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Not Yet Recruiting NCT07633951

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.

Conditions: Neuromuscular Deficits, Genetic Disorder

Sex: All
Ages: 6 Years – 60 Years
Healthy volunteers: No
Phase: NA
Enrollment: 450
Sponsor: Genethon

Location: GENETHON Évry Île-de-France Region

Summary

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

Inclusion Criteria: 1. Pediatric patient 6 years of age or older or adult under 60 years of age with genetic neuromuscular disease 2. Informed patient who signed informed consent 3. No opposition from the holders of parental authority or guardian, for minor patients. 4. Affiliated/beneficiary of a national health insurance scheme Exclusion Criteria: * Gene or cell therapy treatment prior to blood collection

Interested in this study? View the official listing for contact and enrollment details.

View on ClinicalTrials.gov

Source: ClinicalTrials.gov (NCT07633951). StuddyBuddy aggregates publicly available trial information.