Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi

Study Identifier:
AVXS-101-LT-002
ClinicalTrials.gov Identifier:
NCT04042025
EudraCT Identifier:
2019-002611-26
EU CT Number:
N/A
Sponsor:
Novartis Gene Therapies
Active, not recruiting

Study Details

Medical Condition
  • Spinal Muscular Atrophy Type I
  • Spinal Muscular Atrophy Type II
  • Spinal Muscular Atrophy Type III
  • All Spinal Muscular Atrophies
Study Drug
  • Biological: Onasemnogene Abeparvovec-xioi
Date
Feb 2020 - Dec 2035
Phase 1
Phase 2
Phase 3
Phase 4
Patient Requirements
Sex: Female & Male
Age: N/A
Requirements Information
Inclusion and Exclusion Criteria
Inclusion Criteria
  • Any participant with SMA who received onasemnogene abeparvovec-xioi gene replacement therapy in a Novartis Gene Therapies-sponsored clinical study
  • Participant/parent/legal guardian willing and able to complete the informed consent process and comply with study procedures and visit schedule
Exclusion Criteria
  • Parent/legal guardian unable or unwilling to participate in the long-term follow-up safety study

Protocol Summary

This is a long-term follow-up safety and efficacy study of participants in clinical trials for spinal muscular atrophy (SMA) who were treated with onasemnogene abeparvovec-xioi. Participants will roll over from their respective previous (parent) study into this long-term study for continuous monitoring of safety as well as monitoring of continued efficacy and durability of response to onasemnogene abeparvovec-xioi treatment.

Trial Locations

Location
Status
Location
Stanford University Medical Center
Palo Alto, California, United States, 94304
Status
N/A
Location
Children's Hospital Colorado
Aurora, Colorado, United States, 80045
Status
N/A
Location
Ann Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611
Status
N/A
Location
John Hopkins Hospital - David M. Rubenstein Child Health Building
Baltimore, Maryland, United States, 21287
Status
N/A
Location
Massachusetts General Hospital
Boston, Massachusetts, United States, 02114
Status
N/A
Location
Boston Children's Hospital
Boston, Massachusetts, United States, 02115
Status
N/A
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