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

NCT03779334 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 26

Last updated 2026-04-08

Study results available
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Summary

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

Conditions

  • Muscular Atrophy, Spinal

Interventions

DRUG

Risdiplam

Risdiplam will be administered orally.

Sponsors & Collaborators

Principal Investigators

  • Clinical Trials · Hoffmann-La Roche

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
1 Day
Max Age
6 Weeks
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2019-08-07
Primary Completion
2023-02-20
Completion
2027-02-28
FDA Drug
Yes

Countries

  • United States
  • Australia
  • Belgium
  • Brazil
  • Poland
  • Russia
  • Taiwan

Study Locations

More Related Trials

Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03779334 on ClinicalTrials.gov