Registration Study for Rare Type of Pulmonary Hypertension
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: laboratory biomarker analysis, Genetic analysis.
- Кому может быть актуально
- Состояния в реестре: Pulmonary Hypertension. Базовые параметры: Без ограничений · 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
- Китай
- Следующий шаг
- Сохраните исследование, покажите его лечащему врачу и уточните актуальный статус у исследовательского центра. Расходы, документы и поездка →
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Обзор
The knowledge on the rare type of pulmonary hypertension which can not be explained by left heart disease, respiratory disease or congenital heart disease is very limited. Investigators aim to setup a national registration study for the rare type of pulmonary hypertension, to understand the natural history, survival, progression, genetic and environmental contributions to disease.
Подробное описание
The main research contents of this registration study includes:
1. Build a baseline database of the rare type of pulmonary hypertension. Collect general information, on-set symptoms and time, laboratory examination, imaging results, right heart catheterization and treatment information. 2. Follow up recruited patients at regular intervals（6m\~1y). Collect information on change in patients condition, laboratory test and treatment. 3. Conduct genetic testing for gene mutation related or hereditary pulmonary hypertension. Link the clinical database to genetic database. 4. Establish bio-bank for serum/plasma, urine, stool, tissues or cells. 5. Establish prognostic study based on the clinical follow-up and genetic database. 6. Draw diagnostic and treatment algorithm for the rare type of pulmonary hypertension.
Controls subjects: blood sample and medical data collected once.
Вмешательства
- Другое laboratory biomarker analysis
Laboratory results will be analysed to identify disease related biomarkers. - Генная терапия Genetic analysis
Gene sequencing results will be analysed to identify disease related mutations.
Первичные конечные точки
- Survival Rate of Participants [Срок оценки: up to 10 years, at 12 months interval]
- Lung transplantation [Срок оценки: up to 10 years, at 12 months interval]
- Change in New York Heart Association (NYHA) functional class [Срок оценки: up to 10 years, at 3 months interval]
- Change in 6 mint walk distance [Срок оценки: up to 10 years, at 3 months interval]
Вторичные конечные точки (7)
- Genetic alteration in participants with rare type of PH [Срок оценки: Baseline]
- Change in NT-proBNP [Срок оценки: up to 10 years, at 3 months interval]
- Change in hemodynamics [Срок оценки: up to 10 years, at 6 months interval]
- Change in cardiac function [Срок оценки: up to 10 years, at 3-6 months interval]
- Pulmonary endarterectomy (PEA) [Срок оценки: up to 10 years, at 6 months interval]
- Balloon pulmonary angioplasty (BPA) [Срок оценки: up to 10 years, at 6 months interval]
- Medical treatment [Срок оценки: up to 10 years, at 6 months interval]
Критерии участия
Критерии включения
- Participant is willing and able to give informed consent for participation in the study.
- Patients diagnosed as idiopathic pulmonary artery hypertension, hereditary pulmonary artery hypertension, hereditary hemorrhagic telangiectasia associated pulmonary artery hypertension, pulmonary veno-occlusive disease, pulmonary capillary hemangiomatosis associated pulmonary artery hypertension, cavernous transformation of portal vein associated pulmonary artery hypertension, special type of congenital heart disease associated pulmonary artery hypertension, chronic thromboembolism pulmonary hypertension.
- All patients should have undergone right heart catheterization, diagnosed according to the guideline.
Критерии исключения
The participant may not enter the study if ANY of the following apply:
- Patients unwilling or unable to provide written consent for participation in the study.
- Not suffering from the rare type of pulmonary artery hypertension;
Inclusion criteria-Controls
- Participant is willing and able to give informed consent for participation in the study.
- Self-reported to be healthy
Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.
Здоровые добровольцы: Да
Дизайн исследования
- Модель наблюдения
- Когортное
Центры проведения
Китай · 1 центр
- Chinese Academy of Medical Sciences Fuwai Hospital and Peking Union Medical College Hospit — Пекин
Публикации
- Galie N, Corris PA, Frost A, Girgis RE, Granton J, Jing ZC, Klepetko W, McGoon MD, McLaughlin VV, Preston IR, Rubin LJ, Sandoval J, Seeger W, Keogh A. Updated treatment algorithm of pulmonary arterial hypertension. J Am Coll Cardiol. 2013 Dec 24;62(25 Suppl):D60-72. doi: 10.1016/j.jacc.2013.10.031. PMID 24355643
- Simonneau G, Robbins IM, Beghetti M, Channick RN, Delcroix M, Denton CP, Elliott CG, Gaine SP, Gladwin MT, Jing ZC, Krowka MJ, Langleben D, Nakanishi N, Souza R. Updated clinical classification of pulmonary hypertension. J Am Coll Cardiol. 2009 Jun 30;54(1 Suppl):S43-S54. doi: 10.1016/j.jacc.2009.04.012. PMID 19555858
Идентификаторы
NCT: NCT03169010 · RarePH135