Набор скоро начнётся NCT07633951

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.

Без фазы С лечением Neuromuscular Deficits Genetic Disorder

Ориентир для пациента и семьи

Простыми словами

Автоматическая сводка по структурированным данным реестра. Она помогает сориентироваться, но не заменяет официальный протокол или оценку врача.

Что изучают: В протоколе указаны: Dosage of AAV neutralizing antibodies.
Кому может быть актуально: Состояния в реестре: Neuromuscular Deficits, Genetic Disorder. Базовые параметры: 6 лет — 60 лет · Все.
Что важно проверить: Возраст, диагноз и пол — только базовые ориентиры. Предыдущее лечение, анализы и другие обязательные условия указаны ниже в критериях участия.
Где проводится: Франция
Следующий шаг: Сохраните исследование, покажите его лечащему врачу и уточните актуальный статус у исследовательского центра. Расходы, документы и поездка →

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Обзор

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.

Подробное описание

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.

Вмешательства

Диагностический тест 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.

Первичные конечные точки

Percentage of patients with anti-AAV neutralizing antibodies [Срок оценки: Day 1]

Вторичные конечные точки (3)

To estimate seroprevalence in subgroups of patients (age; type of neuromuscular disease; gender; immunosuppressive therapy) [Срок оценки: Day 1]
To analyze the co-prevalence between several AAV serotypes [Срок оценки: Day 1]
Analyze the potential correlation between the presence of neutralizing antibodies and total antibody titer using cellular tests and ELISA method [Срок оценки: Day 1]

Критерии участия

Критерии включения

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