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)
Studieöversikt
Status
Betingelser
Intervention / Behandling
Detaljerad beskrivning
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.
Studietyp
Inskrivning (Beräknad)
Fas
- Inte tillämpbar
Kontakter och platser
Studieorter
-
-
Île-de-France Region
-
Évry, Île-de-France Region, Frankrike, 9100
- GENETHON
-
Kontakt:
- Philippe VERON, Master of Sciences
- E-post: veron@genethon.fr
-
Kontakt:
- Céline LABETOULE, Master of Sciences
- E-post: clabetoule@genethon.fr
-
-
Deltagandekriterier
Urvalskriterier
Åldrar som är berättigade till studier
- Barn
- Vuxen
Tar emot friska volontärer
Beskrivning
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
Hur är studien utformad?
Designdetaljer
- Primärt syfte: Undersökning
- Tilldelning: N/A
- Interventionsmodell: Enskild gruppuppgift
- Maskning: Ingen (Open Label)
Vapen och interventioner
Deltagargrupp / Arm |
Intervention / Behandling |
|---|---|
|
Övrig: Cohort of 450 patients with neuro-muscular disease for AAV sero-analysis
|
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. |
Vad mäter studien?
Primära resultatmått
Resultatmått |
Åtgärdsbeskrivning |
Tidsram |
|---|---|---|
|
Percentage of patients with anti-AAV neutralizing antibodies
Tidsram: Day 1
|
The primary endpoint is the percentage of patients who tested positive for neutralizing antibodies against multiple AAVs that can be used in gene therapy.
|
Day 1
|
Sekundära resultatmått
Resultatmått |
Åtgärdsbeskrivning |
Tidsram |
|---|---|---|
|
To estimate seroprevalence in subgroups of patients (age; type of neuromuscular disease; gender; immunosuppressive therapy)
Tidsram: Day 1
|
Estimation of seroprevalence (positive or negative) of anti-AAV neutralizing antibodies in different subgroups (age, sex, type of neuromuscular disease, immunosuppressive therapy)
|
Day 1
|
|
To analyze the co-prevalence between several AAV serotypes
Tidsram: Day 1
|
Quantification of neutralizing antibody titers against various capsids using cellular tests
|
Day 1
|
|
Analyze the potential correlation between the presence of neutralizing antibodies and total antibody titer using cellular tests and ELISA method
Tidsram: Day 1
|
Prevalence of cross-reactions between multiple capsids
|
Day 1
|
Samarbetspartners och utredare
Sponsor
Studieavstämningsdatum
Studera stora datum
Studiestart (Beräknad)
Primärt slutförande (Beräknad)
Avslutad studie (Beräknad)
Studieregistreringsdatum
Först inskickad
Först inskickad som uppfyllde QC-kriterierna
Första postat (Faktisk)
Uppdateringar av studier
Senaste uppdatering publicerad (Faktisk)
Senaste inskickade uppdateringen som uppfyllde QC-kriterierna
Senast verifierad
Mer information
Termer relaterade till denna studie
Nyckelord
Ytterligare relevanta MeSH-villkor
Andra studie-ID-nummer
- GNT-020-SDM
- ID RCB N° 2026-A00986-45 (Annan identifierare: French health authority (ANSM))
Plan för individuella deltagardata (IPD)
Planerar du att dela individuella deltagardata (IPD)?
IPD-planbeskrivning
Läkemedels- och apparatinformation, studiedokument
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