NCT03779334

A Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy

An Open-Label Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy

• Status: Active Not Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 26 (actual)
• Sponsor: Hoffmann-La Roche · Industry
• Sex: All
• Age: 1 Day – 6 Weeks
• Healthy volunteers: Not accepted

Summary

A global study of oral risdiplam in pre-symptomatic participants with spinal muscular atrophy (SMA).

Detailed description

The study is an open-label, single-arm, multicenter clinical study to investigate the efficacy, safety, pharmacokinetics, and pharmacodynamics of risdiplam in infants aged from birth to 6 weeks who have been genetically diagnosed with SMA but are not yet presenting with symptoms. There will be a screening, treatment, open-label extension (OLE) and a follow-up. All participants will receive risdiplam orally once daily for 2 years followed by an OLE phase of at least 3 years and a follow-up (if applicable), for a total treatment duration of at least 5 years for each participant enrolled.

Conditions

• Muscular Atrophy, Spinal

Interventions

Type	Name	Description
DRUG	Risdiplam	Risdiplam will be administered orally.

Timeline

• Start date: 2019-08-07
• Primary completion: 2023-02-20
• Completion: 2027-02-28
• First posted: 2018-12-19
• Last updated: 2026-04-08
• Results posted: 2024-03-05

Locations

7 sites across 7 countries: United States, Australia, Belgium, Brazil, Poland, Russia, Taiwan

Regulatory

• FDA-regulated drug study