NCT04851873 - Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA) | Biotech Hunter

Enrollment

Participants

Timeline

Start Date

September 8, 2021

Primary Completion Date

June 13, 2023

Study Completion Date

June 13, 2023

Conditions

Spinal Muscular Atrophy

Interventions

GENETIC

OAV101

Gene Therapy - 1.1e14 vector genome (vg)/kg as a one-time IV infusion was administered over approximately 60 minutes.

Trial Locations (13)

Novartis Investigative Site, Randwick

Novartis Investigative Site, Leuven

Novartis Investigative Site, Taipei

Novartis Investigative Site, St Louis

Novartis Investigative Site, Strasbourg

Novartis Investigative Site, Kaohsiung City

Novartis Investigative Site, Garches

Novartis Investigative Site, Lisbon

Novartis Investigative Site, Boston

Novartis Investigative Site, Montreal

Novartis Investigative Site, Roma

Novartis Investigative Site, London

Novartis Investigative Site, Newcastle upon Tyne