At a glance
ClinicalIndex Comparison RecordPhase 2Completed· 174 enrolled
Drug / intervention
Risdiplamdrug
Likely dose
Not stated in record
Structured eligibility isn't available for this trial yet — see the full criteria in the Eligibility tab below.
Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.
An Open-Label Study to Investigate the Safety, Tolerability, and Pharmacokinetics/Pharmacodynamics of Risdiplam (RO7034067) in Adult and Pediatric Patients With Spinal Muscular Atrophy
In Brief
A Phase 2 clinical trial evaluating Risdiplam for Spinal Muscular Atrophy. Completed, enrolled 174 participants across 24 sites in 9 countries.
Detailed Summary
This is a multi-center, exploratory, non-comparative, and open-label study to investigate the safety, tolerability, PK, and PK/PD relationship of risdiplam in adults, children and infants with Spinal Muscular Atrophy (SMA) previously enrolled in Study BP29420 (Moonfish) with the splicing modifier RO6885247 or previously treated with nusinersen, olesoxime or AVXS-101.
Study Details
Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsSpinal Muscular Atrophy
CountriesBelgium, France, Germany, Italy, Netherlands, Poland, Switzerland, United Kingdom, United States
Collaborators--
Timeline
Phase 2CompletedFinished
2017201820192020202120222023202420252026
First PostedJan 2017
Enrollment StartMar 2017
Primary CompletionFeb 2025
TodayJul 2026
First PostedJan 26, 2017
Enrollment StartMar 3, 2017
Primary CompletionFeb 7, 2025
TodayJul 2, 2026
Enrollment to primary: 7.9 yearsPosted 9.4 years ago
Interventions
Risdiplamdrug
Risdiplam will be administered orally once daily.