Active — Not RecruitingNot Applicable

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

United States13 participantsStarted 2017-09-21

Plain-language 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.

Who can participate

SexALL

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Inclusion criteria

✓. Patient who received onasemnogene abeparvovec-xioi in the AVXS-101-CL-101 gene replacement therapy clinical trial for SMA Type 1.
✓. Parent/legal guardian willing and able to complete the informed consent process, comply with study procedures and visit schedule.

What they're measuring

Long-Term Safety

Timeframe: 15 years

Trial details

NCT IDNCT03421977

SponsorNovartis Gene Therapies

Sponsor typeINDUSTRY

Study typeOBSERVATIONAL

Primary completion2030-12-02

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