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An Expanded Access Program for Risdiplam in Participants With Spinal Muscular Atrophy (SMA)

NCT04256265 · Status: APPROVED_FOR_MARKETING · Type: EXPANDED_ACCESS

Last updated 2020-10-05

No results posted yet for this study

Summary

This expanded access program (EAP) will provide access to risdiplam for eligible participants with Type 1 or Type 2 spinal muscular atrophy (SMA) before it is commercially available in the United States for the indication of SMA.

Conditions

  • Muscular Atrophy, Spinal

Interventions

DRUG

Risdiplam

Risdiplam will be administered orally once daily

Sponsors & Collaborators

  • Genentech, Inc.

    lead INDUSTRY

Principal Investigators

  • Clinical Trials · Hoffmann-La Roche

Eligibility

Min Age
2 Months
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Countries

  • United States

Study Locations

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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 NCT04256265 on ClinicalTrials.gov