Clinical Research Directory
Browse clinical research sites, groups, and studies.
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.
Sponsor: Genethon
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.
Key Details
Gender
All
Age Range
6 Years - 60 Years
Study Type
INTERVENTIONAL
Enrollment
450
Start Date
2026-07-31
Completion Date
2027-12-31
Last Updated
2026-06-08
Healthy Volunteers
No
Conditions
Interventions
Dosage of AAV neutralizing antibodies
In each clinical site, the principal investigator will propose to his patients with neuromuscular disease of genetic origin to take a blood sample (4 mL of blood) to determine their level of immunity to certain AAVs used in gene therapy. The collected samples will be prepared and aliquoted on site and then sent frozen to the GENETHON laboratory for analysis. Only one sample per patient will be analyzed. All samples will be analyzed in batches.
Locations (1)
GENETHON
Évry, Île-de-France Region, France