Idiopathic Pulmonary Arterial HypertensionRARE-PAHNCT05584722
Risk and Resilience in Pulmonary Arterial Hypertension and Genetically Susceptible Individuals
An observational study of Idiopathic Pulmonary Arterial Hypertension and Heritable Pulmonary Arterial Hypertension, run by Vanderbilt University Medical Center at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 15 to 80 years
- Conditions the study is for
- Idiopathic Pulmonary Arterial Hypertension, Heritable Pulmonary Arterial Hypertension, Unaffected Mutation Carriers: Healthy Participants With a Known BMPR2 Gene Mutation and Normal Pulmonary Pressure and RV Function on Echo
- How many people
- 150 participants
- Where
- 1 siteUnited States
- Study dates
- 1 Nov 2022to 31 Aug 2026
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 15 to 80 years
- All sexes
- Child, Adult, Older adult
Study population: * 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 may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Vanderbilt University Medical Center
Nashville, Tennessee, 37232
Kelly Burke, RN, kelly.burke@vumc.org
About this study
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
Read the detailed description
Pulmonary arterial hypertension (PAH) is an orphan disease with a delayed diagnosis and markedly elevated mortality from right heart failure. Despite nearly a dozen FDA-approved drugs for PAH, median survival is only seven years. All approved therapies target one of three vasodilatory pathways, and none are disease modifying. This study has two objectives: 1) Understand dynamic and static relationships between molecular markers and PAH progression and resilience; 2) Identify molecular features of PAH risk and resilience in individuals harboring a PAH-causing mutation. It is unknown why some at risk individuals develop PAH and others do not. BMPR2 mutations are present in about 30% of patients with PAH but clinical penetrance is only 20%. Unaffected BMPR2 mutation carriers (UMCs) are a unique and understudied population that may also provide clues to disease trajectory in patients with clinical PAH. Longitudinal natural history studies with molecular profiling in PAH are lacking. Most molecular profiling studies in PAH are cross-sectional which limits understanding of how disease progression and disease markers relate over time. The Investigators propose a strategy of dense clinical and molecular phenotyping at multiple timepoints to overcome inferential limitations of cross-sectional studies. This application will leverage the clinical and research infrastructure built at Vanderbilt over the past 35 years in our study of PAH patients. The investigators share an extensive published record of recruiting patients with this rare disease and related UMCs. The Investigators hypothesize that a comprehensive understanding of risk and resilience over time in patients and genetically susceptible individuals will provide insight into disease severity and identify novel therapeutic targets in patients with PAH. 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. Bioinformatic and network medicine analyses will identify proteins and RNAs associated with changes in clinical outcomes, functional capacity, and RV function in the parent cohort and two external validation cohorts.1b: Test whether adding molecular risk/resilience markers will improve the performance of a widely used PAH risk prediction tool (REVEAL 2.0 Risk Score). Aim 2 will identify the clinical and molecular factors that promote resilience and susceptibility to PAH in a longitudinal cohort of UMCs. UMCs will undergo serial clinical and molecular phenotyping as in Aim 1. Proteins/genes that mirror PAH are "risk factors" and those that mirror a healthy population are "resilience factors". Explanatory models will be developed and tested in validation cohorts. The Investigators will test UMC risk and resilience features for associations with clinical outcomes in PAH patients and risk prediction performance. These studies will identify signatures of risk and resilience to PAH progression and penetrance, offering an initial step toward personalizing care and surveillance guided by biologic data.
- Conditions
- Idiopathic Pulmonary Arterial Hypertension, Heritable Pulmonary Arterial Hypertension, Unaffected Mutation Carriers: Healthy Participants With a Known BMPR2 Gene Mutation and Normal Pulmonary Pressure and RV Function on Echo, Healthy Individuals With no Cardiopulmonary Disease
- Keywords
- pulmonary hypertension
- Medical subject headings
- Familial Primary Pulmonary Hypertension, Hypertension, Pulmonary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Idiopathic or Heritable Pulmonary Arterial Hypertension | Patients diagnosed with pulmonary arterial hypertension, either idiopathic or heritable, 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 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Quality of Life as measured by the emPHasis-10
The emPHasis-10 is a short and easy questionnaire that consists of 10 items that address breathlessness, fatigue, control, and confidence. Each item is scored on a semantic differential six-point scale (0-5), with contrasting adjectives at each end. A total emPHasis-10 score is derived using simple aggregation of the 10 items. emPHasis-10 scores range from 0 to 50, higher scores indicate worse quality of life.
Measured: Baseline to 32 months
Change in Quality of Life as measured by the Pediatric Quality of Life Inventory Version 4.0
The PedsQL (Pediatric Quality of Life Inventory) is a modular instrument for measuring health-related quality of life in children and adolescents ages 2 to 18. The PedsQL 4.0 Generic Core Scales are multidimensional child self-report and parent proxy-report scales developed as the generic core measure that consists of 23 items applicable for healthy school and community populations, as well as pediatric populations with acute and chronic health conditions.
Measured: Baseline to 32 months
Change in meters walked in six-minute walk distance (meters)
The 6MWT measures the distance (in meters), a participant can walk at a comfortable speed on a flat, hard surface in 6 minutes. The intent of the 6MWD test is to evaluate exercise capacity associated with carrying out daily physical activities.
Measured: Baseline to 32 months
Change in diffusing capacity for carbon monoxide (DLCO) on the Pulmonary Function Test
Pulmonary function tests (PFTs) are noninvasive tests that show how well the lungs are working. The tests measure lung volume, capacity, rates of flow, and gas exchange.
DLCO measures the quantity of carbon monoxide (CO) transferred per minute from alveolar gas to red blood cells in pulmonary capillaries and is expressed as mL/min/mm Hg.
Measured: Baseline to 32 months
Change in Tricuspid Annular Plane Systolic Excursion (TAPSE), expressed in mm.
Change from baseline in Tricuspid Annular Plane systolic Excursion (TAPSE) expressed in mm on echocardiogram results at 12-16 months and 24-32 months.
Measured: Baseline to 32 months
Change in Tricuspid Annular Velocity (S'), as assessed by echocardiogram results, expressed in cm/sec
Change from baseline in Tricuspid Annular Velocity (S') on echocardiogram results at 12-16 months and 24-32 months and expressed in cm/sec.
Measured: Baseline to 32 months
Change in Estimated Right Ventricle (RV) Systolic Pressure, as assessed by echocardiogram results, expressed in mmHg
Change from baseline in the estimated right ventricular systolic pressure on echocardiogram results at 12-16 months and 24-32 months and expressed in mmHg.
Measured: Baseline to 32 months
Change in Right Ventricle (RV) Free Wall Longitudinal Strain, as assessed by echocardiogram results, and expressed as percent (%) change in myocardial deformation.
Change from baseline in right ventricle free wall longitudinal strain on echocardiogram results at 12-16 months and 24-32 months and expressed as percent (%) change in myocardial deformation.
Measured: Baseline to 32 months
Change in Daily Step Count as measured by the mHealth device mean daily step count
Change from the baseline activity monitoring period to the 12-16 month activity monitoring period to the 24-32 month activity monitoring period. Data obtained by the mHealth device.
Measured: Baseline to 32 months
Change in Resting Heart Rate (beats per minute)
Monitored regularly using activity tracking device (per second when active, per 5 seconds when inactive). Subject's resting and peak exercise heart rate will also be recorded at baseline, 12-16 months, and 24-32 months. Targets exercise capacity. Heart rate is expressed as beats per minute.
Measured: Baseline to 32 months
Intensity of activity
Intensity of activity is categorized as rest, light, moderate, and vigorous based on calculated metabolic equivalents.
Measured: Baseline to 32 months
Number of patients with a PAH-related Hospitalization Incidence
To assess PAH-related hospitalization incidences from baseline to 12-16 months and 24-32 months. Number of patients will be assessed.
Measured: Baseline to 32 months
Change in Patient Medication Regimen, as measured by percentage (%) of subjects with a change in medication regimen
Change from baseline in patient medication regimen from baseline to 12-16 months and 24-32 months as measured by percentage (%) of subjects with a change in medication regimen.
Measured: Baseline to 32 months
Number of patients with an incidence of death
To assess incidences of death from baseline to 12-16 months and 24-32 months. Number of patients will be assessed.
Measured: Baseline to 32 months
Number of patients with an incidence of lung transplantation
To assess incidences of lung transplantation from baseline to 12-16 months and 24-32 months. Number of patients will be assessed.
Measured: Baseline to 32 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Oct 2022
- Study start1 Nov 2022
- Primary completion31 Aug 2026 (estimated)
- Study completion31 Aug 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Idiopathic Pulmonary Arterial HypertensionStudies of the same condition, open and under way.
- More trials run by Vanderbilt University Medical CenterThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 150 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Vanderbilt University Medical Center
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Anna Hemnes, Vanderbilt University Medical Center
- Investigators
- Evan Brittain, MD, Principal investigator, Vanderbilt Medical CenterAnna Hemnes, MD, Principal investigator, Vanderbilt Medical CenterEric Austin, MD, Principal investigator, Vanderbilt Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05584722
- Sponsor's protocol ID
- 221437
- Other IDs
- 1R01FD007627
- Acronym
- RARE-PAH
- Status last verified
- Mar 2026
- First submitted
- 14 Oct 2022
- Last updated
- 10 Mar 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 10 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Risk and Resilience in Pulmonary Arterial Hypertension and Genetically Susceptible Individuals
- ClinicalTrials.gov ID
- NCT05584722
- Acronym / sponsor ID
- RARE-PAH / 221437
- Conditions
- Idiopathic Pulmonary Arterial Hypertension, Heritable Pulmonary Arterial Hypertension, Unaffected Mutation Carriers: Healthy Participants With a Known BMPR2 Gene Mutation and Normal Pulmonary Pressure and RV Function on Echo, Healthy Individuals With no Cardiopulmonary Disease
- Keywords
- pulmonary hypertension
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 150 participants (estimated)
- Lead sponsor
- Vanderbilt University Medical CenterUniversity, hospital or other
- Study start
- 1 Nov 2022 (actual)
- Primary completion
- 31 Aug 2026 (estimated)
- Study completion
- 31 Aug 2026 (estimated)
- First posted
- 18 Oct 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Detailed description
Pulmonary arterial hypertension (PAH) is an orphan disease with a delayed diagnosis and markedly elevated mortality from right heart failure. Despite nearly a dozen FDA-approved drugs for PAH, median survival is only seven years. All approved therapies target one of three vasodilatory pathways, and none are disease modifying. This study has two objectives: 1) Understand dynamic and static relationships between molecular markers and PAH progression and resilience; 2) Identify molecular features of PAH risk and resilience in individuals harboring a PAH-causing mutation. It is unknown why some at risk individuals develop PAH and others do not. BMPR2 mutations are present in about 30% of patients with PAH but clinical penetrance is only 20%. Unaffected BMPR2 mutation carriers (UMCs) are a unique and understudied population that may also provide clues to disease trajectory in patients with clinical PAH. Longitudinal natural history studies with molecular profiling in PAH are lacking. Most molecular profiling studies in PAH are cross-sectional which limits understanding of how disease progression and disease markers relate over time. The Investigators propose a strategy of dense clinical and molecular phenotyping at multiple timepoints to overcome inferential limitations of cross-sectional studies. This application will leverage the clinical and research infrastructure built at Vanderbilt over the past 35 years in our study of PAH patients. The investigators share an extensive published record of recruiting patients with this rare disease and related UMCs. The Investigators hypothesize that a comprehensive understanding of risk and resilience over time in patients and genetically susceptible individuals will provide insight into disease severity and identify novel therapeutic targets in patients with PAH. 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. Bioinformatic and network medicine analyses will identify proteins and RNAs associated with changes in clinical outcomes, functional capacity, and RV function in the parent cohort and two external validation cohorts.1b: Test whether adding molecular risk/resilience markers will improve the performance of a widely used PAH risk prediction tool (REVEAL 2.0 Risk Score). Aim 2 will identify the clinical and molecular factors that promote resilience and susceptibility to PAH in a longitudinal cohort of UMCs. UMCs will undergo serial clinical and molecular phenotyping as in Aim 1. Proteins/genes that mirror PAH are "risk factors" and those that mirror a healthy population are "resilience factors". Explanatory models will be developed and tested in validation cohorts. The Investigators will test UMC risk and resilience features for associations with clinical outcomes in PAH patients and risk prediction performance. These studies will identify signatures of risk and resilience to PAH progression and penetrance, offering an initial step toward personalizing care and surveillance guided by biologic data.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Idiopathic or Heritable Pulmonary Arterial Hypertension | – | –Patients diagnosed with pulmonary arterial hypertension, either idiopathic or heritable, 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 may qualify
- Ages
- 15 to 80 years
- Sex
- All
- Age groups
- Child, Adult, Older adult
- Study population
- * 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.
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (5 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Change in Quality of Life as measured by the emPHasis-10
Time frame: Baseline to 32 months
The emPHasis-10 is a short and easy questionnaire that consists of 10 items that address breathlessness, fatigue, control, and confidence. Each item is scored on a semantic differential six-point scale (0-5), with contrasting adjectives at each end. A total emPHasis-10 score is derived using simple aggregation of the 10 items. emPHasis-10 scores range from 0 to 50, higher scores indicate worse quality of life.
Change in Quality of Life as measured by the Pediatric Quality of Life Inventory Version 4.0
Time frame: Baseline to 32 months
The PedsQL (Pediatric Quality of Life Inventory) is a modular instrument for measuring health-related quality of life in children and adolescents ages 2 to 18. The PedsQL 4.0 Generic Core Scales are multidimensional child self-report and parent proxy-report scales developed as the generic core measure that consists of 23 items applicable for healthy school and community populations, as well as pediatric populations with acute and chronic health conditions.
Change in meters walked in six-minute walk distance (meters)
Time frame: Baseline to 32 months
The 6MWT measures the distance (in meters), a participant can walk at a comfortable speed on a flat, hard surface in 6 minutes. The intent of the 6MWD test is to evaluate exercise capacity associated with carrying out daily physical activities.
Change in diffusing capacity for carbon monoxide (DLCO) on the Pulmonary Function Test
Time frame: Baseline to 32 months
Pulmonary function tests (PFTs) are noninvasive tests that show how well the lungs are working. The tests measure lung volume, capacity, rates of flow, and gas exchange. DLCO measures the quantity of carbon monoxide (CO) transferred per minute from alveolar gas to red blood cells in pulmonary capillaries and is expressed as mL/min/mm Hg.
Change in Tricuspid Annular Plane Systolic Excursion (TAPSE), expressed in mm.
Time frame: Baseline to 32 months
Change from baseline in Tricuspid Annular Plane systolic Excursion (TAPSE) expressed in mm on echocardiogram results at 12-16 months and 24-32 months.
Change in Tricuspid Annular Velocity (S'), as assessed by echocardiogram results, expressed in cm/sec
Time frame: Baseline to 32 months
Change from baseline in Tricuspid Annular Velocity (S') on echocardiogram results at 12-16 months and 24-32 months and expressed in cm/sec.
Change in Estimated Right Ventricle (RV) Systolic Pressure, as assessed by echocardiogram results, expressed in mmHg
Time frame: Baseline to 32 months
Change from baseline in the estimated right ventricular systolic pressure on echocardiogram results at 12-16 months and 24-32 months and 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.
Time frame: Baseline to 32 months
Change from baseline in right ventricle free wall longitudinal strain on echocardiogram results at 12-16 months and 24-32 months and expressed as percent (%) change in myocardial deformation.
Change in Daily Step Count as measured by the mHealth device mean daily step count
Time frame: Baseline to 32 months
Change from the baseline activity monitoring period to the 12-16 month activity monitoring period to the 24-32 month activity monitoring period. Data obtained by the mHealth device.
Change in Resting Heart Rate (beats per minute)
Time frame: Baseline to 32 months
Monitored regularly using activity tracking device (per second when active, per 5 seconds when inactive). Subject's resting and peak exercise heart rate will also be recorded at baseline, 12-16 months, and 24-32 months. Targets exercise capacity. Heart rate is expressed as beats per minute.
Intensity of activity
Time frame: Baseline to 32 months
Intensity of activity is categorized as rest, light, moderate, and vigorous based on calculated metabolic equivalents.
Number of patients with a PAH-related Hospitalization Incidence
Time frame: Baseline to 32 months
To assess PAH-related hospitalization incidences from baseline to 12-16 months and 24-32 months. Number of patients will be assessed.
Change in Patient Medication Regimen, as measured by percentage (%) of subjects with a change in medication regimen
Time frame: Baseline to 32 months
Change from baseline in patient medication regimen from baseline to 12-16 months and 24-32 months as measured by percentage (%) of subjects with a change in medication regimen.
Number of patients with an incidence of death
Time frame: Baseline to 32 months
To assess incidences of death from baseline to 12-16 months and 24-32 months. Number of patients will be assessed.
Number of patients with an incidence of lung transplantation
Time frame: Baseline to 32 months
To assess incidences of lung transplantation from baseline to 12-16 months and 24-32 months. Number of patients will be assessed.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Mar 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Evan Brittain, MD principal investigator
Vanderbilt Medical Center
Anna Hemnes, MD principal investigator
Vanderbilt Medical Center
Eric Austin, MD principal investigator
Vanderbilt Medical Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Idiopathic Pulmonary Arterial Hypertension and Heritable Pulmonary Arterial Hypertension, run by Vanderbilt University Medical Center at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 10 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Kelly Burke, RN ((615) 343-4682, kelly.burke@vumc.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in Quality of Life as measured by the emPHasis-10 (time frame: Baseline to 32 months). The study lists 15 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Mar 2026.