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.
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
Gene or cell therapy treatment prior to blood collection
Single group
Dosage of AAV neutralizing antibodies
Diagnostic testIn 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.
Percentage of patients with anti-AAV neutralizing antibodies
The primary endpoint is the percentage of patients who tested positive for neutralizing antibodies against multiple AAVs that can be used in gene therapy.
To estimate seroprevalence in subgroups of patients (age; type of neuromuscular disease; gender; immunosuppressive therapy)
Estimation of seroprevalence (positive or negative) of anti-AAV neutralizing antibodies in different subgroups (age, sex, type of neuromuscular disease, immunosuppressive therapy)
To analyze the co-prevalence between several AAV serotypes
Quantification of neutralizing antibody titers against various capsids using cellular tests
Analyze the potential correlation between the presence of neutralizing antibodies and total antibody titer using cellular tests and ELISA method
Prevalence of cross-reactions between multiple capsids
This trial is not recruiting at the moment. You can still explore other options:
