RO7034067
Sponsors
F. Hoffmann-La Roche AG
Conditions
Spinal Muscular Atrophy (SMA)
Phase 2
An Open-Label Study of Risdiplam in Infants with Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy
Active, not recruitingCTIS2023-506009-20-00
Start: 2019-03-29Target: 6Updated: 2025-07-07
An Open-Label Study to Investigate the Safety, Tolerability, and Pharmacokinetics/Pharmacodynamics of Risdiplam (RO7034067) in Adult and Pediatric patients with Spinal Muscular Atrophy
CompletedCTIS2023-506739-14-00
Start: 2017-05-12End: 2025-02-07Target: 110Updated: 2024-12-17
A Phase II, Open-label Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants with Spinal Muscular Atrophy
RecruitingCTIS2023-505602-42-00
Start: 2024-04-30Target: 8Updated: 2026-01-05
Phase 4
A Phase IV Open-Label Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Patients with Spinal Muscular Atrophy After Gene Therapy
RecruitingCTIS2023-504508-26-00
Start: 2024-11-14Target: 15Updated: 2025-10-17
A Phase IV Open-Label 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
RecruitingCTIS2023-505161-81-00
Start: 2024-12-16Target: 15Updated: 2025-10-10