Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi
Phase 3 – large-scale trial before approval
Conditions:
Spinal Muscular Atrophy Type I
Spinal Muscular Atrophy Type II
Spinal Muscular Atrophy Type III
SMA
Sponsor: Novartis Gene Therapies
trial.available_in:
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Overview
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.
Who can participate
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
Locations
9
Australia (1)
Sydney Children's Hospital
Randwick , New South Wales
Belgium (1)
Universitair Ziekenhuis Gent
Ghent
Canada (1)
Children's Hospital of Eastern Ontario Research Institute
Ottawa , Ontario
France (1)
Hôpital Armand Trousseau
Paris
Italy (1)
Instituto Gianninia Gaslini
Genova
Japan (1)
Tokyo Women's Medical University Hospital
Tokyo
Taiwan (1)
National Taiwan University Hospital
Taipei
United Kingdom (1)
Great Ormond Street Hospital for Children NHS Foundation Trust