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NCT04851873

A Phase IIIb, Open-label, Single-arm, Single-dose, Multicenter Study to Evaluate the Safety, Tolerability and Efficacy of Gene Replacement Therapy With Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA)

Study Summary

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.

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Interventions

OAV101GENETIC
Gene Therapy - 1.1e14 vector genome (vg)/kg as a one-time IV infusion was administered over approximately 60 minutes.

Study Locations

FacilityCityStateCountry
Novartis Investigative SiteBostonMassachusettsUnited States
Novartis Investigative SiteSt LouisMissouriUnited States
Novartis Investigative SiteRandwickNew South WalesAustralia
Novartis Investigative SiteLeuvenBelgium
Novartis Investigative SiteMontrealQuebecCanada
Novartis Investigative SiteGarchesFrance
Novartis Investigative SiteStrasbourgFrance
Novartis Investigative SiteRomaRMItaly
Novartis Investigative SiteLisbonPortugal
Novartis Investigative SiteKaohsiung CityTaiwan
Novartis Investigative SiteTaipeiTaiwan
Novartis Investigative SiteLondonUnited Kingdom
Novartis Investigative SiteNewcastle upon TyneUnited Kingdom

Official Trial Information

View on ClinicalTrials.gov

Data sourced from ClinicalTrials.gov. Last updated: April 14, 2026