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. (SeroDysMyo)
Studieoversikt
Status
Intervensjon / Behandling
Detaljert beskrivelse
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.
Studietype
Registrering (Antatt)
Fase
- Ikke aktuelt
Kontakter og plasseringer
Studiesteder
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Île-de-France Region
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Évry, Île-de-France Region, Frankrike, 9100
- GENETHON
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Ta kontakt med:
- Philippe VERON, Master of Sciences
- E-post: veron@genethon.fr
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Ta kontakt med:
- Céline LABETOULE, Master of Sciences
- E-post: clabetoule@genethon.fr
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Deltakelseskriterier
Kvalifikasjonskriterier
Alder som er kvalifisert for studier
- Barn
- Voksen
Tar imot friske frivillige
Beskrivelse
Inclusion Criteria:
- 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
Exclusion Criteria:
- Gene or cell therapy treatment prior to blood collection
Studieplan
Hvordan er studiet utformet?
Designdetaljer
- Primært formål: Screening
- Tildeling: N/A
- Intervensjonsmodell: Enkeltgruppeoppdrag
- Masking: Ingen (Open Label)
Våpen og intervensjoner
Deltakergruppe / Arm |
Intervensjon / Behandling |
|---|---|
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Annen: Cohort of 450 patients with neuro-muscular disease for AAV sero-analysis
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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. |
Hva måler studien?
Primære resultatmål
Resultatmål |
Tiltaksbeskrivelse |
Tidsramme |
|---|---|---|
|
Percentage of patients with anti-AAV neutralizing antibodies
Tidsramme: Day 1
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The primary endpoint is the percentage of patients who tested positive for neutralizing antibodies against multiple AAVs that can be used in gene therapy.
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Day 1
|
Sekundære resultatmål
Resultatmål |
Tiltaksbeskrivelse |
Tidsramme |
|---|---|---|
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To estimate seroprevalence in subgroups of patients (age; type of neuromuscular disease; gender; immunosuppressive therapy)
Tidsramme: Day 1
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Estimation of seroprevalence (positive or negative) of anti-AAV neutralizing antibodies in different subgroups (age, sex, type of neuromuscular disease, immunosuppressive therapy)
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Day 1
|
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To analyze the co-prevalence between several AAV serotypes
Tidsramme: Day 1
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Quantification of neutralizing antibody titers against various capsids using cellular tests
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Day 1
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Analyze the potential correlation between the presence of neutralizing antibodies and total antibody titer using cellular tests and ELISA method
Tidsramme: Day 1
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Prevalence of cross-reactions between multiple capsids
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Day 1
|
Samarbeidspartnere og etterforskere
Sponsor
Studierekorddatoer
Studer hoveddatoer
Studiestart (Antatt)
Primær fullføring (Antatt)
Studiet fullført (Antatt)
Datoer for studieregistrering
Først innsendt
Først innsendt som oppfylte QC-kriteriene
Først lagt ut (Faktiske)
Oppdateringer av studieposter
Sist oppdatering lagt ut (Faktiske)
Siste oppdatering sendt inn som oppfylte QC-kriteriene
Sist bekreftet
Mer informasjon
Begreper knyttet til denne studien
Nøkkelord
Ytterligere relevante MeSH-vilkår
Andre studie-ID-numre
- GNT-020-SDM
- ID RCB N° 2026-A00986-45 (Annen identifikator: French health authority (ANSM))
Plan for individuelle deltakerdata (IPD)
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IPD-planbeskrivelse
Legemiddel- og utstyrsinformasjon, studiedokumenter
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