NCT03306277

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

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
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 22 (actual)
• Sponsor: Novartis Gene Therapies · Industry
• Sex: All
• Age: 180 Days
• Healthy volunteers: Not accepted

Summary

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

Detailed 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.

Conditions

• SMA - Spinal Muscular Atrophy
• Gene Therapy

Interventions

Type	Name	Description
BIOLOGICAL	Onasemnogene Abeparvovec-xioi	Non-replicating recombinant adeno-associated virus serotype 9 (AAV9) containing the complimentary deoxyribonucleic acid (cDNA) of the human SMN gene under the control of the cytomegalovirus (CMV) enhancer/chicken-β-actin-hybrid promoter (CB). The AAV inverted terminal repeat (ITR) has been modified to promote intramolecular annealing of the transgene, thus forming a double-stranded transgene ready for transcription.

Timeline

• Start date: 2017-10-24
• Primary completion: 2019-11-12
• Completion: 2019-11-12
• First posted: 2017-10-11
• Last updated: 2026-01-26
• Results posted: 2020-07-16

Locations

16 sites across 1 country: United States

Regulatory

• FDA-regulated drug study