Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical Trials
Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 IT or OAV101 IV in Clinical Trials
Novartis Pharmaceuticals
175 participants
Dec 19, 2022
INTERVENTIONAL
Conditions
Summary
This is a global, prospective, multi-center study that is designed to assess the long-term safety and efficacy of OAV101 in patients who participated in an OAV101 clinical trial. The assessments of safety and efficacy in Study COAV101A12308 will continue for 5 years after enrollment in this study.
Eligibility
Inclusion Criteria3
- Participated in an OAV101 clinical trial.
- Written informed consent must be obtained before any assessment is performed.
- Patient/Parent/legal guardian willing and able to comply with study procedures.
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Interventions
Onasemnogene abeparvovec is a non-replicating recombinant adeno-associated virus serotype 9 containing the human survival motor neuron gene under the control of the ytomegalovirus enhancer/chicken β-actin-hybrid promoter. Onasemnogene abeparvovec is administered as a one-time intravenous (IV) infusion or intrathecal (IT) injection. Dosage determined by participant weight.
Locations(32)
View Full Details on ClinicalTrials.gov
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NCT05335876