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

NCT05335876 | Recruiting Phase 3 DMC FDA Drug

• 1 other identifier: COAV101A12308
• Study Type: interventional
• Target: 175
• Locations: 19 countries
• Sites: 32

Brief 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.

Conditions

Spinal Muscular Atrophy (SMA)

Keywords

Zolgensma; OAV101; AVXS 101; gene therapy; Muscle atrophy; SBMA; spinal and bulbar muscular atrophy; spinal muscular atrophy; bulbar muscular atrophy; muscle function; myopathy; muscle wasting; atrophied muscle; loss of muscle strength; Spinal Muscular Atrophy (SMA); survival motor neuron 1 gene (SMN1); SMN protein depletion; survival motor neuron 2 gene (SMN2); chromosome 5q13; neurogenetic disorder; onasemnogene abeparvovec

Outcome Measures

Primary Outcomes (2)

• Number of participants with treatment-emergent serious adverse events (SAEs)

  An SAE is defined as any adverse event \[appearance of (or worsening of any pre-existing)\] undesirable sign(s), symptom(s), or medical conditions(s) which meets any one of the following criteria: * fatal * life-threatening * results in persistent or significant disability/incapacity * constitutes a congenital anomaly/birth defect, fetal death or congenital abnormality or birth defect * requires in-patient hospitalization or prolongation of existing hospitalization, unless hospitalization is for routine treatment or monitoring of the studied indication, not associated with any deterioration in condition * is medically significant, e.g. defined as an event that jeopardizes the participant or may require medical or surgical intervention to prevent one of the outcomes listed above

  Up to Year 5

• Number of participants with treatment emergent Adverse Events of Special Interest (AESI)

  The following are important identified and important potential risks (AESI) associated with OAV101: Hepatotoxicity, Transient Thrombocytopenia, Cardiac adverse events, Sensory abnormalities suggestive of ganglionopathy, and Thrombotic microangiopathy. These will be assessed by the investigator.

  Up to Year 5

Secondary Outcomes (9)

• The number of participants demonstrating each developmental milestone according to the Developmental Milestone Checklist

  Up to Year 5

• The number of participants demonstrating maintenance of each developmental milestone according to the Developmental Milestone Checklist

  Up to Year 5

• Change from Baseline in the Hammersmith Functional Motor Scale - Expanded (HFMSE) total score

  Up to Year 5

• Change from Baseline in the Revised Upper Limb Module (RULM) total score

  Up to Year 5

• Systolic and diastolic blood pressure (mmHg)

  Up to Year 5

Study Arms (1)

Intravenous (IV) & Intrathecal (IT) Onasemnogene Abeparvovec

EXPERIMENTAL

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

Biological: onasemnogene abeparvovec

Interventions

onasemnogene abeparvovec BIOLOGICAL

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.

Also known as: Zolgensma

Intravenous (IV) & Intrathecal (IT) Onasemnogene Abeparvovec

Eligibility Criteria

• Age: 0 Years - 100 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (32)

Child Hosp Of The Kings Daughters

Norfolk, Virginia, 23507, United States

RECRUITING

Novartis Investigative Site

Sydney, New South Wales, 2031, Australia

RECRUITING

Novartis Investigative Site

Leuven, 3000, Belgium

RECRUITING

Novartis Investigative Site

Curitiba, Paraná, 81520-060, Brazil

RECRUITING

Novartis Investigative Site

São Paulo, São Paulo, 05403 000, Brazil

RECRUITING

Novartis Investigative Site

Montreal, Quebec, H4A 3J1, Canada

RECRUITING

Novartis Investigative Site

Chongqing, Chongqing Municipality, 400010, China

RECRUITING

Novartis Investigative Site

Guangzhou, Guangdong, 510623, China

RECRUITING

Novartis Investigative Site

Chengdu, Sichuan, 610041, China

RECRUITING

Novartis Investigative Site

Beijing, 100034, China

RECRUITING

Novartis Investigative Site

Beijing, 100069, China

RECRUITING

Novartis Investigative Site

Beijing, 100730, China

RECRUITING

Novartis Investigative Site

Copenhagen, 2100 O, Denmark

RECRUITING

Novartis Investigative Site

Bron, 69677, France

RECRUITING

Novartis Investigative Site

Garches, 92380, France

RECRUITING

Novartis Investigative Site

Strasbourg, 67000, France

RECRUITING

Novartis Investigative Site

Toulouse, 31059, France

RECRUITING

Novartis Investigative Site

Roma, RM, 00168, Italy

RECRUITING

Novartis Investigative Site

Kurume, Fukuoka, 830-0011, Japan

RECRUITING

Novartis Investigative Site

Shinjuku Ku, Tokyo, 1628666, Japan

RECRUITING

Novartis Investigative Site

Kuala Lumpur, 50300, Malaysia

RECRUITING

Novartis Investigative Site

Kuala Lumpur, 59100, Malaysia

RECRUITING

Novartis Investigative Site

Utrecht, 3584 CX, Netherlands

RECRUITING

Novartis Investigative Site

Riyadh, 11211, Saudi Arabia

RECRUITING

Novartis Investigative Site

Singapore, 119074, Singapore

RECRUITING

Novartis Investigative Site

Barcelona, 08035, Spain

RECRUITING

Novartis Investigative Site

Kaohsiung City, 80756, Taiwan

RECRUITING

Novartis Investigative Site

Taipei, 10002, Taiwan

RECRUITING

Novartis Investigative Site

Bangkok, 10700, Thailand

RECRUITING

Novartis Investigative Site

London, WC1N 3JH, United Kingdom

RECRUITING

Novartis Investigative Site

Newcastle upon Tyne, NE1 4LP, United Kingdom

RECRUITING

Novartis Investigative Site

Hanoi, 100000, Vietnam

RECRUITING

MeSH Terms

Conditions

Muscular Atrophy, Spinal; Muscular Atrophy; Bulbo-Spinal Atrophy, X-Linked; Muscular Diseases

Interventions

Zolgensma

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; Neuromuscular Diseases; Neuromuscular Manifestations; Neurologic Manifestations; Atrophy; Pathological Conditions, Anatomical; Pathological Conditions, Signs and Symptoms; Signs and Symptoms; Heredodegenerative Disorders, Nervous System; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Musculoskeletal Diseases

Central Study Contacts

Novartis Pharmaceuticals

CONTACT

1-888-669-6682; novartis.email@novartis.com

Novartis Pharmaceuticals

CONTACT

+41613241111

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 12, 2022
• First Posted: April 20, 2022
• Study Start: December 19, 2022
• Primary Completion (Estimated): September 26, 2030
• Study Completion (Estimated): February 27, 2031
• Last Updated: January 20, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will share

Locations

IT (1); CA (1); ES (1); TW (2); US (1); BE (1); NL (1); MY (2); BR (2); CN (6); FR (4); DK (1); TH (1); GB (2); JP (2); SA (1); VN (1); AU (1); SG (1)