NCT05335876

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

• Status: Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 175 (estimated)
• Sponsor: Novartis Pharmaceuticals · Industry
• Sex: All
• Age: 0 Years – 100 Years
• Healthy volunteers: Not accepted

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.

Detailed 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.

Conditions

• Spinal Muscular Atrophy (SMA)

Interventions

Type	Name	Description
BIOLOGICAL	onasemnogene 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.

Timeline

• Start date: 2022-12-19
• Primary completion: 2030-09-26
• Completion: 2031-02-27
• First posted: 2022-04-20
• Last updated: 2026-01-20

Locations

32 sites across 19 countries: United States, Australia, Belgium, Brazil, Canada, China, Denmark, France, Italy, Japan, Malaysia, Netherlands, Saudi Arabia, Singapore, Spain, Taiwan, Thailand, United Kingdom, Vietnam

Regulatory

• FDA-regulated drug study