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A Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Muscular Atrophy Who Experienced a Plateau or Decline in Function After Gene Therapy

NCT05861999 · Status: RECRUITING · Phase: PHASE4 · Type: INTERVENTIONAL · Enrollment: 28

Last updated 2026-05-08

No results posted yet for this study

Summary

This is an open-label, single-arm, multicenter clinical study to evaluate the effectiveness and safety of risdiplam administered in pediatric participants with SMA and 2 SMN2 copies who previously received onasemnogene abeparvovec and experience a plateau or decline in function. Participants to be enrolled are children \<2 years of age genetically diagnosed with SMA.

Conditions

  • Muscular Atrophy, Spinal

Interventions

DRUG

Risdiplam

Participants will receive risdiplam orally at the currently approved dose. The dose should be adapted for weight and age.

Sponsors & Collaborators

Principal Investigators

  • Clinical Trials · Hoffmann-La Roche

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
3 Months
Max Age
24 Months
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-08-14
Primary Completion
2028-03-31
Completion
2029-03-31
FDA Drug
Yes

Countries

  • United States
  • Germany
  • Israel
  • Poland
  • Qatar
  • United Kingdom

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 NCT05861999 on ClinicalTrials.gov