Hormonal, Metabolic, and Signaling Interactions in PAH

  • End date
    Jul 21, 2032
  • participants needed
  • sponsor
    Vanderbilt University Medical Center
Updated on 4 October 2022
Accepts healthy volunteers


Our hypothesis is that optimal treatment of the dysfunctional metabolic pathways which underlie PAH will improve pulmonary vascular function and consequences of the disease.


Project 1: This project will work to understand why women are affected by pulmonary arterial hypertension (PAH) so much more often than men. This observation is true in heritable, idiopathic and associated forms of PAH. While males and females have some similar hormone levels, certain hormones exist at higher levels in each gender. For example, estrogen levels are much higher in females, and thus seemed the most sensible place to start looking for differences that may be affecting disease. In a small, early study of our heritable patients, we found differences in how patients break down estrogens as compared to healthy control subjects. Now, we want to confirm that what we found is true in a much larger group of patients that includes idiopathic and associated forms of PAH. We will also look to see if testosterone and other androgenic hormones are somehow protective for males. If the observation holds true in the larger group of patients, then we may try to "fix" the hormone imbalance in a mouse model of PAH with a drug therapy, and see if it helps improve the mouse pulmonary hypertension without bad side effects to the animals. If the animal drug studies work, then we may be able to try this drug in patients to see if it will work as a human treatment.

Project 2: Despite major advances in understanding PAH in recent decades, safe, effective and tolerable therapies remain elusive. The metabolic syndrome (central obesity, insulin resistance, high blood pressure and hyperlipidemia-fats in the blood) has been implicated in PAH. Treating the downstream consequences of insulin resistance in the pulmonary vasculature is a new approach to effective intervention against this highly mortal disease. This project will study the role of insulin resistance in pulmonary arterial hypertension and determine if therapies to treat insulin resistance will improve pulmonary arterial hypertension.

Project 3: In Project 3, we are working on the theory that PAH can be treated by fixing cell-cell junctions in blood vessels with a drug called recombinant ACE2(angiotensin converting enzyme 2). This is the only approach so far that has worked to reverse disease in mouse models of heritable PAH, but we need to better understand how it is working and make sure it has long term safety in animal models before starting human trials, hopefully within a few years. Definition: Cell-cell junctions-all of our organs and body structures are made from cells. Normally, these cells (think of a balloon filled with water) line up right next to each other so that the cell membranes touch each other. Materials can flow from one cell to the next. In PAH patients it is believed that the cells in the linings of the small arteries are not able to line up together as they should.

Condition Idiopathic Pulmonary Arterial Hypertension, Heritable Pulmonary Arterial Hypertension, Scleroderma Associated Pulmonary Arterial Hypertension, Appetite Suppressant Associate PAH
Clinical Study IdentifierNCT01884051
SponsorVanderbilt University Medical Center
Last Modified on4 October 2022


Yes No Not Sure

Inclusion Criteria

Project 1
Diagnosis of IPAH (idiopathic pulmonary arterial hypertension), HPAH (heritable pulmonary arterial hypertension), or APAH (associated pulmonary arterial hypertension), family members of affected persons
Age 0-90, age 12-90 for skin biopsy
Other diagnosis
Age greater than 90, age less than 12 or greater than 90 for skin biopsy
Project 2
Diagnosis of IPAH, HPAH, or APAH, family members of affected persons
Subjects with reasonably easy access to clinic for blood collection and other testing
Subject able to tolerate fasting state prior to sample collection and EndoPAT (endothelial function assessment) testing
Other diagnosis
Subjects with difficulty reaching clinic for blood collection and other testing
Subjects unable to tolerate fasting state
Project 3
Diagnosis of IPAH, HPAH, or APAH, family members of affected persons
Other diagnosis
Age less than 7 or greater than 90

Exclusion Criteria

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