Non-interventional Study of Seroprevalence of Pre-existing Antibodies Against Adenovirus-associated Virus Vector (AAV9) and the Progression of Disease in Patients With Plakophilin 2 (PKP2)-Associated Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC)
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
- Это наблюдательное исследование: исследуемое лечение участникам по протоколу не назначают.
- Кому может быть актуально
- Состояния в реестре: Arrhythmogenic Right Ventricular Cardiomyopathy. Базовые параметры: 14 лет — 65 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
- США, Франция, Германия, Италия, Швеция +1
- Следующий шаг
- Сохраните исследование, покажите его лечащему врачу и уточните актуальный статус у исследовательского центра. Расходы, документы и поездка →
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Официальное название
Seroprevalence Study of Pre-existing Antibodies Against Adenovirus-associated Virus Vector (AAV9) in Patients With Plakophilin 2 (PKP2)-Associated Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC)
Обзор
This is a multicenter, non-interventional study to observe the natural progression of the disease and to study the prevalence of pre-existing antibodies to AAV9 used for gene therapy in a population of patients with PKP2 gene-associated ARVC. Participation from all patients is encouraged regardless of interest in or eligibility for gene therapy.
Подробное описание
Patients will receive standard of care treatments and assessments under the care of their healthcare provider. Biologic samples will be collected annually to measure cardiac and other related biomarkers. Clinical and observational data will be collected prospectively for up to 5 years from the date of enrollment, or until the patient withdraws consent/assent, undergoes heart transplantation, or dies.
If consent is provided, there may be a one-time sample collection to evaluate genetics for research purposes. Quality of Life (QoL) questionnaires will be used to assess a patient's wellbeing and quality of life. If not included as part of a patient's standard of care, diagnostic Holter (or equivalent) monitoring will be required annually. No investigational product will be administered. Participation from all patients is encouraged regardless of interest in or eligibility for gene therapy.
Первичные конечные точки
- Investigate the seroprevalence of pre-existing antibodies to AAV9 in patients with PKP2-associated ARVC [Срок оценки: 5 years]
Вторичные конечные точки (4)
- To characterize the burden of illness in patients with pathogenic or likely pathogenic PKP2 mutations [Срок оценки: 5 years]
- To characterize arrhythmic risk in patients with pathogenic or likely pathogenic PKP2 mutations [Срок оценки: 5 years]
- To evaluate functional status and Quality of Life (QoL) in patients with pathogenic or likely pathogenic PKP2 mutations [Срок оценки: 5 years]
- To evaluate heart function as assessed by imaging in patients with pathogenic or likely pathogenic PKP2 mutations [Срок оценки: 5 years]
Критерии участия
Критерии включения
- Ages 14-65 years, inclusive, at the time of consent
- Pathogenic or likely pathogenic PKP2 gene mutation
- Diagnosed with ARVC and meet 2010 Modified Task Force Criteria for ARVC as affected.
- Functioning ICD
Критерии исключения
- Currently receiving systemic immunosuppressive therapy, cytotoxic chemotherapy, immunoglobulin therapy or monoclonal antibody therapy
- History of clinically significant liver disease, hepatitis B virus, hepatitis C virus, human immunodeficiency virus, or tuberculosis infection
- Previously dosed with any investigational or approved gene therapy product at any time
- Concurrent participation in another interventional clinical trial unless approved by the Sponsor. Participation in a noninterventional study may be allowed at the investigator's discretion.
- History of cardiac transplant.
Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.
Здоровые добровольцы: Нет
Дизайн исследования
- Модель наблюдения
- Другое
Центры проведения
США · 8 центров
- University of California San Francisco — San Francisco
- University of Colorado, Denver — Aurora
- John Hopkins University School of Medicine — Baltimore
- Brigham and Women's Hospital — Boston
- Mayo Clinic — Rochester
- New York University — New York
- Cleveland Clinic — Cleveland
- Medical University of South Carolina — Charleston
Франция · 4 центра
- Hopital Louis Pradel — Bron
- Nantes University Hospital — Nantes
- Pitié-Salpêtrière Hospital — Paris
- Hôpital Haut-Lévêque — Pessac
Великобритания · 4 центра
- The Queen Elizabeth Hospital — Glasgow
- Barts & The London Health NHS Trust — London
- St. George's University Hospitals NHS Foundation Trust — London
- Royal Brompton & Harefield NHS Foundation Trust — London
Германия · 2 центра
- University Hospital Muenster — Münster
- Wuerzburg University Hospital — Würzburg
Италия · 2 центра
- Centro Cardiologico Monzino — Milan
- Istituti Clinici Scientifici Maugeri SpA — Pavia
Швеция · 1 центр
- Skåne University Hospital — Malmö
Идентификаторы
NCT: NCT06311708 · TN-401-0011