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

NCT05073133 · Status: COMPLETED · Phase: PHASE4 · Type: INTERVENTIONAL · Enrollment: 16

Last updated 2024-10-09

Study results available
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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.

Conditions

  • Muscular Atrophy, Spinal

Interventions

GENETIC

OAV101

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

Sponsors & Collaborators

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Max Age
24 Months
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-11-04
Primary Completion
2023-08-08
Completion
2023-08-08

Countries

  • Argentina
  • Brazil

Study Locations

More Related Trials

Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05073133 on ClinicalTrials.gov