Risk and Resilience in Pulmonary Arterial Hypertension and Genetically Susceptible Individuals (NCT05584722) | Clinical Trial Compass
RecruitingNot Applicable
Risk and Resilience in Pulmonary Arterial Hypertension and Genetically Susceptible Individuals
United States150 participantsStarted 2022-11-01
Plain-language summary
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
Who can participate
Age range
15 Years – 80 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Change in Quality of Life as measured by the emPHasis-10
Timeframe: Baseline to 32 months
2
Change in Quality of Life as measured by the Pediatric Quality of Life Inventory Version 4.0
Timeframe: Baseline to 32 months
3
Change in meters walked in six-minute walk distance (meters)
Timeframe: Baseline to 32 months
4
Change in diffusing capacity for carbon monoxide (DLCO) on the Pulmonary Function Test
Timeframe: Baseline to 32 months
5
Change in Tricuspid Annular Plane Systolic Excursion (TAPSE), expressed in mm.
Timeframe: Baseline to 32 months
6
Change in Tricuspid Annular Velocity (S'), as assessed by echocardiogram results, expressed in cm/sec
Timeframe: Baseline to 32 months
7
Change in Estimated Right Ventricle (RV) Systolic Pressure, as assessed by echocardiogram results, expressed in mmHg
Change in Right Ventricle (RV) Free Wall Longitudinal Strain, as assessed by echocardiogram results, and expressed as percent (%) change in myocardial deformation.
Timeframe: Baseline to 32 months
9
Change in Daily Step Count as measured by the mHealth device mean daily step count
Timeframe: Baseline to 32 months
10
Change in Resting Heart Rate (beats per minute)
Timeframe: Baseline to 32 months
11
Intensity of activity
Timeframe: Baseline to 32 months
12
Number of patients with a PAH-related Hospitalization Incidence
Timeframe: Baseline to 32 months
13
Change in Patient Medication Regimen, as measured by percentage (%) of subjects with a change in medication regimen
Timeframe: Baseline to 32 months
14
Number of patients with an incidence of death
Timeframe: Baseline to 32 months
15
Number of patients with an incidence of lung transplantation