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

NCT03306277 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 22

Last updated 2026-01-26

Study results available
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Summary

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

Conditions

  • SMA - Spinal Muscular Atrophy
  • Gene Therapy

Interventions

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.

Sponsors & Collaborators

  • Novartis Gene Therapies

    lead INDUSTRY

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Max Age
180 Days
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2017-10-24
Primary Completion
2019-11-12
Completion
2019-11-12
FDA Drug
Yes

Countries

  • United States

Study Locations

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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 NCT03306277 on ClinicalTrials.gov