At a glance
ClinicalIndex Comparison RecordPhase 2Active· 26 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 of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy
In Brief
A Phase 2 clinical trial evaluating Risdiplam for Muscular Atrophy, Spinal. Active but no longer recruiting, targeting 26 participants across 7 sites in 7 countries.
Detailed Summary
A global study of oral risdiplam in pre-symptomatic participants with spinal muscular atrophy (SMA).
Study Details
Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsMuscular Atrophy, Spinal
CountriesAustralia, Belgium, Brazil, Poland, Russia, Taiwan, United States
Collaborators--
Timeline
Phase 2Active
201920202021202220232024202520262027
First PostedDec 2018
Enrollment StartAug 2019
Primary CompletionFeb 2023
TodayJul 2026
Study CompletionFeb 2027
First PostedDec 19, 2018
Enrollment StartAug 7, 2019
Primary CompletionFeb 20, 2023
Study CompletionFeb 28, 2027
TodayJul 2, 2026
Enrollment to primary: 3.5 yearsPosted 7.5 years ago
Interventions
Risdiplamdrug
Risdiplam will be administered orally.