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

> **NCT05861986** · PHASE4 · RECRUITING · sponsor: **Hoffmann-La Roche** · enrollment: 28 (estimated)

## Conditions studied

- Muscular Atrophy, Spinal

## Interventions

- **DRUG:** Risdiplam

## Key facts

- **NCT ID:** NCT05861986
- **Lead sponsor:** Hoffmann-La Roche
- **Sponsor class:** INDUSTRY
- **Phase:** PHASE4
- **Study type:** INTERVENTIONAL
- **Status:** RECRUITING
- **Start date:** 2024-05-30
- **Primary completion:** 2028-03-31
- **Final completion:** 2029-03-31
- **Target enrollment:** 28 (ESTIMATED)
- **Last updated:** 2026-05-04


## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT05861986

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration 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". Retrieved via AI Analytics 2026-07-20 from https://api.ai-analytics.org/clinical/NCT05861986. Licensed CC0.

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