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Active (not recruiting) Phase 3 NCT04042025

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: БГ
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
London
United States (1)
Stanford University Medical Center
Palo Alto , California
Technical details
Status
Active (not recruiting)
Phase
Phase 3
Study type
INTERVENTIONAL
Sex
Male and female
Healthy volunteers
No
Start date
10.02.2020
Completion date
31.12.2035
Registry ID
NCT04042025
Source
anzctr
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Information is automatically extracted from ClinicalTrials.gov. Consult your doctor before taking action.