CI

At a glance

ClinicalIndex Comparison Record
Phase 3Completed· 30 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/NCT03505099
NCT03505099Phase 3Completed
CompareCT.gov

A Global Study of a Single, One-Time Dose of AVXS-101 Delivered to Infants With Genetically Diagnosed and Pre-symptomatic Spinal Muscular Atrophy With Multiple Copies of SMN2

Novartis Gene Therapies·interventional·Posted Apr 23, 2018·Updated Jan 26, 2026

In Brief

A Phase 3 clinical trial evaluating onasemnogene abeparvovec-xioi for Spinal Muscular Atrophy. Completed, enrolled 30 participants across 16 sites in 6 countries.

Detailed Summary

To evaluate the safety and efficacy of intravenous onasemnogene abeparvovec-xioi in pre-symptomatic patients with SMA and 2 or 3 copies SMN2

Study Details

Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsSpinal Muscular Atrophy
CountriesAustralia, Belgium, Canada, Japan, United Kingdom, United States
CollaboratorsPRA Health Sciences

Timeline

Phase 3CompletedFinished
20192020202120222023202420252026
First PostedApr 23, 2018
Enrollment StartApr 2, 2018
Primary CompletionJun 15, 2021
TodayJul 2, 2026
Enrollment to primary: 3.2 yearsPosted 8.2 years ago

Interventions

onasemnogene abeparvovec-xioibiological

A non-replicating recombinant 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.