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

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-aminopyridine
   Columbia University
      2012   Phase 2/Phase 3   NCT01645787   United States
4-cholesten-3-ONE, oxime
   TROPHOS SA
      2011   Phase 2   EUCTR2010-020386-24-GB   Belgium;France;Germany;Italy;Netherlands;United Kingdom
      2010   -   EUCTR2010-020386-24-NL   Belgium;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
      -   Phase 2   EUCTR2010-020386-24-FR   Belgium;France;Germany;Italy;Netherlands;United Kingdom
4-cholesten-3-ONE,oxime
   TROPHOS
      2007   Phase 1   EUCTR2006-006845-14-FR   France
Adipose derived mesenchymal stem cell
   Tehran University of Medical Sciences
      2015   Phase 1/Phase 2   NCT02855112   Iran, Islamic Republic of
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
ANB-004, dose 1
   Biocad
      2023   Phase 1/Phase 2   NCT05747261   Belarus;Russian Federation
ANB-004, dose 2
   Biocad
      2023   Phase 1/Phase 2   NCT05747261   Belarus;Russian Federation
ANB-004, dose 3
   Biocad
      2023   Phase 1/Phase 2   NCT05747261   Belarus;Russian Federation
Apitegromab
   Scholar Rock, Inc.
      2024   -   NCT06877689   -
      2023   Phase 3   NCT05626855   Belgium;France;Germany;Italy;Netherlands;Poland;Spain;United Kingdom;United States
      2023   Phase 3   EUCTR2022-001771-14-FR   Belgium;France;Germany;Italy;Netherlands;Poland;Spain;United Kingdom;United States
      2023   Phase 3   EUCTR2022-001771-14-BE   Belgium;France;Germany;Italy;Netherlands;Poland;Spain;United Kingdom;United States
      2022   Phase 3   NCT05156320   Belgium;France;Germany;Italy;Netherlands;Poland;Spain;United Kingdom;United States
      2022   Phase 3   EUCTR2021-005314-34-NL   Belgium;France;Germany;Italy;Netherlands;Poland;Spain;United Kingdom;United States
      2022   Phase 3   EUCTR2021-005314-34-IT   Belgium;France;Germany;Italy;Netherlands;Poland;Spain;United Kingdom;United States
      2022   Phase 3   EUCTR2021-005314-34-FR   Belgium;France;Germany;Italy;Netherlands;Poland;Spain;United Kingdom;United States
      2022   Phase 3   EUCTR2021-005314-34-ES   Belgium;France;Germany;Italy;Netherlands;Poland;Spain;United Kingdom;United States
      2022   Phase 3   EUCTR2021-005314-34-DE   Belgium;France;Germany;Italy;Netherlands;Poland;Spain;United Kingdom;United States
      2022   Phase 3   EUCTR2021-005314-34-BE   Belgium;France;Germany;Italy;Netherlands;Poland;Spain;United Kingdom;United States
      2019   Phase 2   EUCTR2018-004383-65-NL   Germany;Italy;Netherlands;Spain;United States
      -   Phase 3   EUCTR2022-001771-14-PL   Belgium;France;Germany;Italy;Netherlands;Poland;Spain;United Kingdom;United States
      -   Phase 3   EUCTR2022-001771-14-NL   Belgium;France;Germany;Italy;Netherlands;Poland;Spain;United Kingdom;United States
      -   Phase 3   EUCTR2022-001771-14-DE   Belgium;France;Germany;Italy;Netherlands;Poland;Spain;United Kingdom;United States
      -   Phase 3   EUCTR2021-005314-34-PL   Belgium;France;Germany;Italy;Netherlands;Poland;Spain;United Kingdom;United States
Assessment OF activity and muscle fatigue
   Hospices Civils de Lyon
      2024   -   NCT06321965   France
Assessment OF bulbar function
   Hospices Civils de Lyon
      2024   -   NCT06321965   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
      2018   Phase 3   EUCTR2017-000266-29-IT   Belgium;France;Germany;Italy;Netherlands;Spain;Sweden;United Kingdom
   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
      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
      2014   Phase 1   NCT02122952   United States
   Novartis Gene Therapies, Inc.
      2020   Phase 3   EUCTR2019-002611-26-BE   Australia;Belgium;Canada;France;Italy;Japan;Spain;Taiwan;United Kingdom;United States
      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
   United BioSource, LLC
      2019   -   NCT03955679   United States
BHV-2000
   Biohaven Pharmaceuticals, Inc
      2023   Phase 3   EUCTR2022-000193-25-DE   Belgium;Czech Republic;France;Germany;Italy;Poland;Spain
      2023   Phase 3   EUCTR2022-000193-25-CZ   Belgium;Czech Republic;France;Germany;Italy;Poland;Spain
   Biohaven Pharmaceuticals, Inc.
      2022   Phase 3   EUCTR2022-000193-25-IT   Belgium;Czech Republic;France;Germany;Italy;Poland;Spain;United Kingdom;United States
      -   Phase 3   EUCTR2022-000193-25-PL   Belgium;Czech Republic;Czechia;France;Germany;Italy;Netherlands;Poland;Spain;United Kingdom;United States
BIIB115
   Biogen Idec Research Limited
      2023   Phase 1   EUCTR2022-000956-12-PT   Australia;Belgium;Canada;France;Germany;Israel;Italy;Netherlands;Poland;Portugal;Spain;United Kingdom
      2023   Phase 1   EUCTR2022-000956-12-DE   Australia;Belgium;Canada;France;Germany;Israel;Italy;Netherlands;Poland;Portugal;Spain;United Kingdom
      -   Phase 1   EUCTR2022-000956-12-PL   Australia;Belgium;Canada;France;Germany;Israel;Italy;Netherlands;Poland;Portugal;Spain;United Kingdom
      -   Phase 1   EUCTR2022-000956-12-BE   Australia;Belgium;Canada;France;Germany;Israel;Italy;Netherlands;Poland;Portugal;Spain;United Kingdom
Biocarn
   University of Utah
      2009   -   EUCTR2008-003915-11-DE   Germany
Biocollection
   Hospices Civils de Lyon
      2024   -   NCT06321965   France
Blood draw FOR optional genetic exome sequencing
   LMU Klinikum
      2022   -   NCT05272969   Germany
Blood sample
   Assistance Publique - Hôpitaux de Paris
      2024   -   NCT06147414   France
Borg scale
   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
Cardiac evaluation
   Hospices Civils de Lyon
      2024   -   NCT06321965   France
Celecoxib
   Hugh McMillan
      2019   Phase 2   NCT02876094   Canada
Disease modifying treatments
   Fondazione Policlinico Universitario Agostino Gemelli IRCCS
      2023   -   NCT05769465   Italy
Evaluation OF body composition and metabolism
   Hospices Civils de Lyon
      2024   -   NCT06321965   France
Evaluation OF muscle function
   Hospices Civils de Lyon
      2024   -   NCT06321965   France
Evrysdi
   F. HOFFMANN - LA ROCHE LTD.
      2022   Phase 2;Phase 3   EUCTR2021-003417-19-IT   Belgium;France;Germany;Italy;Netherlands;Poland;Portugal;Spain;United Kingdom;United States
   F. Hoffmann-La Roche Ltd
      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
   F.Hoffmann-La Roche Ltd
      2023   Phase 2;Phase 3   EUCTR2021-003417-19-PT   Belgium;Croatia;France;Germany;Italy;Netherlands;Poland;Portugal;Spain;United Kingdom;United States
      2022   Phase 2;Phase 3   EUCTR2021-003417-19-PL   Belgium;France;Germany;Italy;Netherlands;Poland;Portugal;Spain;United Kingdom;United States
      2022   Phase 2;Phase 3   EUCTR2021-003417-19-NL   Belgium;France;Germany;Italy;Netherlands;Poland;Portugal;Spain;United Kingdom;United States
      2022   Phase 2;Phase 3   EUCTR2021-003417-19-BE   Belgium;Germany;Italy;Netherlands;Poland;Portugal;Spain;United Kingdom;United States
      -   Phase 2;Phase 3   EUCTR2021-003417-19-DE   Belgium;Germany;Italy;Netherlands;Poland;Portugal;Spain;United Kingdom;United States
EXG001-307 injection
   Hangzhou Jiayin Biotech Ltd
      2025   Early Phase 1   NCT06888661   China
      2023   Phase 1/Phase 2   NCT05614531   China
      2022   Early Phase 1   NCT06576388   China
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
First-line cognitive assessment
   Hospices Civils de Lyon
      2024   -   NCT06321965   France
GC101
   GeneCradle Inc
      2024   Phase 1/Phase 2   NCT06421831   China
      2023   Phase 1/Phase 2   NCT05901987   China
      2023   Phase 1/Phase 2   NCT05824169   China
GC101 adeno-associated virus injection
   GeneCradle Inc
      2025   Phase 3   NCT06971094   China
GCB-001
   Genecombio Ltd.
      2025   -   NCT06772402   China
Genetic test: ACE polymorphism
   LMU Klinikum
      2022   -   NCT05272969   Germany
Genetic test: ACTN3 polymorphism
   LMU Klinikum
      2022   -   NCT05272969   Germany
GYM329
   F.Hoffmann-La Roche Ltd
      2023   Phase 2;Phase 3   EUCTR2021-003417-19-PT   Belgium;Croatia;France;Germany;Italy;Netherlands;Poland;Portugal;Spain;United Kingdom;United States
      2022   Phase 2;Phase 3   EUCTR2021-003417-19-PL   Belgium;France;Germany;Italy;Netherlands;Poland;Portugal;Spain;United Kingdom;United States
      2022   Phase 2;Phase 3   EUCTR2021-003417-19-NL   Belgium;France;Germany;Italy;Netherlands;Poland;Portugal;Spain;United Kingdom;United States
      2022   Phase 2;Phase 3   EUCTR2021-003417-19-BE   Belgium;Germany;Italy;Netherlands;Poland;Portugal;Spain;United Kingdom;United States
      -   Phase 2;Phase 3   EUCTR2021-003417-19-DE   Belgium;Germany;Italy;Netherlands;Poland;Portugal;Spain;United Kingdom;United States
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
Interlaminar ultrasound-assisted intrathecal administration OF nusinersen
   Fondazione Policlinico Universitario Agostino Gemelli IRCCS
      2022   -   NCT05644899   Italy
Isis 396443
   BIOGEN IDEC RESEARCH LIMITED
      2021   Phase 4   EUCTR2020-003492-18-IT   Germany;Israel;Italy;Spain;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;Japan;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-IT   Argentina;Australia;Germany;Israel;Italy;Qatar;Taiwan;Turkey;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;Türkiye;United Kingdom;United States
   Ionis Pharmaceuticals, Inc.
      2016   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
      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 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
      2022   Phase 3   EUCTR2021-001294-23-IT   Australia;Belgium;Brazil;Canada;Germany;Hungary;Italy;Japan;Poland;Spain;Switzerland
      2022   Phase 3   EUCTR2020-004708-32-IT   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
      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;Netherlands;Poland;Russian Federation;Saudi Arabia;Spain;Taiwan;Turkey;United Kingdom;United States
   Biogen Idec Research Limited
      2023   Phase 3   EUCTR2021-001294-23-HU   Australia;Belgium;Brazil;Canada;Germany;Hungary;Italy;Japan;Poland;Spain;Switzerland
      2022   Phase 3   EUCTR2021-001294-23-ES   Australia;Belgium;Brazil;Canada;Germany;Hungary;Italy;Japan;Poland;Spain;Switzerland
      2022   Phase 3   EUCTR2020-004708-32-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
      2022   Phase 3   EUCTR2020-004708-32-DE   Brazil;Canada;Chile;China;Colombia;Estonia;France;Germany;Hungary;Italy;Japan;Lebanon;Mexico;Netherlands;Poland;Russian Federation;Saudi Arabia;Spain;Taiwan;United States
      2022   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
      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;Netherlands;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   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;Türkiye;United Kingdom;United States
      2020   Phase 2;Phase 3   EUCTR2019-002663-10-LV   Argentina;Australia;Brazil;Canada;Chile;Colombia;Estonia;France;Germany;Greece;Hungary;Ireland;Italy;Korea, Republic of;Latvia;Lebanon;Mexico;Netherlands;Poland;Russian Federation;Saudi Arabia;Spain;United Kingdom;United States
      2020   Phase 2;Phase 3   EUCTR2019-002663-10-HU   Argentina;Australia;Brazil;Canada;Chile;Colombia;Estonia;France;Germany;Greece;Hungary;Ireland;Italy;Korea, Republic of;Latvia;Lebanon;Mexico;Netherlands;Poland;Russian Federation;Saudi Arabia;Spain;United Kingdom;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;Netherlands;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-PL   Australia;Belgium;Brazil;Canada;Germany;Hungary;Italy;Japan;Poland;Spain;Switzerland
      -   Phase 3   EUCTR2021-001294-23-DE   Belgium;Germany;Hungary;Italy;Japan;Poland;Spain;United States
      -   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 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;Türkiye;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   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
Itraconazole
   Hoffmann-La Roche
      2016   Phase 1   NCT02633709   Netherlands
Laboratory assessment: calcium
   LMU Klinikum
      2022   -   NCT05272969   Germany
Laboratory assessment: creatine kinase
   LMU Klinikum
      2022   -   NCT05272969   Germany
Laboratory assessment: magnesium
   LMU Klinikum
      2022   -   NCT05272969   Germany
Laboratory assessment: phosphate
   LMU Klinikum
      2022   -   NCT05272969   Germany
Laboratory assessment: vitamin D level
   LMU Klinikum
      2022   -   NCT05272969   Germany
Landmark-based intrathecal administration OF nusinersen
   Fondazione Policlinico Universitario Agostino Gemelli IRCCS
      2022   -   NCT05644899   Italy
Leuprolide
   National Center for Research Resources (NCRR)
      1992   Phase 2   NCT00004771   -
Levocarnitine
   University of Utah
      2009   -   EUCTR2008-003915-11-DE   Germany
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
Mestinon
   Universtiy Medical Center Utrecht
      2015   Phase 2   EUCTR2011-004369-34-NL   Netherlands
Midazolam
   Hoffmann-La Roche
      2019   Phase 1   NCT03988907   United States
Mobility study IN DRY conditions
   Assistance Publique - Hôpitaux de Paris
      2025   -   NCT06152302   France
Mobility study IN water conditions
   Assistance Publique - Hôpitaux de Paris
      2025   -   NCT06152302   France
Monthly assessments OF functional motor abilities BY A trained therapist
   CHU de Reims
      2022   -   NCT04576494   France
Muscle ultrasound
   LMU Klinikum
      2022   -   NCT05272969   Germany
Nerve growth factor
   PLA General Hospital
      2010   -   ChiCTR-TRC-10001093   China
NMD670
   NMD Pharma A/S
      2023   Phase 2   NCT05794139   Belgium;Canada;Denmark;Germany;Italy;Netherlands;Spain;United States
      2023   Phase 2   EUCTR2022-002301-24-NL   Belgium;Canada;Denmark;Germany;Italy;Netherlands;Spain;United States
      2023   Phase 2   EUCTR2022-002301-24-IT   Belgium;Canada;Denmark;Germany;Italy;Netherlands;Spain;United States
      2023   Phase 2   EUCTR2022-002301-24-DK   Belgium;Canada;Denmark;Germany;Italy;Netherlands;Spain;United States
      2023   Phase 2   EUCTR2022-002301-24-DE   Belgium;Canada;Denmark;Germany;Italy;Netherlands;Spain;United States
      2023   Phase 2   EUCTR2022-002301-24-BE   Belgium;Canada;Denmark;Germany;Italy;Netherlands;Spain;United States
      -   Phase 2   EUCTR2022-002301-24-ES   Belgium;Canada;Denmark;Germany;Italy;Netherlands;Spain;United States
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
   BIOGEN IDEC RESEARCH LIMITED
      2022   Phase 3   EUCTR2020-004708-32-IT   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
   Berger Zdenek
      2021   Phase 2-3   JPRN-jRCT2061200040   Colombia;Estonia;Ireland;Japan;Latvia;Spain;Taiwan;United States
   Biogen
      2025   Phase 1   NCT06555419   United States
      2022   Phase 3   NCT05067790   Belgium;Brazil;Germany;Hungary;Italy;Japan;Poland;Spain;United States
      2021   Phase 3   NCT04729907   Argentina;Australia;Brazil;Canada;Chile;China;Colombia;Estonia;France;Germany;Greece;Hungary;Ireland;Israel;Italy;Japan;Korea, Republic of;Latvia;Lebanon;Mexico;Poland;Russian Federation;Saudi Arabia;Spain;Taiwan;Turkey;United Kingdom;United States
      2021   Phase 4   NCT04488133   Germany;Israel;Italy;Japan;Spain;United States
      2020   Phase 3   NCT04089566   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
      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;Türkiye;United Kingdom;United States
   CHU de Reims
      2022   -   NCT04576494   France
   Ionis Pharmaceuticals, Inc.
      -   Phase 2   EUCTR2017-000621-12-Outside-EU/EEA   Canada;United States
      -   Phase 2   EUCTR2017-000327-27-Outside-EU/EEA   United States
   Irzhevsky Victoria
      2022   Phase 3   JPRN-jRCT2031220035   Australia;Canada;Estonia;France;Germany;Greece;Hungary;Ireland;Israel;Italy;Japan;Latvia;Lebanon;Poland;Russia;South Korea;Spain;Taiwan;Turkey;United Kingdom;United States
   Northwell Health
      2019   -   NCT03878030   United States
   Ohio State University
      2018   -   NCT04591678   United States
   Toda Yasuo
      2022   Phase 4   JPRN-jRCT2051210186   Germany;Israel;Italy;Japan;Spain;United States
   Tsuda Hiroshi
      2024   Phase 3   JPRN-jRCT2051230096   Belgium;Brazil;Germany;Hungary;Italy;Japan;Poland;Spain;USA
   University Medical Centre Ljubljana
      2017   -   NCT04825119   Slovenia
      2017   -   NCT04587492   Slovenia
Nusinersen injectable product
   Hospital Israelita Albert Einstein
      2020   -   NCT04404764   Brazil
Nusinersen sodium
   BIOGEN IDEC RESEARCH LIMITED
      2022   Phase 3   EUCTR2021-001294-23-IT   Australia;Belgium;Brazil;Canada;Germany;Hungary;Italy;Japan;Poland;Spain;Switzerland
      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;Netherlands;Poland;Russian Federation;Saudi Arabia;Spain;Taiwan;Turkey;United Kingdom;United States
   Biogen Idec Research Limited
      2023   Phase 3   EUCTR2021-001294-23-HU   Australia;Belgium;Brazil;Canada;Germany;Hungary;Italy;Japan;Poland;Spain;Switzerland
      2022   Phase 3   EUCTR2021-001294-23-ES   Australia;Belgium;Brazil;Canada;Germany;Hungary;Italy;Japan;Poland;Spain;Switzerland
      2022   Phase 3   EUCTR2020-004708-32-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
      2022   Phase 3   EUCTR2020-004708-32-DE   Brazil;Canada;Chile;China;Colombia;Estonia;France;Germany;Hungary;Italy;Japan;Lebanon;Mexico;Netherlands;Poland;Russian Federation;Saudi Arabia;Spain;Taiwan;United States
      2022   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
      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;Netherlands;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 4   EUCTR2020-003492-18-ES   Germany;Israel;Italy;Spain;United States
      2021   Phase 4   EUCTR2020-003492-18-DE   Germany;Israel;Italy;Japan;Spain;United States
      2021   Phase 2;Phase 3   EUCTR2019-002663-10-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;Türkiye;United Kingdom;United States
      2020   Phase 2;Phase 3   EUCTR2019-002663-10-LV   Argentina;Australia;Brazil;Canada;Chile;Colombia;Estonia;France;Germany;Greece;Hungary;Ireland;Italy;Korea, Republic of;Latvia;Lebanon;Mexico;Netherlands;Poland;Russian Federation;Saudi Arabia;Spain;United Kingdom;United States
      2020   Phase 2;Phase 3   EUCTR2019-002663-10-HU   Argentina;Australia;Brazil;Canada;Chile;Colombia;Estonia;France;Germany;Greece;Hungary;Ireland;Italy;Korea, Republic of;Latvia;Lebanon;Mexico;Netherlands;Poland;Russian Federation;Saudi Arabia;Spain;United Kingdom;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;Netherlands;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-PL   Australia;Belgium;Brazil;Canada;Germany;Hungary;Italy;Japan;Poland;Spain;Switzerland
      -   Phase 3   EUCTR2021-001294-23-DE   Belgium;Germany;Hungary;Italy;Japan;Poland;Spain;United States
      -   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 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;Türkiye;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   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
Nusinersen treatments
   NYU Langone Health
      2019   Early Phase 1   NCT04050852   United States
OAV101
   NOVARTIS PHARMA AG
      2022   Phase 4   EUCTR2021-006781-21-IT   Australia;Belgium;Brazil;Canada;China;Colombia;Denmark;Egypt;France;Germany;Greece;India;Italy;Japan;Malaysia;Mexico;Netherlands;Portugal;Saudi Arabia;Singapore;South Africa;Spain;Switzerland;Taiwan;Thailand;United Kingdom;United States;Viet Nam
      2021   Phase 3   EUCTR2020-005995-37-IT   Australia;Belgium;Canada;France;Germany;Italy;Portugal;Spain;Switzerland;Taiwan;United Kingdom;United States
   Novartis Pharma AG
      2023   Phase 3   EUCTR2021-006781-21-GR   Australia;Belgium;Brazil;Canada;China;Colombia;Denmark;Egypt;France;Germany;Greece;India;Italy;Japan;Malaysia;Mexico;Netherlands;Saudi Arabia;Singapore;South Africa;Spain;Switzerland;Taiwan;Thailand;United Kingdom;United States;Viet Nam
      2023   Phase 3   EUCTR2021-006781-21-DK   Australia;Belgium;Brazil;Canada;China;Colombia;Denmark;Egypt;France;Germany;Greece;India;Italy;Japan;Malaysia;Mexico;Netherlands;Saudi Arabia;Singapore;South Africa;Spain;Taiwan;Thailand;United Kingdom;United States;Viet Nam
      2023   Phase 3   EUCTR2021-006709-31-FR   Australia;Belgium;Canada;France;Germany;Italy;Japan;Netherlands;Spain;United States
      2023   Phase 3   EUCTR2021-006709-31-DE   Australia;Belgium;Canada;France;Germany;Italy;Japan;Netherlands;Spain;United States
      2023   Phase 3   EUCTR2021-003474-31-GR   Brazil;China;Colombia;Denmark;Egypt;Greece;India;Italy;Malaysia;Mexico;Russian Federation;Saudi Arabia;Singapore;South Africa;Taiwan;Thailand;United Arab Emirates;United States;Viet Nam
      2023   Phase 3   EUCTR2021-003474-31-DK   Brazil;China;Colombia;Denmark;Egypt;Greece;India;Italy;Malaysia;Mexico;Russian Federation;Saudi Arabia;Singapore;South Africa;Taiwan;Thailand;United Arab Emirates;United States;Viet Nam
      2022   Phase 4   EUCTR2021-006781-21-FR   Australia;Belgium;Brazil;Canada;China;Colombia;Denmark;Egypt;France;Germany;Greece;India;Italy;Japan;Malaysia;Mexico;Netherlands;Portugal;Saudi Arabia;Singapore;South Africa;Spain;Switzerland;Taiwan;Thailand;United Kingdom;United States;Viet Nam
      2022   Phase 3   EUCTR2021-006781-21-BE   Australia;Belgium;Brazil;Canada;China;Colombia;Denmark;Egypt;France;Germany;Greece;India;Italy;Japan;Malaysia;Mexico;Netherlands;Saudi Arabia;Singapore;South Africa;Spain;Taiwan;Thailand;United Kingdom;United States;Viet Nam
      2022   Phase 3   EUCTR2021-006709-31-NL   Australia;Belgium;Canada;France;Germany;Italy;Japan;Netherlands;Spain;United States
      2022   Phase 3   EUCTR2021-006709-31-BE   Australia;Belgium;Canada;France;Germany;Italy;Japan;Netherlands;Spain;United States
      2021   Phase 3   EUCTR2020-005995-37-PT   Australia;Belgium;Canada;France;Germany;Italy;Portugal;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;Switzerland;Taiwan;United Kingdom;United States
      -   Phase 4   EUCTR2021-006781-21-NL   Australia;Belgium;Brazil;Canada;China;Colombia;Denmark;Egypt;France;Germany;Greece;India;Italy;Japan;Malaysia;Mexico;Netherlands;Saudi Arabia;Singapore;South Africa;Spain;Switzerland;Taiwan;Thailand;United Kingdom;United States;Viet Nam
   Novartis Pharmaceuticals
      2023   Phase 3   NCT05386680   Australia;Belgium;Canada;France;Italy;Japan;Netherlands;Spain;United States
      2022   Phase 3   NCT05089656   Brazil;China;Colombia;Denmark;Egypt;Greece;India;Malaysia;Mexico;Saudi Arabia;Singapore;South Africa;Taiwan;Thailand;United States;Vietnam
      2021   Phase 4   NCT05073133   Argentina;Brazil
      2021   Phase 3   NCT04851873   Australia;Belgium;Canada;France;Italy;Portugal;Switzerland;Taiwan;United Kingdom;United States
Observational study TO examine safety, tolerability, and effectiveness OF spinraza® prescribed AS part OF standard OF care
   Washington University School of Medicine
      2018   -   NCT03709784   Canada;United States
Okrido 6MG/ML oral solution
   Novartis Pharma AG
      2023   Phase 3   EUCTR2021-003474-31-GR   Brazil;China;Colombia;Denmark;Egypt;Greece;India;Italy;Malaysia;Mexico;Russian Federation;Saudi Arabia;Singapore;South Africa;Taiwan;Thailand;United Arab Emirates;United States;Viet Nam
      2023   Phase 3   EUCTR2021-003474-31-DK   Brazil;China;Colombia;Denmark;Egypt;Greece;India;Italy;Malaysia;Mexico;Russian Federation;Saudi Arabia;Singapore;South Africa;Taiwan;Thailand;United Arab Emirates;United States;Viet Nam
Olesoxime
   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
   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
   TROPHOS SA
      2011   Phase 2   EUCTR2010-020386-24-GB   Belgium;France;Germany;Italy;Netherlands;United Kingdom
      2010   -   EUCTR2010-020386-24-NL   Belgium;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
      -   Phase 2   EUCTR2010-020386-24-FR   Belgium;France;Germany;Italy;Netherlands;United Kingdom
Onasemnogene abeparvovec
   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
      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 PHARMA AG
      2021   Phase 3   EUCTR2020-005995-37-IT   Australia;Belgium;Canada;France;Germany;Italy;Portugal;Spain;Switzerland;Taiwan;United Kingdom;United States
   Novartis Gene Therapies, Inc.
      2020   Phase 3   EUCTR2019-002611-26-BE   Australia;Belgium;Canada;France;Italy;Japan;Spain;Taiwan;United Kingdom;United States
      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
   Novartis Pharma AG
      2023   Phase 3   EUCTR2021-006709-31-FR   Australia;Belgium;Canada;France;Germany;Italy;Japan;Netherlands;Spain;United States
      2023   Phase 3   EUCTR2021-006709-31-DE   Australia;Belgium;Canada;France;Germany;Italy;Japan;Netherlands;Spain;United States
      2023   Phase 3   EUCTR2021-003474-31-GR   Brazil;China;Colombia;Denmark;Egypt;Greece;India;Italy;Malaysia;Mexico;Russian Federation;Saudi Arabia;Singapore;South Africa;Taiwan;Thailand;United Arab Emirates;United States;Viet Nam
      2023   Phase 3   EUCTR2021-003474-31-DK   Brazil;China;Colombia;Denmark;Egypt;Greece;India;Italy;Malaysia;Mexico;Russian Federation;Saudi Arabia;Singapore;South Africa;Taiwan;Thailand;United Arab Emirates;United States;Viet Nam
      2022   Phase 3   EUCTR2021-006709-31-NL   Australia;Belgium;Canada;France;Germany;Italy;Japan;Netherlands;Spain;United States
      2022   Phase 3   EUCTR2021-006709-31-BE   Australia;Belgium;Canada;France;Germany;Italy;Japan;Netherlands;Spain;United States
      2021   Phase 3   EUCTR2020-005995-37-PT   Australia;Belgium;Canada;France;Germany;Italy;Portugal;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;Switzerland;Taiwan;United Kingdom;United States
   Novartis Pharmaceuticals
      2023   -   NCT06019637   Brazil
      2022   Phase 3   NCT05335876   Australia;Belgium;Canada;China;Denmark;France;Italy;Japan;Malaysia;Netherlands;Saudi Arabia;Singapore;Spain;Taiwan;Thailand;United Kingdom;United States;Vietnam
Onasemnogene abeparvovec-xioi
   Novartis Gene Therapies
      2020   Phase 3   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   NCT03505099   Australia;Belgium;Canada;Germany;Israel;Italy;Japan;Korea, Republic of;Spain;Taiwan;United Kingdom;United States
      2017   -   NCT03421977   United States
      2017   Phase 1   NCT03381729   United States
ONE time survey
   Washington University School of Medicine
      2018   -   NCT03709784   Canada;United States
Orfiril saft
   University of Utah
      2009   -   EUCTR2008-003915-11-DE   Germany
Paracetamol
   Copenhagen Neuromuscular Center
      2018   Phase 1;Phase 4   EUCTR2018-002295-40-DK   Denmark
Paracetamol 120MG/5ML oral suspension
   Mette Cathrine Oerngreen
      2019   Phase 4   NCT03648658   Denmark
Phosphate
   CATALYST PHARMACEUTICALS INC.
      2018   Phase 2   EUCTR2018-000160-28-IT   Italy
Prednisolone
   NOVARTIS PHARMA AG
      2021   Phase 3   EUCTR2020-005995-37-IT   Australia;Belgium;Canada;France;Germany;Italy;Portugal;Spain;Switzerland;Taiwan;United Kingdom;United States
Prednisolone sodium phosphate
   Novartis Pharma AG
      2023   Phase 3   EUCTR2021-003474-31-GR   Brazil;China;Colombia;Denmark;Egypt;Greece;India;Italy;Malaysia;Mexico;Russian Federation;Saudi Arabia;Singapore;South Africa;Taiwan;Thailand;United Arab Emirates;United States;Viet Nam
Prednisolone sodium phosphate oral solution
   Novartis Pharma AG
      2023   Phase 3   EUCTR2021-003474-31-DK   Brazil;China;Colombia;Denmark;Egypt;Greece;India;Italy;Malaysia;Mexico;Russian Federation;Saudi Arabia;Singapore;South Africa;Taiwan;Thailand;United Arab Emirates;United States;Viet Nam
Pressure pain threshold
   LMU Klinikum
      2022   -   NCT05272969   Germany
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
      2023   Phase 3   EUCTR2021-006709-31-FR   Australia;Belgium;Canada;France;Germany;Italy;Japan;Netherlands;Spain;United States
      2023   Phase 3   EUCTR2021-006709-31-DE   Australia;Belgium;Canada;France;Germany;Italy;Japan;Netherlands;Spain;United States
      2023   Phase 3   EUCTR2021-003474-31-GR   Brazil;China;Colombia;Denmark;Egypt;Greece;India;Italy;Malaysia;Mexico;Russian Federation;Saudi Arabia;Singapore;South Africa;Taiwan;Thailand;United Arab Emirates;United States;Viet Nam
      2023   Phase 3   EUCTR2021-003474-31-DK   Brazil;China;Colombia;Denmark;Egypt;Greece;India;Italy;Malaysia;Mexico;Russian Federation;Saudi Arabia;Singapore;South Africa;Taiwan;Thailand;United Arab Emirates;United States;Viet Nam
      2022   Phase 3   EUCTR2021-006709-31-NL   Australia;Belgium;Canada;France;Germany;Italy;Japan;Netherlands;Spain;United States
      2022   Phase 3   EUCTR2021-006709-31-BE   Australia;Belgium;Canada;France;Germany;Italy;Japan;Netherlands;Spain;United States
      2021   Phase 3   EUCTR2020-005995-37-PT   Australia;Belgium;Canada;France;Germany;Italy;Portugal;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;Switzerland;Taiwan;United Kingdom;United States
Propofol
   The First Affiliated Hospital of Fujian Medical University
      2022   Phase 1   ChiCTR2200063672   China
Prospective observational registry
   Novartis Pharmaceuticals
      2018   -   NCT04174157   Greece;Ireland;Israel;Japan;Korea, Republic of;Poland;Portugal;Romania;Russian Federation;Taiwan;United States
Pyridostigmine
   UMC Utrecht
      2015   Phase 2   NCT02941328   Netherlands
Pyridostigmine bromide
   Centre Hospitalier Régional de la Citadelle
      2014   Phase 2   NCT02227823   Belgium
   Universtiy Medical Center Utrecht
      2015   Phase 2   EUCTR2011-004369-34-NL   Netherlands
Questionnaires
   Hospices Civils de Lyon
      2024   -   NCT06321965   France
Quick motor function test
   LMU Klinikum
      2022   -   NCT05272969   Germany
Reldesemtiv 150 MG
   Cytokinetics
      2016   Phase 2   NCT02644668   Canada;United States
Reldesemtiv 450 MG
   Cytokinetics
      2016   Phase 2   NCT02644668   Canada;United States
Riluzole
   Assistance Publique - Hôpitaux de Paris
      2006   Phase 2/Phase 3   NCT00774423   France
Risdiplam
   Bakri Elsheikh
      2021   -   NCT05219487   United States
   CHUGAI PHARMACEUTICAL CO., LTD.
      2018   Phase 2   JPRN-jRCT2080223835   Asia except Japan;Europe;Japan;North America;South America
      2018   Phase 2   JPRN-jRCT2080223666   Asia except Japan;Europe;Japan;North America;South America
   Clinic for Special Children
      2022   Phase 4   NCT05522361   United States
   F. HOFFMANN - LA ROCHE LTD.
      2022   Phase 2;Phase 3   EUCTR2021-003417-19-IT   Belgium;France;Germany;Italy;Netherlands;Poland;Portugal;Spain;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
      2019   Phase 2   EUCTR2016-004184-39-BE   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   EUCTR2016-000750-35-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
      2023   Phase 2;Phase 3   EUCTR2021-003417-19-PT   Belgium;Croatia;France;Germany;Italy;Netherlands;Poland;Portugal;Spain;United Kingdom;United States
      2022   Phase 2;Phase 3   EUCTR2021-003417-19-PL   Belgium;France;Germany;Italy;Netherlands;Poland;Portugal;Spain;United Kingdom;United States
      2022   Phase 2;Phase 3   EUCTR2021-003417-19-NL   Belgium;France;Germany;Italy;Netherlands;Poland;Portugal;Spain;United Kingdom;United States
      2022   Phase 2;Phase 3   EUCTR2021-003417-19-BE   Belgium;Germany;Italy;Netherlands;Poland;Portugal;Spain;United Kingdom;United States
      -   Phase 2;Phase 3   EUCTR2021-003417-19-DE   Belgium;Germany;Italy;Netherlands;Poland;Portugal;Spain;United Kingdom;United States
   Genentech, Inc.
      2022   Phase 4   NCT05232929   Puerto Rico;United States
      2014   -   NCT04256265   United States
   Hoffmann-La Roche
      2024   Phase 4   NCT05861999   Germany;Israel;Poland;Qatar;United Kingdom;United States
      2024   Phase 4   NCT05861986   Germany;Poland;United Kingdom;United States
      2024   Phase 2   NCT05808764   Belgium;Canada;Germany;Italy;Netherlands;Norway;Poland;United States
      2022   Phase 2/Phase 3   NCT05115110   Australia;Belgium;Canada;Croatia;Germany;Italy;Japan;Netherlands;Poland;Portugal;Spain;United Kingdom;United States
      2019   Phase 1   NCT03988907   United States
      2019   Phase 2   NCT03779334   Australia;Belgium;Brazil;Canada;China;Italy;Poland;Russian Federation;Saudi Arabia;Taiwan;United States
      2017   Phase 2   NCT03032172   Belgium;France;Germany;Italy;Netherlands;Poland;Switzerland;United Kingdom;United States
      2016   Phase 2   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   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
   Renata Scalco
      2024   Phase 2-3   JPRN-jRCT2051230159   Belgium;Canada;Croatia;Italy;Japan;Netherlands;Poland;Portugal;Spain;United Kingdom;United States
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-jRCT2080223835   Asia except Japan;Europe;Japan;North America;South America
      2018   Phase 2   JPRN-jRCT2080223666   Asia except Japan;Europe;Japan;North America;South America
   F. HOFFMANN - LA ROCHE LTD.
      2022   Phase 2;Phase 3   EUCTR2021-003417-19-IT   Belgium;France;Germany;Italy;Netherlands;Poland;Portugal;Spain;United Kingdom;United States
   F. Hoffmann-La Roche Ltd
      2017   Phase 2   EUCTR2016-000778-40-FR   Belgium;Croatia;France;Germany;Italy;Poland;Spain
      2016   Phase 2   EUCTR2016-000778-40-IT   Belgium;Croatia;France;Germany;Italy;Poland;Spain
      2016   Phase 2   EUCTR2016-000750-35-IT   Australia;Belgium;Bulgaria;Canada;Croatia;France;Germany;Italy;Netherlands;Poland;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
   F.Hoffmann-La Roche Ltd
      2023   Phase 2;Phase 3   EUCTR2021-003417-19-PT   Belgium;Croatia;France;Germany;Italy;Netherlands;Poland;Portugal;Spain;United Kingdom;United States
      2022   Phase 2;Phase 3   EUCTR2021-003417-19-PL   Belgium;France;Germany;Italy;Netherlands;Poland;Portugal;Spain;United Kingdom;United States
      2022   Phase 2;Phase 3   EUCTR2021-003417-19-NL   Belgium;France;Germany;Italy;Netherlands;Poland;Portugal;Spain;United Kingdom;United States
      2022   Phase 2;Phase 3   EUCTR2021-003417-19-BE   Belgium;Germany;Italy;Netherlands;Poland;Portugal;Spain;United Kingdom;United States
      -   Phase 2;Phase 3   EUCTR2021-003417-19-DE   Belgium;Germany;Italy;Netherlands;Poland;Portugal;Spain;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   Belgium;France;Germany;Italy;Netherlands;Poland;Switzerland;United Kingdom;United States
RO7034067/F07 + solvente (RO
   F. HOFFMANN - LA ROCHE LTD.
      2017   Phase 2   EUCTR2016-004184-39-IT   Belgium;France;Germany;Italy;Netherlands;Poland;Switzerland;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
      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-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
      2019   Phase 2   EUCTR2016-004184-39-BE   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   EUCTR2016-000750-35-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
RO72042369/F03-01
   F.Hoffmann-La Roche Ltd
      2023   Phase 2;Phase 3   EUCTR2021-003417-19-PT   Belgium;Croatia;France;Germany;Italy;Netherlands;Poland;Portugal;Spain;United Kingdom;United States
RO7204239
   F.Hoffmann-La Roche Ltd
      2023   Phase 2;Phase 3   EUCTR2021-003417-19-FR   Belgium;Croatia;France;Germany;Italy;Netherlands;Poland;Portugal;Spain;United Kingdom;United States
   Hoffmann-La Roche
      2022   Phase 2/Phase 3   NCT05115110   Australia;Belgium;Canada;Croatia;Germany;Italy;Japan;Netherlands;Poland;Portugal;Spain;United Kingdom;United States
   Renata Scalco
      2024   Phase 2-3   JPRN-jRCT2051230159   Belgium;Canada;Croatia;Italy;Japan;Netherlands;Poland;Portugal;Spain;United Kingdom;United States
RO7204239/F01-01
   F. HOFFMANN - LA ROCHE LTD.
      2022   Phase 2;Phase 3   EUCTR2021-003417-19-IT   Belgium;France;Germany;Italy;Netherlands;Poland;Portugal;Spain;United Kingdom;United States
   F.Hoffmann-La Roche Ltd
      2023   Phase 2;Phase 3   EUCTR2021-003417-19-PT   Belgium;Croatia;France;Germany;Italy;Netherlands;Poland;Portugal;Spain;United Kingdom;United States
      2022   Phase 2;Phase 3   EUCTR2021-003417-19-PL   Belgium;France;Germany;Italy;Netherlands;Poland;Portugal;Spain;United Kingdom;United States
      2022   Phase 2;Phase 3   EUCTR2021-003417-19-NL   Belgium;France;Germany;Italy;Netherlands;Poland;Portugal;Spain;United Kingdom;United States
      2022   Phase 2;Phase 3   EUCTR2021-003417-19-BE   Belgium;Germany;Italy;Netherlands;Poland;Portugal;Spain;United Kingdom;United States
      -   Phase 2;Phase 3   EUCTR2021-003417-19-DE   Belgium;Germany;Italy;Netherlands;Poland;Portugal;Spain;United Kingdom;United States
RO7204239/F03-01
   F.Hoffmann-La Roche Ltd
      2022   Phase 2;Phase 3   EUCTR2021-003417-19-PL   Belgium;France;Germany;Italy;Netherlands;Poland;Portugal;Spain;United Kingdom;United States
      2022   Phase 2;Phase 3   EUCTR2021-003417-19-BE   Belgium;Germany;Italy;Netherlands;Poland;Portugal;Spain;United Kingdom;United States
      -   Phase 2;Phase 3   EUCTR2021-003417-19-DE   Belgium;Germany;Italy;Netherlands;Poland;Portugal;Spain;United Kingdom;United States
RVG 03820
   Universtiy Medical Center Utrecht
      2015   Phase 2   EUCTR2011-004369-34-NL   Netherlands
Salanersen
   Biogen
      2022   Phase 1   NCT05575011   Belgium;Canada;France;Germany;Israel;Italy;Korea, Republic of;Netherlands;Poland;United Kingdom
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
Second-line cognitive assessment
   Hospices Civils de Lyon
      2024   -   NCT06321965   France
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
Sham control
   Novartis Pharmaceuticals
      2022   Phase 3   NCT05089656   Brazil;China;Colombia;Denmark;Egypt;Greece;India;Malaysia;Mexico;Saudi Arabia;Singapore;South Africa;Taiwan;Thailand;United States;Vietnam
Sham procedure
   Berger Zdenek
      2021   Phase 2-3   JPRN-jRCT2061200040   Colombia;Estonia;Ireland;Japan;Latvia;Spain;Taiwan;United States
   Biogen
      2015   Phase 2   NCT02462759   Germany;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
SKG0201 injection
   Kun Sun
      2023   -   NCT06191354   China
Skinfold measurement
   Hospices Civils de Lyon
      2024   -   NCT06321965   France
SMA-DAT application and adam sensor
   Genentech, Inc.
      2022   Phase 4   NCT05232929   Puerto Rico;United States
Sodium phenylbutyrate
   University of Utah
      2007   Phase 1/Phase 2   NCT00528268   United States
   Westat
      2008   Phase 1/Phase 2   NCT00439569   United States
      2008   Phase 1/Phase 2   NCT00439218   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
      2022   Phase 3   EUCTR2021-001294-23-IT   Australia;Belgium;Brazil;Canada;Germany;Hungary;Italy;Japan;Poland;Spain;Switzerland
      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;Netherlands;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
      2022   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
      2021   Phase 4   EUCTR2020-003492-18-ES   Germany;Israel;Italy;Spain;United States
      2021   Phase 4   EUCTR2020-003492-18-DE   Germany;Israel;Italy;Japan;Spain;United States
      2021   Phase 2;Phase 3   EUCTR2019-002663-10-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;Türkiye;United Kingdom;United States
      2020   Phase 2;Phase 3   EUCTR2019-002663-10-LV   Argentina;Australia;Brazil;Canada;Chile;Colombia;Estonia;France;Germany;Greece;Hungary;Ireland;Italy;Korea, Republic of;Latvia;Lebanon;Mexico;Netherlands;Poland;Russian Federation;Saudi Arabia;Spain;United Kingdom;United States
      2020   Phase 2;Phase 3   EUCTR2019-002663-10-HU   Argentina;Australia;Brazil;Canada;Chile;Colombia;Estonia;France;Germany;Greece;Hungary;Ireland;Italy;Korea, Republic of;Latvia;Lebanon;Mexico;Netherlands;Poland;Russian Federation;Saudi Arabia;Spain;United Kingdom;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;Netherlands;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;Türkiye;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;Türkiye;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   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
   Irzhevsky Victoria
      2022   Phase 3   JPRN-jRCT2031220035   Australia;Canada;Estonia;France;Germany;Greece;Hungary;Ireland;Israel;Italy;Japan;Latvia;Lebanon;Poland;Russia;South Korea;Spain;Taiwan;Turkey;United Kingdom;United States
   Toda Yasuo
      2022   Phase 4   JPRN-jRCT2051210186   Germany;Israel;Italy;Japan;Spain;United States
   Tsuda Hiroshi
      2024   Phase 3   JPRN-jRCT2051230096   Belgium;Brazil;Germany;Hungary;Italy;Japan;Poland;Spain;USA
SRK-015
   Scholar Rock, Inc.
      2023   Phase 3   EUCTR2022-001771-14-FR   Belgium;France;Germany;Italy;Netherlands;Poland;Spain;United Kingdom;United States
      2023   Phase 3   EUCTR2022-001771-14-BE   Belgium;France;Germany;Italy;Netherlands;Poland;Spain;United Kingdom;United States
      2022   Phase 3   EUCTR2021-005314-34-NL   Belgium;France;Germany;Italy;Netherlands;Poland;Spain;United Kingdom;United States
      2022   Phase 3   EUCTR2021-005314-34-IT   Belgium;France;Germany;Italy;Netherlands;Poland;Spain;United Kingdom;United States
      2022   Phase 3   EUCTR2021-005314-34-FR   Belgium;France;Germany;Italy;Netherlands;Poland;Spain;United Kingdom;United States
      2022   Phase 3   EUCTR2021-005314-34-ES   Belgium;France;Germany;Italy;Netherlands;Poland;Spain;United Kingdom;United States
      2022   Phase 3   EUCTR2021-005314-34-DE   Belgium;France;Germany;Italy;Netherlands;Poland;Spain;United Kingdom;United States
      2022   Phase 3   EUCTR2021-005314-34-BE   Belgium;France;Germany;Italy;Netherlands;Poland;Spain;United Kingdom;United States
      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   EUCTR2022-001771-14-PL   Belgium;France;Germany;Italy;Netherlands;Poland;Spain;United Kingdom;United States
      -   Phase 3   EUCTR2022-001771-14-NL   Belgium;France;Germany;Italy;Netherlands;Poland;Spain;United Kingdom;United States
      -   Phase 3   EUCTR2022-001771-14-DE   Belgium;France;Germany;Italy;Netherlands;Poland;Spain;United Kingdom;United States
      -   Phase 3   EUCTR2021-005314-34-PL   Belgium;France;Germany;Italy;Netherlands;Poland;Spain;United Kingdom;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-IT   Argentina;Australia;Germany;Israel;Italy;Qatar;Taiwan;Turkey;United Kingdom;United States
      2015   Phase 2   EUCTR2014-002098-12-DE   Australia;Canada;Germany;Italy;Qatar;Taiwan;Türkiye;United Kingdom;United States
   Ionis Pharmaceuticals, Inc.
      2016   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
      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 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
Taldefgrobep alfa
   Biohaven Pharmaceuticals, Inc
      2023   Phase 3   EUCTR2022-000193-25-DE   Belgium;Czech Republic;France;Germany;Italy;Poland;Spain
      2023   Phase 3   EUCTR2022-000193-25-CZ   Belgium;Czech Republic;France;Germany;Italy;Poland;Spain
   Biohaven Pharmaceuticals, Inc.
      2022   Phase 3   NCT05337553   Belgium;Czechia;France;Germany;Italy;Netherlands;Poland;Spain;United Kingdom;United States
      2022   Phase 3   EUCTR2022-000193-25-IT   Belgium;Czech Republic;France;Germany;Italy;Poland;Spain;United Kingdom;United States
      -   Phase 3   EUCTR2022-000193-25-PL   Belgium;Czech Republic;Czechia;France;Germany;Italy;Netherlands;Poland;Spain;United Kingdom;United States
Taldefgrobep alfa
   Biohaven Pharmaceuticals, Inc.
      2023   Phase 3   EUCTR2022-000193-25-BE   Belgium;Czech Republic;Czechia;France;Germany;Italy;Netherlands;Poland;Spain;United Kingdom;United States
Testosterone
   National Center for Research Resources (NCRR)
      1992   Phase 2   NCT00004771   -
Thecaflex DRX system
   Biogen
      2025   Phase 1   NCT06555419   United States
TRO19622
   TROPHOS
      2010   -   EUCTR2010-020386-24-IT   Belgium;Germany;Italy;Netherlands;United Kingdom
      2007   Phase 1   EUCTR2006-006845-14-FR   France
   TROPHOS SA
      2011   Phase 2   EUCTR2010-020386-24-GB   Belgium;France;Germany;Italy;Netherlands;United Kingdom
      2010   -   EUCTR2010-020386-24-NL   Belgium;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
      -   Phase 2   EUCTR2010-020386-24-FR   Belgium;France;Germany;Italy;Netherlands;United Kingdom
Valproate sodium
   University of Utah
      2009   -   EUCTR2008-003915-11-DE   Germany
Valproate, levocarnitine
   All India Institute of Medical Sciences, New Delhi
      2013   Phase 3   NCT01671384   India
Valproic acid
   University of Utah
      2007   Phase 2   NCT00481013   United States
      2003   Phase 1   NCT00374075   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
Vesemnogene lantuparvovec
   Lantu Biopharma
      2024   Phase 1/Phase 2   NCT06288230   China
Vital signs
   LMU Klinikum
      2022   -   NCT05272969   Germany
VPA
   University of Utah
      2007   Phase 2   NCT00481013   United States
Zolgensma
   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
      2023   Phase 3   EUCTR2021-006709-31-FR   Australia;Belgium;Canada;France;Germany;Italy;Japan;Netherlands;Spain;United States
      2023   Phase 3   EUCTR2021-006709-31-DE   Australia;Belgium;Canada;France;Germany;Italy;Japan;Netherlands;Spain;United States
      2023   Phase 3   EUCTR2021-003474-31-GR   Brazil;China;Colombia;Denmark;Egypt;Greece;India;Italy;Malaysia;Mexico;Russian Federation;Saudi Arabia;Singapore;South Africa;Taiwan;Thailand;United Arab Emirates;United States;Viet Nam
      2023   Phase 3   EUCTR2021-003474-31-DK   Brazil;China;Colombia;Denmark;Egypt;Greece;India;Italy;Malaysia;Mexico;Russian Federation;Saudi Arabia;Singapore;South Africa;Taiwan;Thailand;United Arab Emirates;United States;Viet Nam
      2022   Phase 3   EUCTR2021-006709-31-NL   Australia;Belgium;Canada;France;Germany;Italy;Japan;Netherlands;Spain;United States
      2022   Phase 3   EUCTR2021-006709-31-BE   Australia;Belgium;Canada;France;Germany;Italy;Japan;Netherlands;Spain;United States
      2021   Phase 3   EUCTR2020-005995-37-FR   Australia;Belgium;Canada;France;Germany;Italy;Portugal;Spain;Switzerland;Taiwan;United Kingdom;United States
   Novartis Pharmaceuticals
      2018   -   NCT04174157   Greece;Ireland;Israel;Japan;Korea, Republic of;Poland;Portugal;Romania;Russian Federation;Taiwan;United States
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;Switzerland;Taiwan;United Kingdom;United States
      2021   Phase 3   EUCTR2020-005995-37-BE   Australia;Belgium;Canada;France;Germany;Italy;Portugal;Switzerland;Taiwan;United Kingdom;United States
Zolgensma 2 X 1013 VG (genomi vettoriali)/ML soluzione PER infusione
   NOVARTIS PHARMA AG
      2022   Phase 4   EUCTR2021-006781-21-IT   Australia;Belgium;Brazil;Canada;China;Colombia;Denmark;Egypt;France;Germany;Greece;India;Italy;Japan;Malaysia;Mexico;Netherlands;Portugal;Saudi Arabia;Singapore;South Africa;Spain;Switzerland;Taiwan;Thailand;United Kingdom;United States;Viet Nam
Zolgensma 2 X 1013 VG (vector genomes)/ML solution FOR infusion
   Novartis Pharma AG
      2023   Phase 3   EUCTR2021-006781-21-GR   Australia;Belgium;Brazil;Canada;China;Colombia;Denmark;Egypt;France;Germany;Greece;India;Italy;Japan;Malaysia;Mexico;Netherlands;Saudi Arabia;Singapore;South Africa;Spain;Switzerland;Taiwan;Thailand;United Kingdom;United States;Viet Nam
      2023   Phase 3   EUCTR2021-006781-21-DK   Australia;Belgium;Brazil;Canada;China;Colombia;Denmark;Egypt;France;Germany;Greece;India;Italy;Japan;Malaysia;Mexico;Netherlands;Saudi Arabia;Singapore;South Africa;Spain;Taiwan;Thailand;United Kingdom;United States;Viet Nam
      2022   Phase 4   EUCTR2021-006781-21-FR   Australia;Belgium;Brazil;Canada;China;Colombia;Denmark;Egypt;France;Germany;Greece;India;Italy;Japan;Malaysia;Mexico;Netherlands;Portugal;Saudi Arabia;Singapore;South Africa;Spain;Switzerland;Taiwan;Thailand;United Kingdom;United States;Viet Nam
      2022   Phase 3   EUCTR2021-006781-21-BE   Australia;Belgium;Brazil;Canada;China;Colombia;Denmark;Egypt;France;Germany;Greece;India;Italy;Japan;Malaysia;Mexico;Netherlands;Saudi Arabia;Singapore;South Africa;Spain;Taiwan;Thailand;United Kingdom;United States;Viet Nam
      -   Phase 4   EUCTR2021-006781-21-NL   Australia;Belgium;Brazil;Canada;China;Colombia;Denmark;Egypt;France;Germany;Greece;India;Italy;Japan;Malaysia;Mexico;Netherlands;Saudi Arabia;Singapore;South Africa;Spain;Switzerland;Taiwan;Thailand;United Kingdom;United States;Viet Nam