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

Plain-language summary

This study provides long-term follow-up of patients with spinal muscular atrophy who had previously taken part in an OAV101 (onasemnogene abeparvovec) clinical trial. It enrolls patients who participated in an OAV101 clinical trial and can comply with the study procedures. The study is in Phase 3 and is currently recruiting participants.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

Phase 3

Status

Active, not recruiting

Sponsor

Novartis Pharmaceuticals

Intervention

onasemnogene abeparvovec

Start date

12/19/2022

Estimated completion

4/30/2031

Countries

United States, Australia, Belgium, Brazil, Canada, China, Denmark, France, Italy, Japan, Malaysia, Netherlands, Saudi Arabia, Singapore, Spain, Taiwan, Thailand, United Kingdom, Vietnam

Related genes

Locations

• Norfolk, United States
• Sydney, Australia
• Leuven, Belgium
• Curitiba, Brazil
• São Paulo, Brazil
• Montreal, Canada
• Chongqing, China
• Guangzhou, China
• Chengdu, China
• Beijing, China
• Beijing, China
• Beijing, China
• Copenhagen, Denmark
• Bron, France
• Garches, France
• Strasbourg, France
• Toulouse, France
• Roma, Italy
• Kurume, Japan
• Shinjuku Ku, Japan
• Kuala Lumpur, Malaysia
• Kuala Lumpur, Malaysia
• Utrecht, Netherlands
• Riyadh, Saudi Arabia
• Singapore, Singapore
• Barcelona, Spain
• Kaohsiung City, Taiwan
• Taipei, Taiwan
• Bangkok, Thailand
• London, United Kingdom
• Newcastle upon Tyne, United Kingdom
• Hanoi, Vietnam

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

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.