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Pre-Symptomatic Study of Intravenous Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy (SMA) for Patients With Multiple Copies of SMN2

NCT03505099 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 30

Last updated 2026-01-26

Study results available
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Summary

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

Conditions

Interventions

BIOLOGICAL

onasemnogene abeparvovec-xioi

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.

Sponsors & Collaborators

  • PRA Health Sciences

    collaborator INDUSTRY

  • Novartis Gene Therapies

    lead INDUSTRY

Principal Investigators

  • Novartis Pharmaceuticals · Novartis Pharmaceuticals

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Max Age
42 Days
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2018-04-02
Primary Completion
2021-06-15
Completion
2021-06-15
FDA Drug
Yes

Countries

  • United States
  • Australia
  • Belgium
  • Canada
  • Japan
  • 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 NCT03505099 on ClinicalTrials.gov