At a glance
ClinicalIndex Comparison RecordPhase 2Completed· 62 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.
A Two Part Seamless, Open-label, Multicenter Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Infants With Type 1 Spinal Muscular Atrophy
In Brief
A Phase 2 clinical trial evaluating Risdiplam for Muscular Atrophy, Spinal. Completed, enrolled 62 participants across 32 sites in 16 countries.
Detailed Summary
Open-label, multi-center clinical study is to assess the safety, tolerability, pharmacokinetic (PK), pharmacodynamics (PD), and efficacy of Risdiplam (RO7034067) in infants with Type 1 spinal muscular atrophy (SMA). The study consists of two parts, an exploratory dose finding part (Part 1) and a confirmatory part (Part 2) which will investigate Risdiplam (RO7034067) for 24-months at the dose selected in Part 1.
Study Details
Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsMuscular Atrophy, Spinal
CountriesBelgium, Brazil, China, Croatia, France, Italy, Japan, Poland, Russia, Saudi Arabia, Serbia, Spain, Switzerland, Turkey (Türkiye), Ukraine, United States
Collaborators--
Timeline
Phase 2CompletedFinished
2017201820192020202120222023202420252026
First PostedSep 2016
Enrollment StartDec 2016
Primary CompletionNov 2019
Study CompletionDec 2023
TodayJul 2026
First PostedSep 23, 2016
Enrollment StartDec 23, 2016
Primary CompletionNov 14, 2019
Study CompletionDec 22, 2023
TodayJul 2, 2026
Enrollment to primary: 2.9 yearsPosted 9.8 years ago
Interventions
Risdiplamdrug
Risdiplam will be administered orally.