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

NCT03032172 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 174

Last updated 2025-10-01

Study results available
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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

Interventions

DRUG

Risdiplam

Risdiplam will be administered orally once daily.

Sponsors & Collaborators

Principal Investigators

  • Clinical Trials · Hoffmann-La Roche

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
6 Months
Max Age
60 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2017-03-03
Primary Completion
2025-02-07
Completion
2025-02-07
FDA Drug
Yes

Countries

  • United States
  • Belgium
  • France
  • Germany
  • Italy
  • Netherlands
  • Poland
  • Switzerland
  • United Kingdom

Study Locations

More Related Trials

Entities

Diseases
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 NCT03032172 on ClinicalTrials.gov