de Paris
      2006   Phase 2/Phase 3   NCT00774423   France;
Risdiplam   
   CHUGAI PHARMACEUTICAL CO., LTD.
      2018   Phase 2   JPRN-JapicCTI-183891   Japan, Asia except Japan, North America, South America, Europe;
      2018   Phase 2   JPRN-JapicCTI-173722   Japan, Asia except Japan, North America, South America, Europe;
   F. HOFFMANN - LA ROCHE LTD.
      2022   Phase 2;Phase 3   EUCTR2021-003417-19-IT   Belgium;Italy;Netherlands;Poland;United Kingdom;United States;
      2019   Phase 2   EUCTR2018-002087-12-IT   Australia;Belgium;Brazil;China;Italy;Poland;Russian Federation;Saudi Arabia;United States;
   F. Hoffmann-La Roche Ltd
      2020   Phase 2   EUCTR2016-000750-35-GB   Belgium;Brazil;Bulgaria;Canada;China;Croatia;France;Germany;Italy;Japan;Poland;Russian Federation;Serbia;Spain;Turkey;United Kingdom;United States;
      2019   Phase 2   EUCTR2018-002087-12-PL   Australia;Belgium;Brazil;China;Italy;Poland;Russian Federation;Saudi Arabia;United States;
      2019   Phase 2   EUCTR2016-004184-39-NL   Belgium;France;Germany;Italy;Netherlands;Poland;Switzerland;United Kingdom;United States;
      2018   Phase 2   EUCTR2018-002087-12-BE   Australia;Belgium;Brazil;China;Italy;Poland;Russian Federation;Saudi Arabia;United States;
      2018   Phase 2   EUCTR2016-000778-40-HR   Belgium;Brazil;Canada;China;Croatia;France;Germany;Italy;Japan;Lebanon;Poland;Russian Federation;Saudi Arabia;Serbia;Spain;Switzerland;Turkey;Ukraine;United Kingdom;United States;
      2018   Phase 2   EUCTR2016-000750-35-HR   Argentina;Belgium;Brazil;Bulgaria;Canada;China;Croatia;France;Germany;Hungary;Italy;Japan;Mexico;Poland;Romania;Russian Federation;Serbia;Spain;Switzerland;Turkey;Ukraine;United Kingdom;United States;
      2017   Phase 2   EUCTR2016-004184-39-GB   Belgium;France;Germany;Italy;Netherlands;Poland;Switzerland;United Kingdom;United States;
      2016   Phase 2   EUCTR2016-000778-40-BE   Belgium;Brazil;Canada;China;Croatia;France;Germany;Italy;Japan;Lebanon;Poland;Russian Federation;Saudi Arabia;Serbia;Spain;Switzerland;Turkey;Ukraine;United Kingdom;United States;
      2016   Phase 2   EUCTR2016-000750-35-DE   Argentina;Belgium;Brazil;Bulgaria;Canada;China;Croatia;France;Germany;Hungary;Italy;Japan;Mexico;Poland;Romania;Russian Federation;Serbia;Spain;Switzerland;Turkey;Ukraine;United Kingdom;United States;
      2016   Phase 2   EUCTR2016-000750-35-BE   Argentina;Belgium;Brazil;Bulgaria;Canada;China;Croatia;France;Germany;Hungary;Italy;Japan;Mexico;Poland;Romania;Russian Federation;Serbia;Spain;Switzerland;Turkey;Ukraine;United Kingdom;United States;
      -   Phase 2   EUCTR2016-004184-39-PL   Belgium;France;Germany;Italy;Netherlands;Poland;Switzerland;United Kingdom;United States;
      -   Phase 2   EUCTR2016-004184-39-FR   Belgium;France;Germany;Italy;Netherlands;Poland;Switzerland;United Kingdom;United States;
      -   Phase 2   EUCTR2016-004184-39-DE   Belgium;France;Germany;Italy;Netherlands;Poland;Switzerland;United Kingdom;United States;
      -   Phase 2   EUCTR2016-004184-39-BE   Belgium;France;Germany;Italy;Netherlands;Poland;Switzerland;United Kingdom;United States;
      -   Phase 2   EUCTR2016-000750-35-PL   Argentina;Belgium;Brazil;Bulgaria;Canada;China;Croatia;France;Germany;Hungary;Italy;Japan;Mexico;Poland;Romania;Russian Federation;Serbia;Spain;Switzerland;Turkey;Ukraine;United Kingdom;United States;
   F.Hoffmann-La Roche Ltd
      2022   Phase 2;Phase 3   EUCTR2021-003417-19-PL   Belgium;Italy;Netherlands;Poland;United Kingdom;United States;
      -   Phase 2;Phase 3   EUCTR2021-003417-19-NL   Belgium;Italy;Netherlands;Poland;United Kingdom;United States;
      -   Phase 2;Phase 3   EUCTR2021-003417-19-BE   Belgium;Italy;Netherlands;Poland;United Kingdom;United States;
   Genentech, Inc.
      2022   Phase 4   NCT05232929   United States;
      2014   -   NCT04256265   United States;
   Hoffmann-La Roche
      2022   Phase 2/Phase 3   NCT05115110   Belgium;Germany;Italy;Netherlands;Poland;United Kingdom;United States;
      2019   Phase 2   NCT03779334   Australia;Belgium;Brazil;Canada;China;Italy;Poland;Russian Federation;Saudi Arabia;Taiwan;United States;
      2019   Phase 1   NCT03988907   United States;
      2017   Phase 2   NCT03032172   Belgium;France;Germany;Italy;Netherlands;Poland;Switzerland;United Kingdom;United States;
      2016   Phase 2/Phase 3   NCT02913482   Australia;Belgium;Brazil;China;Croatia;France;Germany;Italy;Japan;Poland;Russian Federation;Saudi Arabia;Serbia;Spain;Switzerland;Taiwan;Turkey;Ukraine;United States;
      2016   Phase 2/Phase 3   NCT02908685   Argentina;Australia;Belgium;Brazil;Canada;China;Croatia;France;Germany;Italy;Japan;Poland;Russian Federation;Serbia;Spain;Sweden;Switzerland;Taiwan;Turkey;United Kingdom;United States;
      2016   Phase 1   NCT02633709   Netherlands;
SESAME OIL, REFINED   
   F. Hoffmann-La Roche Ltd
      2016   Phase 2;Phase 3   EUCTR2015-001589-25-NL   Belgium;France;Germany;Italy;Netherlands;Poland;United Kingdom;
      2015   Phase 2;Phase 3   EUCTR2015-001589-25-IT   Belgium;France;Germany;Italy;Netherlands;Poland;United Kingdom;
      2015   Phase 2;Phase 3   EUCTR2015-001589-25-GB   Belgium;France;Germany;Italy;Netherlands;Poland;United Kingdom;
      2015   Phase 2;Phase 3   EUCTR2015-001589-25-FR   Belgium;France;Germany;Italy;Netherlands;Poland;United Kingdom;
      2015   Phase 2;Phase 3   EUCTR2015-001589-25-BE   Belgium;France;Germany;Italy;Netherlands;Poland;United Kingdom;
      -   Phase 2;Phase 3   EUCTR2015-001589-25-PL   Belgium;France;Germany;Italy;Netherlands;Poland;United Kingdom;
SMART   
   Institute of Medical Genetics, Tokyo Women's Medical University
      2016   Phase 2B   JPRN-JMA-IIA00259   Japan;
      2016   Phase 2B   JPRN-JMA-IIA00231   Japan;
      2014   Phase 2A   JPRN-JMA-IIA00190   Japan;
SRK-015   
   Scholar Rock, Inc.
      2019   Phase 2   NCT03921528   Italy;Netherlands;Spain;United States;
      2019   Phase 2   EUCTR2018-004383-65-NL   Germany;Italy;Netherlands;Spain;United States;
      2019   Phase 2   EUCTR2018-004383-65-IT   Germany;Italy;Netherlands;Spain;United States;
      2019   Phase 2   EUCTR2018-004383-65-ES   Canada;Germany;Italy;Netherlands;Spain;United States;
      2019   Phase 2   EUCTR2018-004383-65-DE   Germany;Italy;Netherlands;Spain;United States;
      -   Phase 3   EUCTR2021-005314-34-NL   Belgium;France;Germany;Italy;Netherlands;Poland;Spain;United Kingdom;United States;
      -   Phase 3   EUCTR2021-005314-34-BE   Belgium;France;Germany;Italy;Netherlands;Poland;Spain;United Kingdom;United States;
Salbutamol   
   ISTITUTO NEUROLOGICO CARLO BESTA
      2008   -   EUCTR2007-001088-32-IT   Italy;
   The Children's Hospital, Zhejiang University School of Medicine
      2020   Phase 4   ChiCTR2000041208   China;
Sham Procedure   
   Biogen
      2015   Phase 2   NCT02462759   Germany;United States;
Sham procedure   
   Biogen
      2014   Phase 3   NCT02292537   Canada;France;Germany;Hong Kong;Italy;Japan;Korea, Republic of;Spain;Sweden;United Kingdom;United States;
      2014   Phase 3   NCT02193074   Australia;Belgium;Canada;France;Germany;Hong Kong;Italy;Japan;Korea, Republic of;Spain;Sweden;Taiwan;Turkey;United Kingdom;United States;
Sodium phenylbutyrate   
   Westat
      2008   Phase 1/Phase 2   NCT00439569   United States;
      2008   Phase 1/Phase 2   NCT00439218   United States;
Sodium phenylbutyrate (NaPB)   
   University of Utah
      2007   Phase 1/Phase 2   NCT00528268   United States;
Somatotropin   
   University Hospital Freiburg
      2007   Phase 2   NCT00533221   Germany;
Somatropin   
   Klinik Neuropädiatrie und Muskelkrankheiten
      2006   -   EUCTR2005-002822-78-DE   Germany;
Spinraza   
   BIOGEN IDEC RESEARCH LIMITED
      2021   Phase 4   EUCTR2020-003492-18-IT   Germany;Israel;Italy;Spain;United States;
      2020   Phase 2;Phase 3   EUCTR2019-002663-10-IT   Argentina;Australia;Brazil;Canada;Chile;Colombia;Estonia;France;Germany;Greece;Hungary;Ireland;Israel;Italy;Korea, Republic of;Latvia;Lebanon;Mexico;Poland;Russian Federation;Saudi Arabia;Spain;Taiwan;Turkey;United Kingdom;United States;
      -   Phase 3   EUCTR2015-001870-16-IT   Australia;Belgium;Canada;France;Germany;Hong Kong;Italy;Japan;Korea, Republic of;Spain;Sweden;Taiwan;Turkey;United Kingdom;United States;
   Biogen Idec Research Limited
      2021   Phase 4   EUCTR2020-003492-18-ES   Germany;Israel;Italy;Spain;United States;
      2021   Phase 4   EUCTR2020-003492-18-DE   Germany;Israel;Italy;Spain;United States;
      2021   Phase 2;Phase 3   EUCTR2019-002663-10-DE   Argentina;Australia;Brazil;Canada;Chile;Colombia;Estonia;France;Germany;Greece;Hungary;Ireland;Israel;Italy;Korea, Republic of;Latvia;Lebanon;Mexico;Poland;Russian Federation;Saudi Arabia;Spain;Taiwan;Turkey;United Kingdom;United States;
      2020   Phase 2;Phase 3   EUCTR2019-002663-10-LV   Argentina;Australia;Brazil;Canada;Chile;Colombia;Estonia;Hungary;Ireland;Italy;Korea, Republic of;Latvia;Lebanon;Mexico;Poland;Russian Federation;Saudi Arabia;Spain;United States;
      2020   Phase 2;Phase 3   EUCTR2019-002663-10-HU   Argentina;Australia;Brazil;Canada;Chile;Colombia;Estonia;Hungary;Ireland;Italy;Korea, Republic of;Latvia;Lebanon;Mexico;Poland;Russian Federation;Saudi Arabia;Spain;United States;
      2020   Phase 2;Phase 3   EUCTR2019-002663-10-GB   Argentina;Australia;Brazil;Canada;Chile;Colombia;Estonia;Germany;Greece;Hungary;Ireland;Israel;Italy;Korea, Republic of;Latvia;Lebanon;Mexico;Poland;Russian Federation;Saudi Arabia;Spain;Taiwan;Turkey;United Kingdom;United States;
      2020   Phase 2;Phase 3   EUCTR2019-002663-10-FR   Argentina;Australia;Brazil;Canada;Chile;Colombia;Estonia;France;Germany;Greece;Hungary;Ireland;Israel;Italy;Korea, Republic of;Latvia;Lebanon;Mexico;Poland;Russian Federation;Saudi Arabia;Spain;Taiwan;Turkey;United Kingdom;United States;
      2020   Phase 2;Phase 3   EUCTR2019-002663-10-ES   Argentina;Australia;Brazil;Canada;Chile;Colombia;Estonia;Greece;Hungary;Ireland;Italy;Korea, Republic of;Latvia;Lebanon;Mexico;Poland;Russian Federation;Saudi Arabia;Spain;United States;
      2016   Phase 3   EUCTR2015-001870-16-SE   Australia;Belgium;Canada;France;Germany;Hong Kong;Italy;Japan;Korea, Republic of;Spain;Sweden;Turkey;United Kingdom;United States;
      2016   Phase 3   EUCTR2015-001870-16-GB   Australia;Belgium;Canada;France;Germany;Hong Kong;Italy;Japan;Korea, Republic of;Spain;Sweden;Turkey;United Kingdom;United States;
      2016   Phase 3   EUCTR2015-001870-16-DE   Australia;Belgium;Canada;France;Germany;Hong Kong;Italy;Japan;Korea, Republic of;Spain;Sweden;Turkey;United Kingdom;United States;
      2016   Phase 3   EUCTR2015-001870-16-BE   Australia;Belgium;Canada;France;Germany;Hong Kong;Italy;Japan;Korea, Republic of;Spain;Sweden;Turkey;United Kingdom;United States;
      2015   Phase 2   EUCTR2014-003657-33-GB   Germany;United Kingdom;United States;
      2015   Phase 2   EUCTR2014-003657-33-DE   Germany;United Kingdom;United States;
      2015   Phase 2   EUCTR2014-002098-12-GB   Australia;Germany;Italy;Qatar;Taiwan;Turkey;United Kingdom;United States;
      2015   Phase 2   EUCTR2014-002098-12-DE   Australia;Canada;Germany;Italy;Qatar;Taiwan;Turkey;United Kingdom;United States;
      -   Phase 2;Phase 3   EUCTR2019-002663-10-PL   Australia;Brazil;Canada;Chile;China;Colombia;Estonia;France;Germany;Greece;Hungary;Ireland;Israel;Italy;Japan;Korea, Republic of;Latvia;Lebanon;Mexico;Netherlands;Poland;Russian Federation;Saudi Arabia;Spain;Taiwan;Turkey;United Kingdom;United States;
      -   Phase 2;Phase 3   EUCTR2019-002663-10-NL   Australia;Brazil;Canada;Chile;China;Colombia;Estonia;France;Germany;Greece;Hungary;Ireland;Israel;Italy;Japan;Korea, Republic of;Latvia;Lebanon;Mexico;Netherlands;Poland;Russian Federation;Saudi Arabia;Spain;Taiwan;Turkey;United Kingdom;United States;
      -   Phase 2;Phase 3   EUCTR2019-002663-10-IE   Argentina;Australia;Brazil;Canada;Chile;China;Colombia;Estonia;France;Germany;Greece;Hungary;Ireland;Israel;Italy;Korea, Republic of;Latvia;Lebanon;Mexico;Netherlands;Poland;Russian Federation;Saudi Arabia;Spain;Taiwan;Turkey;United Kingdom;United States;
      -   Phase 2;Phase 3   EUCTR2019-002663-10-GR   Argentina;Australia;Brazil;Canada;Chile;Colombia;Estonia;France;Germany;Greece;Hungary;Ireland;Israel;Italy;Korea, Republic of;Latvia;Lebanon;Mexico;Poland;Russian Federation;Saudi Arabia;Spain;Taiwan;Turkey;United States;
Sulfate   
   The Children's Hospital, Zhejiang University School of Medicine
      2020   Phase 4   ChiCTR2000041208   China;
Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide   
   Biogen Idec Research Limited
      2016   Phase 3   EUCTR2015-001870-16-SE   Australia;Belgium;Canada;France;Germany;Hong Kong;Italy;Japan;Korea, Republic of;Spain;Sweden;Turkey;United Kingdom;United States;
      2016   Phase 3   EUCTR2015-001870-16-GB   Australia;Belgium;Canada;France;Germany;Hong Kong;Italy;Japan;Korea, Republic of;Spain;Sweden;Turkey;United Kingdom;United States;
      2016   Phase 3   EUCTR2015-001870-16-DE   Australia;Belgium;Canada;France;Germany;Hong Kong;Italy;Japan;Korea, Republic of;Spain;Sweden;Turkey;United Kingdom;United States;
      2016   Phase 3   EUCTR2015-001870-16-BE   Australia;Belgium;Canada;France;Germany;Hong Kong;Italy;Japan;Korea, Republic of;Spain;Sweden;Turkey;United Kingdom;United States;
      2015   Phase 2   EUCTR2014-003657-33-DE   Germany;United Kingdom;United States;
      2015   Phase 2   EUCTR2014-002098-12-DE   Australia;Canada;Germany;Italy;Qatar;Taiwan;Turkey;United Kingdom;United States;
      2015   -   EUCTR2014-002098-12-IT   Argentina;Australia;Germany;Israel;Italy;Qatar;Taiwan;Turkey;United Kingdom;United States;
   Ionis Pharmaceuticals, Inc.
      2016   -   EUCTR2015-001870-16-ES   Australia;Belgium;Canada;France;Germany;Hong Kong;Italy;Japan;Korea, Republic of;Spain;Sweden;Taiwan;Turkey;United Kingdom;United States;
      2015   Phase 3   EUCTR2014-001947-18-SE   Canada;France;Germany;Hong Kong;Italy;Japan;Korea, Republic of;Spain;Sweden;United Kingdom;United States;
      2015   Phase 3   EUCTR2014-001947-18-DE   Canada;France;Germany;Hong Kong;Italy;Japan;Korea, Republic of;Spain;Sweden;United Kingdom;United States;
      2015   Phase 3   EUCTR2013-004422-29-DE   Australia;Belgium;Canada;France;Germany;Hong Kong;Italy;Japan;Korea, Republic of;Spain;Sweden;Taiwan;Turkey;United Kingdom;United States;
      2015   Phase 3   EUCTR2013-004422-29-BE   Australia;Belgium;Canada;France;Germany;Hong Kong;Italy;Japan;Korea, Republic of;Spain;Sweden;Taiwan;United Kingdom;United States;
      2014   Phase 3   EUCTR2013-004422-29-SE   Australia;Belgium;Canada;France;Germany;Hong Kong;Italy;Japan;Korea, Republic of;Spain;Sweden;Taiwan;Turkey;United Kingdom;United States;
      2014   Phase 3   EUCTR2013-004422-29-GB   Australia;Belgium;Canada;France;Germany;Hong Kong;Italy;Japan;Korea, Republic of;Spain;Sweden;Taiwan;Turkey;United Kingdom;United States;
      -   Phase 3   EUCTR2015-001870-16-FR   Australia;Belgium;Canada;France;Germany;Hong Kong;Italy;Japan;Korea, Republic of;Spain;Sweden;Taiwan;Turkey;United Kingdom;United States;
      -   Phase 2   EUCTR2017-000621-12-Outside-EU/EEA   Canada;United States;
      -   Phase 2   EUCTR2017-000327-27-Outside-EU/EEA   United States;
   Isis Pharmaceuticals, Inc.
      2015   Phase 3   EUCTR2014-001947-18-IT   Canada;France;Germany;Hong Kong;Italy;Japan;Korea, Republic of;Spain;Sweden;United Kingdom;United States;
      2015   Phase 3   EUCTR2014-001947-18-ES   Canada;France;Germany;Hong Kong;Italy;Japan;Korea, Republic of;Spain;Sweden;United Kingdom;United States;
      2015   Phase 3   EUCTR2013-004422-29-ES   Australia;Belgium;Canada;France;Germany;Hong Kong;Italy;Japan;Korea, Republic of;Spain;Sweden;Taiwan;United Kingdom;United States;
      2014   Phase 3   EUCTR2013-004422-29-IT   Australia;Belgium;Canada;France;Germany;Hong Kong;Italy;Japan;Korea, Republic of;Spain;Sweden;Taiwan;United Kingdom;United States;
      -   Phase 3   EUCTR2014-001947-18-GB   Canada;France;Germany;Hong Kong;Italy;Japan;Korea, Republic of;Spain;Sweden;United Kingdom;United States;
      -   Phase 3   EUCTR2014-001947-18-FR   Canada;France;Germany;Hong Kong;Italy;Japan;Korea, Republic of;Spain;Sweden;United Kingdom;United States;
TRO19622   
   TROPHOS
      2010   -   EUCTR2010-020386-24-IT   Belgium;Germany;Italy;Netherlands;United Kingdom;
      2007   -   EUCTR2006-006845-14-FR   France;
   TROPHOS SA
      2011   Phase 2   EUCTR2010-020386-24-GB   Belgium;France;Germany;Italy;Netherlands;United Kingdom;
      2010   Phase 2   EUCTR2010-020386-24-DE   Belgium;Germany;Italy;Netherlands;United Kingdom;
      2010   Phase 2   EUCTR2010-020386-24-BE   Belgium;Germany;Italy;Netherlands;United Kingdom;
      2010   -   EUCTR2010-020386-24-NL   Belgium;Germany;Italy;Netherlands;United Kingdom;
      -   Phase 2   EUCTR2010-020386-24-FR   Belgium;France;Germany;Italy;Netherlands;United Kingdom;
Testosterone   
   National Center for Research Resources (NCRR)
      1992   Phase 2   NCT00004771   -
VALPROATE SODIUM   
   University of Utah
      2009   -   EUCTR2008-003915-11-DE   Germany;
VOLMAX*30CPR 4MG R.P.   
   ISTITUTO NEUROLOGICO CARLO BESTA
      2008   -   EUCTR2007-001088-32-IT   Italy;
Valproate, Levocarnitine   
   All India Institute of Medical Sciences, New Delhi
      2013   Phase 3   NCT01671384   India;
Valproic Acid (VPA)   
   University of Utah
      2007   Phase 2   NCT00481013   United States;
Valproic Acid and Levocarnitine   
   University of Utah
      2008   Phase 1/Phase 2   NCT00661453   Canada;Germany;United States;
      2005   Phase 2   NCT00227266   Canada;United States;
Vitamin D   
   LMU Klinikum
      2022   -   NCT05272969   Germany;
Zolgensma   
   AveXis, Inc.
      2018   -   NCT04174157   Ireland;Israel;Japan;Portugal;United States;
   NOVARTIS PHARMA AG
      2021   Phase 3   EUCTR2020-005995-37-IT   Australia;Belgium;Canada;France;Germany;Italy;Portugal;Spain;Switzerland;Taiwan;United Kingdom;United States;
   Novartis Pharma AG
      2021   Phase 3   EUCTR2020-005995-37-FR   Australia;Belgium;Canada;France;Germany;Italy;Portugal;Spain;Switzerland;Taiwan;United Kingdom;United States;
      -   Phase 3   EUCTR2021-003474-31-DK   Brazil;China;Colombia;Denmark;Egypt;India;Italy;Malaysia;Mexico;Russian Federation;Saudi Arabia;Singapore;South Africa;Taiwan;Thailand;United Arab Emirates;United States;Vietnam;
Zolgensma 2 x 1013 vector genomes/mL solution for infusion   
   Novartis Pharma AG
      2021   Phase 3   EUCTR2020-005995-37-PT   Australia;Belgium;Canada;France;Germany;Italy;Portugal;Spain;Switzerland;Taiwan;United Kingdom;United States;
      2021   Phase 3   EUCTR2020-005995-37-BE   Australia;Belgium;Canada;France;Germany;Italy;Portugal;Spain;Switzerland;Taiwan;United Kingdom;United States;