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20 Trials

   

Trial + Data / EventsStatusPhNRegionInterventionsSponsorConditionsPrimary complStudy compl
Evrysdi (risdiplam) / Roche
RISE, NCT05522361: Risdiplam in Patients With Spinal Muscular Atrophy Previously Treated With Nusinersen

Active, not recruiting
4
10
US
Risdiplam
Clinic for Special Children, Genentech, Inc.
Spinal Muscular Atrophy
12/25
06/26
ChiCTR2300072288: Effectiveness and Safety of Risdiplam in Chinese Patients with Spinal Muscular Atrophy (SMA): a Real-World, Multi-Center Cohort Study

Recruiting
4
60
 
NA
Peking University First Hospital; Peking University First Hospital, Shanghai Roche Pharmaceuticals Co., Ltd.
Spinal Muscular Atrophy (SMA)
 
 
HINALEA 1, NCT05861986: A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy

Recruiting
4
28
Europe, US
risdiplam, RO7034067
Hoffmann-La Roche
Muscular Atrophy, Spinal
01/27
03/28
HINALEA 2, NCT05861999: A Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Muscular Atrophy Who Experienced a Plateau or Decline in Function After Gene Therapy

Recruiting
4
28
Europe, US, RoW
risdiplam, RO7034067
Hoffmann-La Roche
Muscular Atrophy, Spinal
01/27
03/28
WeSMA, NCT05232929: Long-term Follow-up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA)

Active, not recruiting
4
402
US
Risdiplam, Evrysdi®, SMA-DAT Application and ADAM Sensor
Genentech, Inc.
Spinal Muscular Atrophy
12/26
12/26
2021-006709-31: Phase IIIb, open-label, multi-center study to evaluate safety, tolerability and efficacy study of OAV101 administered intrathecally to participants with spinal muscular atrophy (SMA) who have discontinued treatment with nusinersen or risdiplam

Ongoing
3
28
Europe
OAV101, OAV101, Solution for injection, Zolgensma
Novartis Pharma AG, Novartis Pharma AG
Spinal Muscular Atrophy, Spinal Muscular Atrophy, Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
 
 
SAPPHIRE, NCT05156320 / 2021-005314-34: Efficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinersen or Risdiplam

Completed
3
188
Europe, US
Apitegromab, SRK-015, Placebo
Scholar Rock, Inc.
Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3, Spinal Muscular Atrophy Type 2, SMA, Neuromuscular Diseases, Muscular Atrophy, Atrophy, Muscular Atrophy, Spinal, Neuromuscular Manifestations, Anti-myostatin
12/24
12/24
STRENGTH, NCT05386680: Phase IIIb, Open-label, Multi-center Study to Evaluate Safety, Tolerability and Efficacy of OAV101 Administered Intrathecally to Participants With SMA Who Discontinued Treatment With Nusinersen or Risdiplam

Completed
3
27
Europe, Canada, Japan, US, RoW
OAV101, AVXS-101, Zolgensma
Novartis Pharmaceuticals
Spinal Muscular Atrophy
11/24
11/24
ASCEND, NCT05067790 / 2021-001294-23: A Study to Learn About the Effect of Higher Doses of Nusinersen (BIIB058) Given as Injections to Participants With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Risdiplam

Active, not recruiting
3
45
Europe, Japan, US, RoW
Nusinersen, BIIB058, Spinraza
Biogen
Spinal Muscular Atrophy
06/27
06/27
MANATEE, NCT05115110 / 2021-003417-19: A Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Participants With Spinal Muscular Atrophy

Active, not recruiting
2/3
259
Europe, Canada, Japan, US, RoW
RO7204239, Placebo, Risdiplam, RO7034067, Evrysdi
Hoffmann-La Roche
Spinal Muscular Atrophy (SMA)
02/29
02/29
SUNFISH, NCT02908685 / 2016-000750-35: A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy (SMA) Participants

Completed
2
231
Europe, Canada, Japan, US, RoW
Placebo, Risdiplam, RO7034067
Hoffmann-La Roche
Muscular Atrophy, Spinal
09/19
10/23
FIREFISH, NCT02913482 / 2016-000778-40: Investigate Safety, Tolerability, PK, PD and Efficacy of Risdiplam (RO7034067) in Infants With Type1 Spinal Muscular Atrophy

Completed
2
62
Europe, Japan, US, RoW
Risdiplam, RO7034067, Evrysdi
Hoffmann-La Roche
Muscular Atrophy, Spinal
11/19
12/23
Rainbowfish, NCT03779334 / 2018-002087-12: A Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy

Active, not recruiting
2
26
Europe, US, RoW
Risdiplam, Evrysdi
Hoffmann-La Roche
Muscular Atrophy, Spinal
02/23
03/27
Jewelfish, NCT03032172 / 2016-004184-39: A Study of Risdiplam (RO7034067) in Adult and Pediatric Participants With Spinal Muscular Atrophy

Completed
2
174
Europe, US
Risdiplam, RO7034067
Hoffmann-La Roche
Spinal Muscular Atrophy
02/25
02/25
Pupfish, NCT05808764: A Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy

Recruiting
2
10
Europe, Canada, US
Risdiplam, Evrysdi
Hoffmann-La Roche
Muscular Atrophy, Spinal
10/25
10/25
OPAL, NCT07047144: A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy

Not yet recruiting
2
52
NA
Apitegromab, SRK-015, Nusinersen, Spinraza, Risdiplam, Evrysdi
Scholar Rock, Inc.
Spinal Muscular Atrophy, SMA, Spinal Muscular Atrophy Type 2, Spinal Muscular Atrophy Type 3, Neuromuscular Manifestations, Anti-myostatin
11/28
03/29
NCT04718181: Bioavailability and Bioequivalence of Two Risdiplam Tablets in Healthy Participants

Completed
1
131
US
risdiplam, Evrysdi, omeprazole, Non-investigational medicinal product
Hoffmann-La Roche
Muscular Atrophy, Spinal
01/23
01/23
REACH, NCT06978985: Adult SMA Research and Clinical Hub

Recruiting
N/A
600
Europe
Risdiplam, Evrysdi, Nusinersen Injectable Product, Spinraza
Newcastle-upon-Tyne Hospitals NHS Trust, Newcastle University, John Walton Muscular Dystrophy Research Centre, Biogen, Roche Products Limited
Spinal Muscular Atrophy (SMA)
04/27
04/27
NCT05219487: Investigating NMJ Defects in SMA Following Central and Peripheral SMN Restoration

Recruiting
N/A
30
US
Risdiplam, Evrysdi®
Bakri Elsheikh, Genentech, Inc.
Spinal Muscular Atrophy
12/25
12/25
NCT04888702: Holter of Movement in Patients with SMA Undergoing Treatment.

Active, not recruiting
N/A
30
Europe
Actimyo
Centre Hospitalier Universitaire de Liege, SYSNAV, Erasme University Hospital, Queen Fabiola Children's University Hospital, Centre Hospitalier Régional de la Citadelle
Spinal Muscular Atrophy
01/25
05/25
Trial + Data / EventsStatusPhNRegionInterventionsSponsorConditionsPrimary complStudy compl
Evrysdi (risdiplam) / Roche
RISE, NCT05522361: Risdiplam in Patients With Spinal Muscular Atrophy Previously Treated With Nusinersen

Active, not recruiting
4
10
US
Risdiplam
Clinic for Special Children, Genentech, Inc.
Spinal Muscular Atrophy
12/25
06/26
ChiCTR2300072288: Effectiveness and Safety of Risdiplam in Chinese Patients with Spinal Muscular Atrophy (SMA): a Real-World, Multi-Center Cohort Study

Recruiting
4
60
 
NA
Peking University First Hospital; Peking University First Hospital, Shanghai Roche Pharmaceuticals Co., Ltd.
Spinal Muscular Atrophy (SMA)
 
 
HINALEA 1, NCT05861986: A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy

Recruiting
4
28
Europe, US
risdiplam, RO7034067
Hoffmann-La Roche
Muscular Atrophy, Spinal
01/27
03/28
HINALEA 2, NCT05861999: A Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Muscular Atrophy Who Experienced a Plateau or Decline in Function After Gene Therapy

Recruiting
4
28
Europe, US, RoW
risdiplam, RO7034067
Hoffmann-La Roche
Muscular Atrophy, Spinal
01/27
03/28
WeSMA, NCT05232929: Long-term Follow-up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA)

Active, not recruiting
4
402
US
Risdiplam, Evrysdi®, SMA-DAT Application and ADAM Sensor
Genentech, Inc.
Spinal Muscular Atrophy
12/26
12/26
2021-006709-31: Phase IIIb, open-label, multi-center study to evaluate safety, tolerability and efficacy study of OAV101 administered intrathecally to participants with spinal muscular atrophy (SMA) who have discontinued treatment with nusinersen or risdiplam

Ongoing
3
28
Europe
OAV101, OAV101, Solution for injection, Zolgensma
Novartis Pharma AG, Novartis Pharma AG
Spinal Muscular Atrophy, Spinal Muscular Atrophy, Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
 
 
SAPPHIRE, NCT05156320 / 2021-005314-34: Efficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinersen or Risdiplam

Completed
3
188
Europe, US
Apitegromab, SRK-015, Placebo
Scholar Rock, Inc.
Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3, Spinal Muscular Atrophy Type 2, SMA, Neuromuscular Diseases, Muscular Atrophy, Atrophy, Muscular Atrophy, Spinal, Neuromuscular Manifestations, Anti-myostatin
12/24
12/24
STRENGTH, NCT05386680: Phase IIIb, Open-label, Multi-center Study to Evaluate Safety, Tolerability and Efficacy of OAV101 Administered Intrathecally to Participants With SMA Who Discontinued Treatment With Nusinersen or Risdiplam

Completed
3
27
Europe, Canada, Japan, US, RoW
OAV101, AVXS-101, Zolgensma
Novartis Pharmaceuticals
Spinal Muscular Atrophy
11/24
11/24
ASCEND, NCT05067790 / 2021-001294-23: A Study to Learn About the Effect of Higher Doses of Nusinersen (BIIB058) Given as Injections to Participants With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Risdiplam

Active, not recruiting
3
45
Europe, Japan, US, RoW
Nusinersen, BIIB058, Spinraza
Biogen
Spinal Muscular Atrophy
06/27
06/27
MANATEE, NCT05115110 / 2021-003417-19: A Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Participants With Spinal Muscular Atrophy

Active, not recruiting
2/3
259
Europe, Canada, Japan, US, RoW
RO7204239, Placebo, Risdiplam, RO7034067, Evrysdi
Hoffmann-La Roche
Spinal Muscular Atrophy (SMA)
02/29
02/29
SUNFISH, NCT02908685 / 2016-000750-35: A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy (SMA) Participants

Completed
2
231
Europe, Canada, Japan, US, RoW
Placebo, Risdiplam, RO7034067
Hoffmann-La Roche
Muscular Atrophy, Spinal
09/19
10/23
FIREFISH, NCT02913482 / 2016-000778-40: Investigate Safety, Tolerability, PK, PD and Efficacy of Risdiplam (RO7034067) in Infants With Type1 Spinal Muscular Atrophy

Completed
2
62
Europe, Japan, US, RoW
Risdiplam, RO7034067, Evrysdi
Hoffmann-La Roche
Muscular Atrophy, Spinal
11/19
12/23
Rainbowfish, NCT03779334 / 2018-002087-12: A Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy

Active, not recruiting
2
26
Europe, US, RoW
Risdiplam, Evrysdi
Hoffmann-La Roche
Muscular Atrophy, Spinal
02/23
03/27
Jewelfish, NCT03032172 / 2016-004184-39: A Study of Risdiplam (RO7034067) in Adult and Pediatric Participants With Spinal Muscular Atrophy

Completed
2
174
Europe, US
Risdiplam, RO7034067
Hoffmann-La Roche
Spinal Muscular Atrophy
02/25
02/25
Pupfish, NCT05808764: A Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy

Recruiting
2
10
Europe, Canada, US
Risdiplam, Evrysdi
Hoffmann-La Roche
Muscular Atrophy, Spinal
10/25
10/25
OPAL, NCT07047144: A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy

Not yet recruiting
2
52
NA
Apitegromab, SRK-015, Nusinersen, Spinraza, Risdiplam, Evrysdi
Scholar Rock, Inc.
Spinal Muscular Atrophy, SMA, Spinal Muscular Atrophy Type 2, Spinal Muscular Atrophy Type 3, Neuromuscular Manifestations, Anti-myostatin
11/28
03/29
NCT04718181: Bioavailability and Bioequivalence of Two Risdiplam Tablets in Healthy Participants

Completed
1
131
US
risdiplam, Evrysdi, omeprazole, Non-investigational medicinal product
Hoffmann-La Roche
Muscular Atrophy, Spinal
01/23
01/23
REACH, NCT06978985: Adult SMA Research and Clinical Hub

Recruiting
N/A
600
Europe
Risdiplam, Evrysdi, Nusinersen Injectable Product, Spinraza
Newcastle-upon-Tyne Hospitals NHS Trust, Newcastle University, John Walton Muscular Dystrophy Research Centre, Biogen, Roche Products Limited
Spinal Muscular Atrophy (SMA)
04/27
04/27
NCT05219487: Investigating NMJ Defects in SMA Following Central and Peripheral SMN Restoration

Recruiting
N/A
30
US
Risdiplam, Evrysdi®
Bakri Elsheikh, Genentech, Inc.
Spinal Muscular Atrophy
12/25
12/25
NCT04888702: Holter of Movement in Patients with SMA Undergoing Treatment.

Active, not recruiting
N/A
30
Europe
Actimyo
Centre Hospitalier Universitaire de Liege, SYSNAV, Erasme University Hospital, Queen Fabiola Children's University Hospital, Centre Hospitalier Régional de la Citadelle
Spinal Muscular Atrophy
01/25
05/25

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