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

Completed · Phase 3

Conditions studied: Spinal Muscular Atrophy

In brief

To evaluate the safety, tolerability and efficacy of intravenous administration of OAV101 (AVXS-101) in patients with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene weighing ≥ 8.5 kg and ≤ 21 kg, over a 12 month period.

Key facts

Study ID

NCT04851873

Run by

Novartis Pharmaceuticals

People needed

24

Starts

2021-09-08

Expected to finish

2023-06-13

Last updated by the study team

2024-10-09

Who can join

Age: any, up to 17. Sex: any. Healthy volunteers: not accepted.

Where it is running

• Novartis Investigative Site — Boston, Massachusetts, United States
• Novartis Investigative Site — St Louis, Missouri, United States
• Novartis Investigative Site — Randwick, New South Wales, Australia
• Novartis Investigative Site — Leuven, Belgium
• Novartis Investigative Site — Montreal, Quebec, Canada
• Novartis Investigative Site — Garches, France
• Novartis Investigative Site — Strasbourg, France
• Novartis Investigative Site — Roma, RM, Italy
• Novartis Investigative Site — Lisbon, Portugal
• Novartis Investigative Site — Kaohsiung City, Taiwan
• Novartis Investigative Site — Taipei, Taiwan
• Novartis Investigative Site — London, United Kingdom
• Novartis Investigative Site — Newcastle upon Tyne, United Kingdom

Full record on ClinicalTrials.gov

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