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Recruiting NCT05861986

A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy

Conditions: Muscular Atrophy, Spinal

Sex: All

Ages: 3 Months – 24 Months

Healthy volunteers: No

Phase: PHASE4

Enrollment: 28

Sponsor: Hoffmann-La Roche

Location: University of Arkansas for Medical Sciences Little Rock Arkansas

Summary

This is an open-label, single-arm, multicenter clinical study to evaluate the effectiveness and safety of risdiplam administered as an early intervention in pediatric participants with spinal muscular atrophy (SMA) and 2 SMN2 copies who have previously received onasemnogene abeparvovec. Participants are children \< 2 years of age genetically diagnosed with SMA.

Eligibility Criteria

Inclusion Criteria: * \

Interested in this study? View the official listing for contact and enrollment details.

View on ClinicalTrials.gov

Source: ClinicalTrials.gov (NCT05861986). StuddyBuddy aggregates publicly available trial information.