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

Exploring Risdiplam's impact on young SMA patients post-gene therapy.

Recruiting
3-24 years
All
Phase 4

This study looks at how safe and effective a medicine called risdiplam is for young children with a disease called Spinal Muscular Atrophy (SMA). SMA is a genetic disorder that makes muscles weak. The study is for kids under 2 years old who have SMA and have already been treated with a special gene therapy called onasemnogene abeparvovec. This study is open-label, meaning everyone knows what treatment is given, and single-arm, meaning all participants get the same treatment.

Participants need to have 2 copies of a gene called SMN2, and must have received onasemnogene abeparvovec 3 to 7 months before joining the study. Children who need certain types of breathing support or have other health issues may not be able to join.

  • Length of Study: Participation requires regular follow-up visits.
  • Eligibility: Only for kids under 2 with specific genetic conditions.
  • Risks: Potential risks related to treatment and previous health conditions.
Study details
    Muscular Atrophy
    Spinal

NCT05861986

Hoffmann-La Roche

2 March 2025

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