Hoffmann-La Roche: Muscular Atrophy, Spinal trials

The registry lists 18 Muscular Atrophy, Spinal studies with Hoffmann-La Roche as lead sponsor. 1 are open to participants now and 7 have results posted.

Phase 1 5Phase 2 7Phase 3 1Phase 4 3No phase 3(a study in two phases counts in both)

Studies

Open to participants first, then studies with results posted, then the rest; newest registration first within each.

• A Study of Risdiplam in Participants With Type I and Type II Spinal Muscle Atrophy (SMA)RecruitingNCT07531719
• A Drug-drug Interaction Study With Risdiplam Multiple Dose and Midazolam in Healthy ParticipantsCompletedNCT03988907Phase 1Results posted
• A Study to Investigate the Effect of Hepatic Impairment on the Pharmacokinetics and Safety and Tolerability of a Single Oral Dose of Risdiplam Compared to Matched Healthy Participants With Normal Hepatic FunctionCompletedNCT03920865Phase 1Results posted
• A Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular AtrophyActive, not recruitingNCT03779334Phase 2Results posted
• A Study of Risdiplam (RO7034067) in Adult and Pediatric Participants With Spinal Muscular AtrophyCompletedNCT03032172Phase 2Results posted
• Investigate Safety, Tolerability, PK, PD and Efficacy of Risdiplam (RO7034067) in Infants With Type1 Spinal Muscular AtrophyCompletedNCT02913482Phase 2Results posted
• A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy (SMA) ParticipantsCompletedNCT02908685Phase 2Results posted
• A Study to Evaluate Long Term Safety, Tolerability, and Effectiveness of Olesoxime in Patients With Spinal Muscular Atrophy (SMA)CompletedNCT02628743Phase 2Results posted
• 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 TherapyActive, not recruitingNCT05861999Phase 4
• A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene TherapyActive, not recruitingNCT05861986Phase 4
• A Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular AtrophyCompletedNCT05808764Phase 2
• Long-term Follow-up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA)CompletedNCT05232929Phase 4
• A Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Participants With Spinal Muscular AtrophyCompletedNCT05115110Phase 2/3
• Bioavailability and Bioequivalence of Two Risdiplam Tablets in Healthy ParticipantsCompletedNCT04718181Phase 1
• An Expanded Access Program for Risdiplam in Participants With Spinal Muscular Atrophy (SMA)Approved for marketingNCT04256265
• A Study to Investigate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Risdiplam (RO7034067) Given by Mouth in Healthy VolunteersCompletedNCT02633709Phase 1
• A Study of RO6885247 in Adult and Pediatric Patients With Spinal Muscular Atrophy (MOONFISH)TerminatedNCT02240355Phase 1
• A Study to Collect Blood Samples From Patients With Spinal Muscular Atrophy for Biomarker AnalysisCompletedNCT01910168