NCT05073133

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

A Phase IV Open-label, Single-arm, Single-dose, Multicenter Study to Evaluate the saFEty, toLerability and effIcacy of Gene Replacement Therapy With intravenousOAV101(AVXS101) in Pediatric Patients From Latin America With Spinal Muscular Atrophy (SMA) - OFELIA

• Status: Completed
• Phase: Phase 4
• Study type: Interventional
• Enrollment: 16 (actual)
• Sponsor: Novartis Pharmaceuticals · Industry
• Sex: All
• Age: 24 Months
• Healthy volunteers: Not accepted

Summary

This was a phase IV Open-label, single-arm, single-dose, multicenter study, to evaluate the safety, tolerability and efficacy of intravenous administration of OAV101 (AVXS-101) in patients with SMA with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene ≤ 24 months and weighing ≤ 17 kg, over a 18-month period post infusion.

Detailed description

This is an open-label, single arm, multi-center study to evaluate the safety, tolerability, and efficacy of IV OAV101 in symptomatic SMA pediatric participants. The study enrolled participants ≤ 24 months old that weigh ≤ 17 kg. Participants who met eligibility criteria at Screening and Baseline visits received a single dose of IV OAV101 t the approved dose of 1.1e14 vg/kg and were followed for 18 months. The study included a 20-day screening period in which there were 2 Screening visits, during which, eligibility was assessed (Screening 1), weight was collected for dose calculation (Screening 2), and baseline assessments were performed prior to treatment. On Day -1, participants were admitted to the hospital for pre-treatment baseline procedures including prednisolone treatment per study protocol. On Day 1, participants received a single IV infusion of OAV101. Participants were discharged 12-48 hours after the infusion, based on Investigator judgment. Safety monitoring was performed on an ongoing basis per protocol requirement and was evaluated by the clinical safety team as well as DMC (Data monitoring committee).

Conditions

• Muscular Atrophy, Spinal

Interventions

Type	Name	Description
GENETIC	OAV101	A single Gene Therapy IV infusion at 1.1e14 vector genome (vg)/kg over approximately 60 minutes

Timeline

• Start date: 2021-11-04
• Primary completion: 2023-08-08
• Completion: 2023-08-08
• First posted: 2021-10-11
• Last updated: 2024-10-09
• Results posted: 2024-05-31

Locations

5 sites across 2 countries: Argentina, Brazil