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

ClinicalTrials.gov Identifier: NCT05335876

Novartis Reference Number: COAV101A12308

Last Update: Feb 08, 2023

All compounds are either investigational or being studied for (a) new use(s). Efficacy and safety have not been established. There is no guarantee that they will become commercially available for the use(s) under investigation.

Study Description

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 15 years from the date of OAV101 administration in the parent study.

Condition

Spinal Muscular Atrophy (SMA)

Phase

Phase 3

Overall status

Recruiting

Start date

Dec 19, 2022

Completion date

Oct 18, 2039

Gender

All

Age(s)

(Child, Adult, Older Adult)

Interventions

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.

Eligibility Criteria

Inclusion Criteria:

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.

Exclusion Criteria:

There are no exclusion criteria for this study.