Ignite Creation Date:
2025-12-25 @ 2:34 AM
Ignite Modification Date:
2026-02-20 @ 6:05 PM
Study NCT ID:
NCT03779334
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2025-11-26
First Post:
2018-12-17
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
A Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy
Sponsor:
Hoffmann-La Roche
Organization:
Study Overview
Official Title:
An Open-Label Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy
Status:
ACTIVE_NOT_RECRUITING
Status Verified Date:
2025-11
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
Rainbowfish
Brief 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.
Study Oversight
Has Oversight DMC:
True
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?: