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

Study Identifier:
AVXS-101-LT-001
ClinicalTrials.gov Identifier:
NCT03421977
EudraCT Identifier:
N/A
EU CT Number:
N/A
Sponsor:
Novartis Gene Therapies
Active, not recruiting

Study Details

Medical Condition
  • Spinal Muscular Atrophy Type I
Study Drug
  • Biological: Onasemnogene Abeparvovec-xioi
Date
Sep 2017 - Dec 2030
Patient Requirements
Sex: Female & Male
Age: N/A
Requirements Information
Inclusion and Exclusion 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
  • 1\. Parent/legal guardian unable or unwilling to participate in the long term follow up safety study.

Protocol 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.

Trial Locations

Location
Status
Location
Nationwide Children's Hospital
Columbus, Ohio, United States, 43205
Status
N/A

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