Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical Trials (SPECTRUM)

Novartis

Status and phase: Enrolling; Phase 3

Conditions: Spinal Muscular Atrophy (SMA)

Treatments: Biological: onasemnogene abeparvovec

Study type: Interventional

Funder types: Industry

Identifiers

NCT05335876

COAV101A12308

Details and patient eligibility

About

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.

Full 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.

Enrollment: 175 estimated patients

Sex: All

Ages: Under 100 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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175 participants in 1 patient group

Intravenous (IV) & Intrathecal (IT) Onasemnogene Abeparvovec

Experimental group

Description:

Patients who received OAV101 IT or OAV101 IV in clinical trials (COAV101A12306, COAV101B12301 and COAV101B12302)

Treatment:

Biological: onasemnogene abeparvovec