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Long-Term Follow-up Study for Patients From AVXS-101-CL-101

NCT03421977 · Status: ACTIVE_NOT_RECRUITING · Type: OBSERVATIONAL · Enrollment: 13

Last updated 2025-04-25

No results posted yet for this study

Summary

This is a long term, safety follow up study of patients in the AVXS-101-CL-101 gene replacement therapy clinical trial for SMA Type 1 delivering onasemnogene abeparvovec-xioi. Patients will roll over from the parent study into this long-term study for continuous safety monitoring for up to 15 years.

Conditions

  • Spinal Muscular Atrophy 1

Interventions

BIOLOGICAL

Onasemnogene Abeparvovec-xioi

Patients received treatment with onasemnogene abeparvovec-xioi in the parent study, AVXS-101-CL-101

Sponsors & Collaborators

  • Novartis Gene Therapies

    lead INDUSTRY

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2017-09-21
Primary Completion
2030-12-02
Completion
2030-12-02
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 NCT03421977 on ClinicalTrials.gov