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

Last updated: January 16, 2026
Sponsor: Novartis Pharmaceuticals
Overall Status: Active - Recruiting

Phase

3

Condition

Muscular Dystrophy

Myasthenia Gravis (Chronic Weakness)

Spinal Muscular Atrophy

Treatment

onasemnogene abeparvovec

Clinical Study ID

NCT05335876
COAV101A12308
  • Ages < 100
  • All Genders

Study 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.

Eligibility Criteria

Inclusion

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

Exclusion Criteria:

There are no exclusion criteria for this study.

Study Design

Total Participants: 175
Treatment Group(s): 1
Primary Treatment: onasemnogene abeparvovec
Phase: 3
Study Start date:
December 19, 2022
Estimated Completion Date:
February 27, 2031

Study 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.

Connect with a study center

  • Novartis Investigative Site

    Randwick, New South Wales 2031
    Australia

    Site Not Available

  • Novartis Investigative Site

    Sydney 2147714, New South Wales 2155400 2031
    Australia

    Active - Recruiting

  • Novartis Investigative Site

    Leuven, 3000
    Belgium

    Site Not Available

  • Novartis Investigative Site

    Leuven 2792482, 3000
    Belgium

    Active - Recruiting

  • Novartis Investigative Site

    Curitiba 3464975, Paraná 3455077 81520-060
    Brazil

    Active - Recruiting

  • Novartis Investigative Site

    São Paulo 3448439, São Paulo 3448433 05403 000
    Brazil

    Active - Recruiting

  • Novartis Investigative Site

    Montreal, Quebec H4A 3J1
    Canada

    Site Not Available

  • Novartis Investigative Site

    Montreal 6077243, Quebec 6115047 H4A 3J1
    Canada

    Active - Recruiting

  • Novartis Investigative Site

    Chongqing, Chongqing 400010
    China

    Site Not Available

  • Novartis Investigative Site

    Chongqing 1814906, Chongqing Municipality 1814905 400010
    China

    Active - Recruiting

  • Novartis Investigative Site

    Guangzhou, Guangdong 510623
    China

    Site Not Available

  • Novartis Investigative Site

    Guangzhou 1809858, Guangdong 1809935 510623
    China

    Active - Recruiting

  • Novartis Investigative Site

    Chengdu, Sichuan 610041
    China

    Site Not Available

  • Novartis Investigative Site

    Chengdu 1815286, Sichuan 1794299 610041
    China

    Active - Recruiting

  • Novartis Investigative Site

    Beijing, 100069
    China

    Site Not Available

  • Novartis Investigative Site

    Beijing 1816670, 100730
    China

    Active - Recruiting

  • Novartis Investigative Site

    Copenhagen, 2100 O
    Denmark

    Site Not Available

  • Novartis Investigative Site

    Copenhagen 2618425, 2100 O
    Denmark

    Active - Recruiting

  • Novartis Investigative Site

    Bron 3029931, 69677
    France

    Active - Recruiting

  • Novartis Investigative Site

    Bron Cedex, 69677
    France

    Site Not Available

  • Novartis Investigative Site

    Garches, 92380
    France

    Site Not Available

  • Novartis Investigative Site

    Garches 3016675, 92380
    France

    Active - Recruiting

  • Novartis Investigative Site

    Strasbourg, 67000
    France

    Site Not Available

  • Novartis Investigative Site

    Strasbourg 2973783, 67000
    France

    Active - Recruiting

  • Novartis Investigative Site

    Toulouse 2972315, 31059
    France

    Active - Recruiting

  • Novartis Investigative Site

    Toulouse Cedex, 31059
    France

    Site Not Available

  • Novartis Investigative Site

    Roma, RM 00168
    Italy

    Site Not Available

  • Novartis Investigative Site

    Roma 8957247, RM 00168
    Italy

    Active - Recruiting

  • Novartis Investigative Site

    Kurume city, Fukuoka 830-0011
    Japan

    Site Not Available

  • Novartis Investigative Site

    Kurume 1858088, Fukuoka 1863958 830-0011
    Japan

    Active - Recruiting

  • Novartis Investigative Site

    Shinjuku ku, Tokyo 162 8666
    Japan

    Active - Recruiting

  • Novartis Investigative Site

    Shinjuku Ku, Tokyo 1850144 1628666
    Japan

    Active - Recruiting

  • Novartis Investigative Site

    Kuala Lumpur, 50300
    Malaysia

    Site Not Available

  • Novartis Investigative Site

    Kuala Lumpur 1735161, 59100
    Malaysia

    Active - Recruiting

  • Novartis Investigative Site

    Utrecht, 3584CX
    Netherlands

    Site Not Available

  • Novartis Investigative Site

    Utrecht 2745912, 3584 CX
    Netherlands

    Active - Recruiting

  • Novartis Investigative Site

    Riyadh, 11211
    Saudi Arabia

    Site Not Available

  • Novartis Investigative Site

    Riyadh 108410, 11211
    Saudi Arabia

    Active - Recruiting

  • Novartis Investigative Site

    Singapore, 119074
    Singapore

    Site Not Available

  • Novartis Investigative Site

    Singapore 1880252, 119074
    Singapore

    Active - Recruiting

  • Novartis Investigative Site

    Barcelona, Catalunya 08035
    Spain

    Site Not Available

  • Novartis Investigative Site

    Barcelona 3128760, 08035
    Spain

    Active - Recruiting

  • Novartis Investigative Site

    Kaohsiung, 80756
    Taiwan

    Site Not Available

  • Novartis Investigative Site

    Kaohsiung City 1673820, 80756
    Taiwan

    Active - Recruiting

  • Novartis Investigative Site

    Taipei, 10002
    Taiwan

    Site Not Available

  • Novartis Investigative Site

    Taipei 1668341, 10002
    Taiwan

    Active - Recruiting

  • Novartis Investigative Site

    Bangkok, 10700
    Thailand

    Site Not Available

  • Novartis Investigative Site

    Bangkok 1609350, 10700
    Thailand

    Active - Recruiting

  • Novartis Investigative Site

    London, WC1N 3JH
    United Kingdom

    Site Not Available

  • Novartis Investigative Site

    London 2643743, WC1N 3JH
    United Kingdom

    Active - Recruiting

  • Novartis Investigative Site

    Newcastle Upon Tyne, NE1 4LP
    United Kingdom

    Site Not Available

  • Novartis Investigative Site

    Newcastle upon Tyne 2641673, NE1 4LP
    United Kingdom

    Active - Recruiting

  • Child Hosp of the Kings Daughters

    Norfolk, Virginia 23507
    United States

    Site Not Available

  • Child Hosp Of The Kings Daughters

    Norfolk 4776222, Virginia 6254928 23507
    United States

    Active - Recruiting

  • Novartis Investigative Site

    Hanoi, 100000
    Vietnam

    Site Not Available

  • Novartis Investigative Site

    Hanoi 1581130, 100000
    Vietnam

    Active - Recruiting

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