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