An interventional study of Dosage of AAV neutralizing antibodies in Neuromuscular Deficits and Genetic Disorder, sponsored by Genethon. Not yet recruiting at 1 site in France. Open to participants aged 6 Years to 60 Years. Per ClinicalTrials.gov, last updated 2026-06-08.
Sponsored by Genethon · Not applicable, Interventional, and Screening
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.
This research aims to determine the seroprevalence and immunological profiles of recent and/or emerging AAV capsids that could be used in future gene therapy programs. It will thus make it possible to: 1/ improve knowledge of the prevalence of anti-AAV antibodies (total and neutralizing) in these specific populations; 2/ identify the clinical, demographic or biological factors likely to influence this seroprevalence (age, sex, type of pathology, immunosuppressive treatment followed); 3/ to characterize the phenomena of cross-reactivity between different serotypes or variants of capsids, a major challenge for the design of new generations of vectors.
The data generated will contribute to a better estimation of the populations potentially eligible for gene therapies, by making it possible to anticipate exclusion rates related to pre-existing immunity. They may also guide the rational selection of alternative capsids that are less immunogenic or evade cross-immunity, as well as the development of immunomodulation or re-assay strategies.
583 studies on the registry are indexed under Genetic Diseases, Inborn; 157 are open to participants now.
This study's planned enrollment of 450 is above the median of 40 across 305 interventional studies indexed under Genetic Diseases, Inborn.
Browse Genetic Diseases, Inborn studies →Genethon is the lead sponsor of 15 studies on the registry; 5 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Diagnostic Test: 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.
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.
Time frame: Day 1
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)
Time frame: Day 1
To analyze the co-prevalence between several AAV serotypes
Quantification of neutralizing antibody titers against various capsids using cellular tests
Time frame: Day 1
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
Time frame: Day 1
Plan to share: No — Individual participant data (IPD) will not be shared due to the sensitive nature of the data and the need to protect patient confidentiality, in accordance with applicable data protection regulations.
No publications or documents are linked to this record.
Start is estimated for Jul 2026; primary completion for Dec 2027, 17 months later.
Of 64 non-industry interventional studies with no phase under Genetic Diseases, Inborn, half reached primary completion within 41 months of starting (the median), counting those still running; a quarter within 22 and three quarters within 60. This study's planned 17 months is below that.
Counting every sponsor, half of 66 studies reached it within 41 months; a quarter within 22 and three quarters within 60.
How these figures are worked out, and which studies are left out of them.
This study is not yet recruiting, as verified in May 2026. You cannot join it, but the record below documents what was studied.
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