Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 IT or OAV101 IV in Clinical Trials
Novartis Pharmaceuticals
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.
Description
The study is comprised of a Baseline Visit and 2 Follow-up Periods. For Follow-up Periods 1 and 2, which includes Baseline through Year 5 visits, assessments will be performed at the Investigational site. For the first 2 years (Follow-up Period 1), visits will occur every 6 months. For Years 3 to 5 (Follow-up Period 2) follow-up visits will be conducted annually. All patients will enter the study at the baseline visit and continue for 5 years.
Eligibility
- Age range
- 0–100 years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: 1. Participated in an OAV101 clinical trial. 2. Written informed consent must be obtained before any assessment is performed. 3. Patient/Parent/legal guardian willing and able to comply with study procedures. Exclusion Criteria: There are no exclusion criteria for this study.
Interventions
- Biologicalonasemnogene abeparvovec
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)
- Child Hosp Of The Kings DaughtersNorfolk, Virginia
- Novartis Investigative SiteSydney, New South Wales
- Novartis Investigative SiteLeuven
- Novartis Investigative SiteCuritiba, Paraná
- Novartis Investigative SiteSão Paulo, São Paulo
- Novartis Investigative SiteMontreal, Quebec