OAV101

GENETIC4 trials

Sponsors

Novartis Pharmaceuticals

Conditions

Muscular Atrophy, SpinalSpinal Muscular AtrophyType 2 Spinal Muscular Atrophy

Phase 3

Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA)

CompletedNCT04851873

Novartis PharmaceuticalsSpinal Muscular Atrophy

Start: 2021-09-08End: 2023-06-13Updated: 2024-10-09

Efficacy and Safety of Intrathecal OAV101 (AVXS-101) in Pediatric Patients With Type 2 Spinal Muscular Atrophy (SMA)

CompletedNCT05089656

Novartis PharmaceuticalsType 2 Spinal Muscular Atrophy

Start: 2022-02-01End: 2025-04-29Updated: 2026-01-13

Phase IIIb, Open-label, Multi-center Study to Evaluate Safety, Tolerability and Efficacy of OAV101 Administered Intrathecally to Participants With SMA Who Discontinued Treatment With Nusinersen or Risdiplam

CompletedNCT05386680

Novartis PharmaceuticalsSpinal Muscular Atrophy

Start: 2023-01-12End: 2024-11-29Updated: 2026-01-13

Phase 4

Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA) (OFELIA)

CompletedNCT05073133

Novartis PharmaceuticalsMuscular Atrophy, Spinal

Start: 2021-11-04End: 2023-08-08Updated: 2024-10-09

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