NCT05073133View on ClinicalTrials.gov

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

PHASE4CompletedINTERVENTIONAL
Enrollment

16

Participants

Timeline

Start Date

November 4, 2021

Primary Completion Date

August 8, 2023

Study Completion Date

August 8, 2023

Conditions
Muscular Atrophy, Spinal
Interventions
GENETIC

OAV101

A single Gene Therapy IV infusion at 1.1e14 vector genome (vg)/kg over approximately 60 minutes

Trial Locations (5)

C1181ACH

Novartis Investigative Site, CABA

C1245AAM

Novartis Investigative Site, Buenos Aires

30130-000

Novartis Investigative Site, Belo Horizonte

90035-903

Novartis Investigative Site, Porto Alegre

05403-000

Novartis Investigative Site, São Paulo

Sponsors
All Listed Sponsors
lead

Novartis Pharmaceuticals

INDUSTRY