Registration Study for Rare Type of Pulmonary Hypertension

Recruiting

• Conditions: Pulmonary Hypertension

• Registration Number: NCT03169010
• Lead Sponsor: China National Center for Cardiovascular Diseases

Brief Summary

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.

Study Timeline

• Study Start: 2017-05-06
• Study First Submitted: 2017-05-23
• Study First Submitted QC: 2017-05-25
• Study First Posted: 2017-05-30
• Status Verified: 2023-09
• Last Update Submitted: 2023-09-28
• Last Update Posted: 2023-10-02
• Primary Completion: 2040-12-31
• Study Completion: 2040-12-31

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 2000

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

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Survival Rate of Participants	up to 10 years, at 12 months interval
Lung transplantation	up to 10 years, at 12 months interval
Change in New York Heart Association (NYHA) functional class	up to 10 years, at 3 months interval
Change in 6 mint walk distance	up to 10 years, at 3 months interval

Secondary Outcome Measures

Name	Time	Method
Change in NT-proBNP	up to 10 years, at 3 months interval
Pulmonary endarterectomy (PEA)	up to 10 years, at 6 months interval	e.g. operated versus non-operated
Genetic alteration in participants with rare type of PH	Baseline	To identify the major genetic alterations in participants with rare type of PH
Change in hemodynamics	up to 10 years, at 6 months interval
Change in cardiac function	up to 10 years, at 3-6 months interval	Measured by Cardiac MRI
Balloon pulmonary angioplasty (BPA)	up to 10 years, at 6 months interval	e.g. BPA versus non-BPA
Medical treatment	up to 10 years, at 6 months interval	e.g. mono- versus combination therapy

Trial Locations

Locations (1)

Chinese Academy of Medical Sciences Fuwai Hospital and Peking Union Medical College Hospital; 🇨🇳 Beijing, Beijing, China