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

NCT03461289 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 33

Last updated 2026-01-26

Study results available
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Summary

Phase 3, open-label, single-arm, single-dose, trial 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 a biallelic pathogenic mutation of the survival motor neuron 1 gene (SMN1) with one or two copies of survival motor neuron 2 gene (SMN2). Up to 30 patients \< 6 months (\< 180 days) of age at the time of gene replacement therapy (Day 1) will be enrolled.

Conditions

Interventions

BIOLOGICAL

Onasemnogene Abeparvovec-xioi

Onasemnogene abeparvovec-xioi is a non-replicating recombinant adeno-associated virus serotype 9 (AAV9) containing the human survival motor neuron (SMN) gene under the control of the cytomegalovirus (CMV) enhancer/chicken β-actin-hybrid promoter (CB).

Sponsors & Collaborators

  • Novartis Gene Therapies

    lead INDUSTRY

Principal Investigators

  • AveXis Medinfo · Sponsor GmbH

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Max Age
6 Months
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2018-08-16
Primary Completion
2020-09-11
Completion
2020-09-11
FDA Drug
Yes

Countries

  • Belgium
  • France
  • Italy
  • United Kingdom

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03461289 on ClinicalTrials.gov