Gene Replacement Therapy Clinical Trial for Patients With Spinal Muscular Atrophy Type 1 (STR1VE)

Brief Summary:
Phase 3 pivotal US trial studying open-label intravenous administration of onasemnogene abeparvovec-xioi in SMA Type 1 patients

Detailed Description:
Phase 3, open-label, single-arm, single-dose, study of onasemnogene abeparvovec-xioi (gene replacement therapy) in patients 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) patients < 6 months (< 180 days) of age at the time of gene replacement therapy (Day 1) will be enrolled.


Study Type: Interventional  (Clinical Trial)
Estimated Enrollment: 20 participants
Intervention Model: Single Group Assignment
Intervention Model Description: one-time intravenous administration of onasemnogene abeparvovec-xioi
Masking: None (Open Label)
Primary Purpose: Treatment
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
Actual Study Start Date: October 24, 2017
Estimated Primary Completion Date: November 12, 2019
Estimated Study Completion Date: November 12, 2019

Arm:
- Experimental: Onasemnogene Abeparvovec-xioi