CI

At a glance

ClinicalIndex Comparison Record
Phase 3Completed· 22 enrolled
Drug / intervention
Onasemnogene Abeparvovec-xioibiological
Likely dose
Not stated in record
Structured eligibility isn't available for this trial yet — see the full criteria in the Eligibility tab below.

Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.

Search/NCT03306277
NCT03306277Phase 3Completed
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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

Novartis Gene Therapies·interventional·Posted Oct 11, 2017·Updated Jan 26, 2026

In Brief

A Phase 3 clinical trial evaluating Onasemnogene Abeparvovec-xioi for SMA - Spinal Muscular Atrophy and Gene Therapy. Completed, enrolled 22 participants across 16 sites.

Detailed Summary

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

Study Details

Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsSMA - Spinal Muscular Atrophy, Gene Therapy
CountriesUnited States
Collaborators--

Timeline

Phase 3CompletedFinished
201820192020202120222023202420252026
First PostedOct 11, 2017
Enrollment StartOct 24, 2017
Primary CompletionNov 12, 2019
TodayJul 2, 2026
Enrollment to primary: 2.1 yearsPosted 8.7 years ago

Interventions

Onasemnogene Abeparvovec-xioibiological

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.