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

Muscular Atrophy, Spinal Clinical Trial

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

Clinical Trial Summary

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

Clinical Trial 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. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT03779334
Study type Interventional
Source Hoffmann-La Roche
Contact
Status Active, not recruiting
Phase Phase 2
Start date August 7, 2019
Completion date March 31, 2027
View Details
See also
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