At a glance
ClinicalIndex Comparison Record- ✓Confirmed diagnosis of 5q-autosomal recessive SMA
- ✓Part 1: Type 2 or 3 SMA (ambulant or non-ambulant)
- ✓Part 2: Type 2 or 3 SMA non-ambulant status
- ✓Part 2: RULM entry item A score ≥2
- ✕Prior or concomitant SMN2-targeting antisense oligonucleotide, splicing modifier, or gene therapy
- ✕Any history of cell therapy
- ✕Hospitalization for pulmonary event within last 2 months or planned at screening
- ✕Scoliosis or hip fixation surgery in past year or planned within 18 months
Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.
A Two Part Seamless, Multi-Center Randomized, Placebo-Controlled, Double-Blind Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy Patients
In Brief
A Phase 2 clinical trial evaluating Placebo and Risdiplam for Muscular Atrophy, Spinal. Completed, enrolled 231 participants across 45 sites in 16 countries.
Detailed Summary
Multi-center, randomized, double-blind, placebo-controlled study to assess the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of Risdiplam in adult and pediatric participants with Type 2 and Type 3 SMA. The study consists of two parts, an exploratory dose finding part (Part 1) of Risdiplam for 12 weeks and a confirmatory part (Part 2) of Risdiplam for 24 months.
Study Details
Timeline
Interventions
Placebo will be administered orally (via mouth) or through a feeding tube (naso-gastric or gastrostomy tube).
Risdiplam will be administered orally (via mouth) or through a feeding tube (naso-gastric or gastrostomy tube).