Multicenter Epidemiological Study for the Evaluation of the Seroprevalence of Total and Neutralizing Antibodies Against Adenoassociated Virus Serotypes Used in Gene Therapy in Patients With Neuromuscular Diseases of Genetic Origin.

Starting soon · Not applicable

Conditions studied: Neuromuscular Deficits, Genetic Disorder

In brief

This study aims to assess the overall seroprevalence of neutralizing antibodies against different AAVs potentially used in gene therapy in patients with genetic neuromuscular diseases.

Key facts

Study ID

NCT07633951

Run by

Genethon

People needed

450

Starts

2026-07-31

Expected to finish

2027-12-31

Last updated by the study team

2026-06-08

Who can join

Age: 6 and older, up to 60. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Pediatric patient 6 years of age or older or adult under 60 years of age with genetic neuromuscular disease
• Informed patient who signed informed consent
• No opposition from the holders of parental authority or guardian, for minor patients.
• Affiliated/beneficiary of a national health insurance scheme

You may not qualify if…

• Gene or cell therapy treatment prior to blood collection

Where it is running

• GENETHON — Évry, Île-de-France Region, France

Full record on ClinicalTrials.gov

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