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RECRUITINGPhase 3INTERVENTIONAL

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

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

Important: This information is not medical advice. Talk to your doctor about whether a clinical trial is right for you.

About This Trial

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.

Who May Be Eligible (Plain English)

Who May Qualify: 1. Participated in an OAV101 clinical trial. 2. Written willing to sign a consent form must be obtained before any assessment is performed. 3. Patient/Parent/legal guardian willing and able to comply with study procedures. Who Should NOT Join This Trial: There are no exclusion criteria for this study. Always talk to your doctor about whether this trial is right for you.

Original Eligibility Criteria

View original clinical language
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.

Treatments Being Tested

BIOLOGICAL

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.

Locations (20)

Child Hosp Of The Kings Daughters
Norfolk, Virginia, United States
Novartis Investigative Site
Sydney, New South Wales, Australia
Novartis Investigative Site
Leuven, Belgium
Novartis Investigative Site
Curitiba, Paraná, Brazil
Novartis Investigative Site
São Paulo, São Paulo, Brazil
Novartis Investigative Site
Montreal, Quebec, Canada
Novartis Investigative Site
Chongqing, Chongqing Municipality, China
Novartis Investigative Site
Guangzhou, Guangdong, China
Novartis Investigative Site
Chengdu, Sichuan, China
Novartis Investigative Site
Beijing, China
Novartis Investigative Site
Beijing, China
Novartis Investigative Site
Beijing, China
Novartis Investigative Site
Copenhagen, Denmark
Novartis Investigative Site
Bron, France
Novartis Investigative Site
Garches, France
Novartis Investigative Site
Strasbourg, France
Novartis Investigative Site
Toulouse, France
Novartis Investigative Site
Roma, RM, Italy
Novartis Investigative Site
Kurume, Fukuoka, Japan
Novartis Investigative Site
Shinjuku Ku, Tokyo, Japan