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

NCT ID: NCT05335876

Last Updated: 2024-12-10

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE3

Total Enrollment

175 participants

Study Classification

INTERVENTIONAL

Study Start Date

2022-12-19

Study Completion Date

2030-06-26

Brief Summary

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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.

Detailed Description

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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.

Conditions

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Spinal Muscular Atrophy (SMA)

Keywords

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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

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Intravenous (IV) & Intrathecal (IT) Onasemnogene Abeparvovec

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

Group Type EXPERIMENTAL

onasemnogene abeparvovec

Intervention Type 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.

Interventions

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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.

Intervention Type BIOLOGICAL

Other Intervention Names

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Zolgensma

Eligibility Criteria

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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.
Minimum Eligible Age

0 Years

Maximum Eligible Age

100 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Novartis Pharmaceuticals

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Child Hosp Of The Kings Daughters

Norfolk, Virginia, United States

Site Status RECRUITING

Novartis Investigative Site

Randwick, New South Wales, Australia

Site Status RECRUITING

Novartis Investigative Site

Leuven, , Belgium

Site Status RECRUITING

Novartis Investigative Site

Montreal, Quebec, Canada

Site Status RECRUITING

Novartis Investigative Site

Chongqing, Chongqing Municipality, China

Site Status RECRUITING

Novartis Investigative Site

Guangzhou, Guangdong, China

Site Status RECRUITING

Novartis Investigative Site

Chengdu, Sichuan, China

Site Status RECRUITING

Novartis Investigative Site

Beijing, , China

Site Status RECRUITING

Novartis Investigative Site

Beijing, , China

Site Status RECRUITING

Novartis Investigative Site

Copenhagen, , Denmark

Site Status RECRUITING

Novartis Investigative Site

Bron, , France

Site Status RECRUITING

Novartis Investigative Site

Garches, , France

Site Status RECRUITING

Novartis Investigative Site

Strasbourg, , France

Site Status RECRUITING

Novartis Investigative Site

Toulouse, , France

Site Status RECRUITING

Novartis Investigative Site

Roma, RM, Italy

Site Status RECRUITING

Novartis Investigative Site

Kurume, Fukuoka, Japan

Site Status RECRUITING

Novartis Investigative Site

Shinjuku Ku, Tokyo, Japan

Site Status RECRUITING

Novartis Investigative Site

Kuala Lumpur, , Malaysia

Site Status RECRUITING

Novartis Investigative Site

Kuala Lumpur, , Malaysia

Site Status RECRUITING

Novartis Investigative Site

Utrecht, , Netherlands

Site Status RECRUITING

Novartis Investigative Site

Riyadh, , Saudi Arabia

Site Status RECRUITING

Novartis Investigative Site

Singapore, , Singapore

Site Status RECRUITING

Novartis Investigative Site

Barcelona, Catalonia, Spain

Site Status RECRUITING

Novartis Investigative Site

Kaohsiung City, , Taiwan

Site Status RECRUITING

Novartis Investigative Site

Taipei, , Taiwan

Site Status RECRUITING

Novartis Investigative Site

Bangkok, , Thailand

Site Status RECRUITING

Novartis Investigative Site

London, , United Kingdom

Site Status RECRUITING

Novartis Investigative Site

Newcastle upon Tyne, , United Kingdom

Site Status RECRUITING

Novartis Investigative Site

Hanoi, , Vietnam

Site Status RECRUITING

Countries

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

Central Contacts

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Novartis Pharmaceuticals

Role: CONTACT

Phone: 1-888-669-6682

Email: [email protected]

Novartis Pharmaceuticals

Role: CONTACT

Phone: +41613241111

Email: [email protected]

Facility Contacts

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Erin Smith

Role: primary

Other Identifiers

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COAV101A12308

Identifier Type: -

Identifier Source: org_study_id