MedTrace Logo

Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi

NCT04042025 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 85

Last updated 2025-04-25

No results posted yet for this study

Summary

This is a long-term follow-up safety and efficacy study of participants in clinical trials for spinal muscular atrophy (SMA) who were treated with onasemnogene abeparvovec-xioi. Participants will roll over from their respective previous (parent) study into this long-term study for continuous monitoring of safety as well as monitoring of continued efficacy and durability of response to onasemnogene abeparvovec-xioi treatment.

Conditions

  • Spinal Muscular Atrophy Type I
  • Spinal Muscular Atrophy Type II
  • Spinal Muscular Atrophy Type III
  • SMA

Interventions

BIOLOGICAL

Onasemnogene Abeparvovec-xioi

Onasemnogene abeparvovec-xioi is a non-replicating recombinant adeno-associated virus serotype 9 containing the human survival motor neuron gene under the control of the cytomegalovirus enhancer/chicken β-actin-hybrid promoter. Onasemnogene abeparvovec-xioi administered as a one-time intravenous (IV) infusion or intrathecal (IT) injection. Dosage determined by participant weight.

Sponsors & Collaborators

  • Novartis Gene Therapies

    lead INDUSTRY

Principal Investigators

  • Sitra Tauscher-Wisniewski, MD · Novartis Gene Therapies, Inc.

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-02-10
Primary Completion
2035-12-31
Completion
2035-12-31
FDA Drug
Yes

Countries

  • United States
  • Australia
  • Belgium
  • Canada
  • France
  • Italy
  • Japan
  • Taiwan
  • 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 NCT04042025 on ClinicalTrials.gov