Last Update: May 24, 2024
Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 IT or OAV101 IV in Clinical Trials
ClinicalTrials.gov Identifier:
Novartis Reference Number:COAV101A12308
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 previous clinical
trial. The study is comprised of a Baseline Period and 3 Follow-up Periods. Follow-up Periods 1
and 2 consist of in-person visits and Period 3 consists of tele-visits. Follow-up Periods
1 and 2, which includes Baseline through Year 5 visits, assessments will be performed at
the Investigational site. During Follow-up Period 3 (Year 6 to up to Year 15 after OAV101
administration), participants/caregivers will be contacted using tele-visits annually for
remote assessments. All patients will enter the study at the baseline visit and continue
until 15 years since OAV101 administration is reached. Total duration of participation in
the study will be dependent upon time of enrollment relative to OAV101 administration and
will vary by participant.

Spinal Muscular Atrophy (SMA)
Phase3
Recruiting
260
Dec 19, 2022
All
- (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:

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.

Worldwide Contacts

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