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.

ConditionsNeuromuscular Deficits Genetic Disorder

Trial statusNot yet recruiting

Trial phaseNot applicable

Trial typeInterventional

Biological sexAll

Age6-60

SponsorGenethon

About this trial

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.

Eligibility criteria

Qualifiers

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

Disqualifiers

Gene or cell therapy treatment prior to blood collection

Trial design

Treatments tested in this trial

Dosage of AAV neutralizing antibodies

Treatment groups

450 Participants

are divided into 1 treatment group

Sponsors and collaborators

Genethon

Lead sponsor

Source ClinicalTrials.gov