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Contact the study team using the details below to take part. If there are no contact details below please ask your doctor in the first instance.

Contact Information:

Novartis Pharmaceuticals +41613241111


Novartis Pharmaceuticals 1-888-669-6682
novartis.email@novartis.com


Study Location:

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Be Part of Research - Trial Details - Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical Trials
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Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical Trials

Recruiting

Open to: ALL

Age: 0.0 - 100.0

Newcastle upon Tyne London

Medical Conditions

Spinal Muscular Atrophy (SMA)

This information is provided directly by researchers, and we recognise that it isn't always easy to understand. We are working with researchers to improve the accessibility of this information. In some summaries, you may come across links to external websites. These websites will have more information to help you better understand the study.


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.

Start dates may differ between countries and research sites. The research team are responsible for keeping the information up-to-date.  

The recruitment start and end dates are as follows:

Dec 2022 Sep 2030

INTERVENTIONAL

Intervention Type : BIOLOGICAL
Intervention Description : 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 Arm Group : Intravenous (IV) & Intrathecal (IT) Onasemnogene Abeparvovec;



You can take part if:



You may not be able to take part if:


This is in the inclusion criteria above


Below are the locations for where you can take part in the trial. Please note that not all sites may be open.

  • Novartis Investigative Site
    Newcastle upon Tyne
    NE1 4LP
  • Novartis Investigative Site
    London
    WC1N 3JH

Novartis Pharmaceuticals 1-888-669-6682
novartis.email@novartis.com


Novartis Pharmaceuticals +41613241111



The study is sponsored by Novartis Pharmaceuticals




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Read full details for Trial ID: NCT05335876
Last updated 16 January 2026

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