Understanding risk and resilience in pulmonary arterial hypertension

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This observational study aims to explore the molecular markers associated with pulmonary arterial hypertension (PAH) and identify why some individuals with genetic mutations develop the disease while others do not. By focusing on high-risk populations, including those with known BMPR2 gene mutations and healthy controls, the study will conduct longitudinal assessments to gather dynamic data on disease progression and resilience. The goal is to create molecular signatures that could lead to new therapeutic targets and improve understanding of PAH severity.

Why it matters

Who can join: the full rules

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.

Where it's running

• Vanderbilt University Medical Center
  Nashville, Tennessee, United States Recruiting

Study contacts

• Principal investigator: Evan Brittain, MD — Vanderbilt Medical Center
• Study coordinator: Kelly Burke, RN
• Email: kelly.burke@vumc.org
• Phone: (615) 343-4682

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Investigating Lipid Accumulation in the Right Ventricle of Patients with Pulmonary Arterial Hypertension

  This study is trying to see how fat buildup in the right side of the heart affects the health and exercise ability of people with pulmonary arterial hypertension.

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• Understanding hormonal and metabolic factors in pulmonary arterial hypertension

  This study is trying to see how hormones like estrogen and testosterone, along with metabolic factors, affect pulmonary arterial hypertension (PAH) differently in men and women to help improve treatment options.

  Recruiting Vanderbilt University Medical Center · Nashville, Tennessee · NCT01884051

• Using 129Xe MRI to Diagnose and Monitor Pulmonary Arterial Hypertension

  This study is testing if a special type of MRI can help doctors spot different types of Pulmonary Arterial Hypertension and track how the disease changes over time in people with this condition.

  Recruiting Phase 2 Duke University · Durham, North Carolina · NCT06104228

• Understanding how low-load leg exercises affect muscle and blood flow in PAH patients

  This substudy tests whether a gentle leg exercise program can change blood flow and oxygen use in the calf muscles of adults with pulmonary arterial hypertension.

  Recruiting University of Pennsylvania · Philadelphia, Pennsylvania · NCT07781709

• Sotatercept in Japanese children with pulmonary arterial hypertension

  This study tests whether sotatercept is safe and well tolerated in Japanese children with PAH when added to standard PAH treatment.

  Recruiting at 3 sites Phase 2 Merck Sharp & Dohme LLC · Izumi, Osaka and 2 other locations · NCT07646860

• DIAG723 in Adults With HHT

  A first-in-human study tests DIAG723 versus placebo in adults with hereditary hemorrhagic telangiectasia to see if it is safe and possibly helpful.

  Recruiting at 3 sites Phase 1/2 Diagonal Therapeutics, Inc. · Camperdown, New South Wales and 10 other locations · NCT07623525

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.