NCT03032172

A Study of Risdiplam (RO7034067) in Adult and Pediatric Participants With Spinal Muscular Atrophy

An Open-Label Study to Investigate the Safety, Tolerability, and Pharmacokinetics/Pharmacodynamics of Risdiplam (RO7034067) in Adult and Pediatric Patients With Spinal Muscular Atrophy

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 174 (actual)
• Sponsor: Hoffmann-La Roche · Industry
• Sex: All
• Age: 6 Months – 60 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Spinal Muscular Atrophy

Interventions

Type	Name	Description
DRUG	Risdiplam	Risdiplam will be administered orally once daily.

Timeline

• Start date: 2017-03-03
• Primary completion: 2025-02-07
• Completion: 2025-02-07
• First posted: 2017-01-26
• Last updated: 2025-10-01
• Results posted: 2025-10-01

Locations

24 sites across 9 countries: United States, Belgium, France, Germany, Italy, Netherlands, Poland, Switzerland, United Kingdom

Regulatory

• FDA-regulated drug study