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Risk and Resilience in Pulmonary Arterial Hypertension and Genetically Susceptible Individuals

Who can join

As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.

Age
15 Years to 80 Years
Sex
All sexes
Healthy volunteers
Not stated
Compensation
Not stated

Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.

What to expect

No one can promise a trial will help you personally — that is what the research is trying to find out.

What participation involves

  • Time periods mentioned: 4 years

Only what the study's summary and detailed description on ClinicalTrials.gov say. Time periods may describe the whole study or a part of it — ask the study team what applies to you.

Show the study's own words
  • "Aim 1 will identify static and dynamic molecular features of disease progression and resilience. 1a: Perform serial clinical, proteomic, and gene expression profiling in HPAH, IPAH, and healthy controls 3 times over 4 years."
Compensation
Not stated
  • Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
  • Participants are monitored closely by the research team throughout the study.
  • Some participants may receive access to an investigational treatment, which is still being studied.
  • Your participation helps researchers learn whether this approach could help future patients.

About this study

In plain language

This study looks at why some people develop pulmonary arterial hypertension (PAH), or high blood pressure in the lungs' arteries, while others do not. It studies people with PAH, healthy people with a certain gene change, and other healthy people to find clues that may help guide future treatments.

Pulmonary arterial hypertension (PAH) is a severe disease with a delayed diagnosis and markedly elevated mortality. High-risk populations, such as those with known genetic defects, provide a unique opportunity to determine the features of susceptibility and resilience to PAH. This proposal will fundamentally overturn the prevailing understanding of PAH by creating molecularly-driven signatures of susceptibility and resilience, provide novel insight into disease severity, and potentially identify new therapeutic targets. Funding Source - FDA OOPD

Compensation is read from the study's own description and may be missing — always confirm with the study team.

Eligibility

Who can join

  • Ages 15 to 80.
  • One study group has PAH with no known cause or PAH that runs in families, diagnosed using standard rules.
  • Another group is healthy with a known BMPR2 gene change. A heart ultrasound must show normal lung blood pressure and normal function on the heart's right side.
  • A third group is healthy with no heart or lung disease.
  • For people with PAH: WHO class I–III, meaning symptoms cause no, mild, or marked limits on physical activity.
  • PAH medicines must have been stable for 3 months before joining. One change in a water pill dose is allowed. Changes to IV prostacyclin, a medicine given through a vein, are allowed to manage side effects.

Who can't join

  • Cannot do normal activities because of being in a wheelchair or bed, needing a cane or walker, chest pain, arthritis, or another condition.
  • Pregnant.
  • PAH with a cause other than an unknown cause or an inherited cause.
  • Class IV heart failure, the most severe level.
  • More than 2 water pill dose changes in the past 3 months.

Simplified for readability. The official criteria below are what the study team uses.

Official eligibility criteria

Inclusion Criteria: * Children and Adults, aged 15 - 80 * Diagnosed with idiopathic or heritable, pulmonary arterial hypertension (PAH), defined according to standard criteria * Unaffected Mutation Carriers: Healthy participants with a known BMPR2 gene mutation and normal pulmonary pressure and RV function on echo * Healthy Controls: Healthy individuals without cardiopulmonary disease. * WHO functional class I-III * Stable PAH-specific medication regimen for three months prior to enrollment. Subjects with only a single diuretic adjustment in the prior three months will be included. Adjustments in IV prostacyclin for side effect management are allowed. Exclusion Criteria: * Prohibited from normal activity due to wheelchair bound status, bed bound status, reliance on a cane/walker, activity-limiting angina, activity-limiting osteoarthritis, or other condition that limits activity. * Pregnancy * Diagnosis of PAH etiology other than idiopathic, heritable * Functional class IV heart failure * Requirement of \> 2 diuretic adjustment in the prior three months.

Study sites (1)

TN