Registration Study for Rare Type of Pulmonary Hypertension
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: laboratory biomarker analysis, Genetic analysis.
- Who it may be relevant to
- Registry conditions: Pulmonary Hypertension. Basic parameters: No limits · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- China
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Overview
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.
Detailed description
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.
Interventions
- Other laboratory biomarker analysis
Laboratory results will be analysed to identify disease related biomarkers. - Genetic Genetic analysis
Gene sequencing results will be analysed to identify disease related mutations.
Primary outcome measures
- Survival Rate of Participants [Time frame: up to 10 years, at 12 months interval]
- Lung transplantation [Time frame: up to 10 years, at 12 months interval]
- Change in New York Heart Association (NYHA) functional class [Time frame: up to 10 years, at 3 months interval]
- Change in 6 mint walk distance [Time frame: up to 10 years, at 3 months interval]
Secondary outcome measures (7)
- Genetic alteration in participants with rare type of PH [Time frame: Baseline]
- Change in NT-proBNP [Time frame: up to 10 years, at 3 months interval]
- Change in hemodynamics [Time frame: up to 10 years, at 6 months interval]
- Change in cardiac function [Time frame: up to 10 years, at 3-6 months interval]
- Pulmonary endarterectomy (PEA) [Time frame: up to 10 years, at 6 months interval]
- Balloon pulmonary angioplasty (BPA) [Time frame: up to 10 years, at 6 months interval]
- Medical treatment [Time frame: up to 10 years, at 6 months interval]
Eligibility criteria
Inclusion criteria
- 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.
Exclusion criteria
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
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: Yes
Study design
- Observational model
- Cohort
Study locations
China · 1 center
- Chinese Academy of Medical Sciences Fuwai Hospital and Peking Union Medical College Hospit — Beijing
Publications
- 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
Identifiers
NCT: NCT03169010 · RarePH135