Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi
Фаза 3 – широко изпитване преди одобрение
Заболявания:
Spinal Muscular Atrophy Type I
Spinal Muscular Atrophy Type II
Spinal Muscular Atrophy Type III
SMA
Спонсор: Novartis Gene Therapies
Налично на:
БГ
Обобщение
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.
Кой може да участва
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
Места на провеждане
9
Австралия (1)
Sydney Children's Hospital
Randwick , New South Wales
Белгия (1)
Universitair Ziekenhuis Gent
Ghent
Канада (1)
Children's Hospital of Eastern Ontario Research Institute
Ottawa , Ontario
Франция (1)
Hôpital Armand Trousseau
Paris
Италия (1)
Instituto Gianninia Gaslini
Genova
Япония (1)
Tokyo Women's Medical University Hospital
Tokyo
Taiwan (1)
National Taiwan University Hospital
Taipei
Великобритания (1)
Great Ormond Street Hospital for Children NHS Foundation Trust