MedTrace Logo

A Study of Risdiplam in Participants With Type I and Type II Spinal Muscle Atrophy (SMA)

NCT07531719 · Status: NOT_YET_RECRUITING · Type: OBSERVATIONAL · Enrollment: 30

Last updated 2026-05-15

No results posted yet for this study

Summary

This multicenter, non-interventional, prospective study with a retrospective component aims to evaluate the real-world clinical outcomes, safety profile, and motor function improvements of Risdiplam treatment in participants with Type I and Type II Spinal Muscle Atrophy over 24 months of treatment.

The study will allow retrospective collection of medical history data and previous participants visits from the medical records.

Conditions

  • Spinal Muscle Atrophy

Sponsors & Collaborators

Principal Investigators

  • Clinical Trials · Hoffmann-La Roche

Eligibility

Max Age
5 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2026-06-01
Primary Completion
2028-03-01
Completion
2029-03-01

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 NCT07531719 on ClinicalTrials.gov