Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1 — STR1VE  

Gene Therapy Clinical Trial

Official title: Phase 3, Open-Label, Single-Arm, Single-Dose Gene Replacement Therapy Clinical Trial for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies Delivering AVXS-101 by Intravenous Infusion

Status Completed

Clinical Trial Summary

Phase 3 pivotal US trial studying open-label intravenous administration of onasemnogene abeparvovec-xioi in spinal muscular atrophy (SMA) Type 1 participants.

Clinical Trial Description

Phase 3, open-label, single-arm, single-dose, study of onasemnogene abeparvovec-xioi (gene replacement therapy) in participants with spinal muscular atrophy (SMA) Type 1 who meet enrollment criteria and are genetically defined by nonfunctional survival motor neuron 1 gene (SMN1) with 1 or 2 copies of survival motor neuron 2 gene (SMN2). Fifteen (15) participants < 6 months (< 180 days) of age at the time of gene replacement therapy (Day 1) will be enrolled. ;

Related Conditions & MeSH terms

• Atrophy
• Gene Therapy
• Muscular Atrophy
• Muscular Atrophy, Spinal
• SMA - Spinal Muscular Atrophy

• NCT number: NCT03306277
• Study type: Interventional
• Source: Novartis
• Status: Completed
• Phase: Phase 3
• Start date: October 24, 2017
• Completion date: November 12, 2019