NCT02908685

A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy (SMA) Participants

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

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 231 (actual)
• Sponsor: Hoffmann-La Roche · Industry
• Sex: All
• Age: 2 Years – 25 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Muscular Atrophy, Spinal

Interventions

Type	Name	Description
DRUG	Placebo	Placebo will be administered orally (via mouth) or through a feeding tube (naso-gastric or gastrostomy tube).
DRUG	Risdiplam	Risdiplam will be administered orally (via mouth) or through a feeding tube (naso-gastric or gastrostomy tube).

Timeline

• Start date: 2016-10-19
• Primary completion: 2019-09-06
• Completion: 2023-10-02
• First posted: 2016-09-21
• Last updated: 2024-04-24
• Results posted: 2021-06-15

Locations

45 sites across 16 countries: United States, Belgium, Brazil, Canada, China, Croatia, France, Germany, Italy, Japan, Poland, Russia, Serbia, Spain, Turkey (Türkiye), United Kingdom