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

About the study

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 take part

You may be eligible to participate in the study if you meet the following criteria:

INCLUSION CRITERIA

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.

EXCLUSION CRITERIA

Exclusion Criteria:

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

Study Locations

Enter your ZIP code/Postal code/PIN code to locate study sites near you:

How to Apply

Contact the study center to learn if this study is a good match for you.

Study’s details

Contition

Spinal Muscular Atrophy 1

Participants needed

Est. Completion Date

Dec 31, 2033

Treatment type

Observational

Sponsor

Novartis

ClinicalTrials.gov identifier

NCT03421977

Study number

AVXS 101-LT-001

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