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

Plain-language summary

This is a long-term safety follow-up study for patients who received onasemnogene abeparvovec-xioi (Zolgensma) in the AVXS-101-CL-101 gene replacement therapy trial for SMA Type 1. Patients roll over from the parent study into this long-term study for continuous safety monitoring for up to 15 years. Eligibility requires having received onasemnogene abeparvovec-xioi in AVXS-101-CL-101 and a parent or legal guardian willing and able to complete the informed consent process and comply with study procedures and the visit schedule.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

N/A

Status

Active, not recruiting

Sponsor

Novartis Gene Therapies

Intervention

Onasemnogene Abeparvovec-xioi (Zolgensma)

Start date

9/21/2017

Estimated completion

12/2/2030

Countries

United States

Related genes

Locations

• Columbus, United States

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

Inclusion Criteria:

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

Exclusion Criteria:

1\. Parent/legal guardian unable or unwilling to participate in the long term follow up safety study.