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The goal of this crossover trial is to determine whether the study drug dehydroepiandrosterone (DHEA) improves right ventricular longitudinal strain measured by cardiac magnetic resonance imaging at 18 weeks AND 40 weeks compared to placebo and to assess side effects and safety in pulmonary arterial hypertension.
Pulmonary hypertension (PH) is a heterogenous clinical disease characterized foremost by an abnormal increase in pulmonary artery pressure. Pulmonary vasculopathy, characterized by pathologic remodeling and vasoconstriction of the pulmonary arterioles, results in progressive dyspnea, exercise intolerance, right ventricular (RV) failure, and death. Female sex is the strongest clinical risk factor for PAH, with a 4:1 female-to-male ratio reported from the largest registry. Despite the increased risk of PAH in women, women with PAH have better survival than men. RV function is an important cause of morbidity and mortality in PAH as well as highly prevalent heart and lung diseases, but determinants of the RV response are entirely unknown. We and others have shown that female sex is associated with better RV systolic function in both health and disease, including PAH and left heart failure. Targeted PAH therapy leads to greater improvements in RVEF (demonstrated after just several months of treatment) in women as compared to men and partially explains better outcomes in women. Demonstration that DHEA has direct RV and sex-based effects will support the hypothesis that sex hormones play an important role in disease pathogenesis and provide insight into sex hormone manipulation as a treatment strategy in PAH.
The goal of this crossover trial is to correlate sex and sex hormones (particularly DHEA) to pulmonary vascular and RV phenotype differences in men and women with PAH. The study seeks to leverage a safe and available hormone treatment to gain further insight into 1) RV effects (a novel and critical end point in PH and PAH), 2) effects on two key PAH pathways in vivo and in vitro as a means for understanding sex-based differences in PAH, and 3) efficiency planning for a future Phase II parallel trial of DHEA as a novel treatment strategy in PAH.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| DHEA to placebo | Other | DHEA tablet (50 mg) taken by mouth once a day for 18 weeks, followed by 4 week washout period and then 1 placebo tablet taken by mouth once a day for 18 weeks |
|
| Placebo to DHEA | Other | 1 placebo tablet taken by mouth once a day for 18 weeks, followed by 4 week washout period and then 1 DHEA tablet taken by mouth once a day for 18 weeks |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| DHEA tablet | Drug | DHEA tablet (50 mg) taken by mouth once a day for 18 weeks. All participants in this crossover trial will receive DHEA during Treatment Period 1 or Treatment Period 2. There is a 4 week washout between Treatment Period 1 and Treatment Period 2 |
| Measure | Description | Time Frame |
|---|---|---|
| Change in Right Ventricular (RV) Longitudinal Strain, % Cardiac Magnetic Resonance Imaging (MRI) | Longitudinal strain is determined using standard cine imaging and Tissue Tracking (Strain) software (Tissue Tracking plugin, Circle Cardiovascular Imaging). Study MRIs were performed at a core MRI facility on a single Siemens 1.5T Aera with full Advanced Cardiac Package and XQ Gradients (45 mT/m @ 200 T/m/s) used for research grade imaging. 2D RV longitudinal strain: Measured from RV free wall of 4-chamber view cine CMR image. RV wall is divided into 6 equal segments. Strain can be either positive, which indicates lengthening, or negative, which indicates shortening. Normal circumferential and longitudinal strain are negative numbers, those for radial strain are positive. | Baseline to 18 weeks (treatment period 1), 18 weeks to 40 weeks (treatment period 2) |
| Change in Right Ventricular (RV) †RV Radial Strain, %, | †Short axis, RV radial strain, % by Cardiac Magnetic Resonance Imaging (MRI). Study MRIs were performed at a core MRI facility on a single Siemens 1.5T Aera with full Advanced Cardiac Package and XQ Gradients (45 mT/m @ 200 T/m/s) used for research grade imaging. Measurements were made using CVI42, Circle Cardiovascular Imaging. 2D RV longitudinal strain: Measured from RV free wall of 4-chamber view cine CMR image. RV wall is divided into 6 equal segments. Strain can be either positive, which indicates lengthening, or negative, which indicates shortening. Normal circumferential and longitudinal strain are negative numbers, those for radial strain are positive. | Baseline to 18 weeks (treatment period 1), 18 weeks to 40 weeks (treatment period 2) |
| †RV Circumferential Strain, % | Circumferential strain is determined using standard cine imaging and Tissue Tracking (Strain) software (Tissue Tracking plugin, Circle Cardiovascular Imaging). Cardiac Study MRIs were performed at a core MRI facility on a single Siemens 1.5T Aera with full Advanced Cardiac Package and XQ Gradients (45 mT/m @ 200 T/m/s) used for research grade imaging. Measurements were made using CVI42, Circle Cardiovascular Imaging. 2D RV circumferential strain: Measured from RV free wall of 4-chamber view cine CMR image. RV wall is divided into 6 equal segments. Strain can be either positive, which indicates lengthening, or negative, which indicates shortening. Normal circumferential and longitudinal strain are negative numbers, those for radial strain are positive. |
| Measure | Description | Time Frame |
|---|---|---|
| Change in Six Minute Walk Distance (6MWD) Between DHEA and Placebo | Change in 6MWD from baseline to 18 weeks (treatment period 1) and 18 week to 40 weeks (treatment period 2) in both groups. | Baseline to 18 weeks (treatment period 1), 18 weeks to 40 weeks (treatment period 2) |
| Change in World Health Organization (WHO) Functional Class |
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Inclusion Criteria:
Diagnosis of PAH that is 1) idiopathic, 2) heritable or 3) associated with connective tissue disease, congenital systemic-to-pulmonary shunt, porto-pulmonary hypertension, drug or toxin use.
Documentation of the following at any time prior to study entry:
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| Name | Affiliation | Role |
|---|---|---|
| Corey E Ventetuolo, MD, MS | Brown University | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Rhode Island Hospital Pulmonary Hypertension Center | Providence | Rhode Island | 02903 | United States |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 40766123 | Derived | Sanders R, Walsh T, Baird GL, Atalay MK, Agarwal S, Arcuri D, Klinger JR, Mullin CJ, Simmons J, Singh N, Whittenhall M, Ventetuolo CE. Effects of Dehydroepiandrosterone in Pulmonary Hypertension (EDIPHY): A Randomized, Double-Blind, Placebo-Controlled Crossover Trial. medRxiv [Preprint]. 2025 Aug 2:2025.08.01.25332627. doi: 10.1101/2025.08.01.25332627. |
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| ID | Title | Description |
|---|---|---|
| FG000 | DHEA to Placebo | DHEA tablet (50 mg) taken by mouth once a day for 18 weeks DHEA tablet: DHEA tablet (50 mg) taken by mouth once a day for 18 weeks. Following washout period of 4 weeks. Placebo (1 tablet once a day) for 18 weeks |
| FG001 | Placebo to DHEA | 1 placebo tablet taken by mouth once a day for 18 weeks Placebo: 1 placebo tablet taken by mouth once a day for 18 weeks DHEA tablet: DHEA tablet (50 mg) taken by mouth once a day for 18 weeks. |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | DHEA to placebo | DHEA tablet (50 mg) taken by mouth once a day for 18 weeks DHEA tablet: DHEA tablet (50 mg) taken by mouth once a day for 18 weeks. then Placebo: 1 placebo tablet taken by mouth once a day for 18 weeks |
| BG001 | Placebo to DHEA |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Change in Right Ventricular (RV) Longitudinal Strain, % Cardiac Magnetic Resonance Imaging (MRI) | Longitudinal strain is determined using standard cine imaging and Tissue Tracking (Strain) software (Tissue Tracking plugin, Circle Cardiovascular Imaging). Study MRIs were performed at a core MRI facility on a single Siemens 1.5T Aera with full Advanced Cardiac Package and XQ Gradients (45 mT/m @ 200 T/m/s) used for research grade imaging. 2D RV longitudinal strain: Measured from RV free wall of 4-chamber view cine CMR image. RV wall is divided into 6 equal segments. Strain can be either positive, which indicates lengthening, or negative, which indicates shortening. Normal circumferential and longitudinal strain are negative numbers, those for radial strain are positive. | All patients receive both DHEA and placebo during this crossover study. One group received DHEA first, followed by a washout period, then placebo. The second group received placebo first, followed by a washout period, then DHEA. | Posted | Median | 95% Confidence Interval | % Change in RV Longitudinal Strain | Baseline to 18 weeks (treatment period 1), 18 weeks to 40 weeks (treatment period 2) |
Adverse event data was collected at 2 weeks, 18 weeks, 19 weeks, 22 weeks, 24 weeks, 40 weeks and 42 weeks
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | DHEA | DHEA tablet (50 mg) taken by mouth once a day for 18 weeks DHEA tablet: DHEA tablet (50 mg) taken by mouth once a day for 18 weeks. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| 10016450 | Musculoskeletal and connective tissue disorders | MEDRA | Systematic Assessment | Femoral neck fracture |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| 10000147 | Skin and subcutaneous tissue disorders | MEDRA | Systematic Assessment | Hair growth on chin |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Corey Ventetuolo, MD | Brown University Health, Center for Advanced Lung Care | 401-444-2733 | corey_ventetuolo@brown.edu |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Aug 12, 2021 | Aug 27, 2025 | Prot_SAP_001.pdf |
| ICF | No | No | Yes | Informed Consent Form | Sep 20, 2023 | Aug 29, 2025 | ICF_002.pdf |
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| ID | Term |
|---|---|
| D000081029 | Pulmonary Arterial Hypertension |
| ID | Term |
|---|---|
| D006976 | Hypertension, Pulmonary |
| D008171 | Lung Diseases |
| D012140 | Respiratory Tract Diseases |
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| ID | Term |
|---|---|
| D003687 | Dehydroepiandrosterone |
| ID | Term |
|---|---|
| D000737 | Androstenols |
| D000736 | Androstenes |
| D000731 | Androstanes |
| D013256 | Steroids |
| D000072473 |
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|
| Placebo | Other | 1 placebo tablet taken by mouth once a day for 18 weeks. All participants in this crossover trial will receive placebo during this crossover study during Treatment Period 1 or Treatment Period 2. There is a 4 week washout between Treatment Period 1 and Treatment Period 2 |
|
| Baseline to 18 weeks (treatment period 1), 18 weeks to 40 weeks (treatment period 2) |
| RV End Diastolic Volume (RVEDV), mL | % Change in RV End Diastolic Volume (RVEDV), mL by Cardiac Magnetic Resonance Imaging (MRI), strain from baseline to 18 weeks (treatment period 1) and 18 week to 40 weeks (treatment period 2) in both groups: DHEA to Placebo (DHEA taken first, followed by washout and then Placebo) and Placebo to DHEA (placebo taken first, followed by washout and then DHEA). Short-axis steady state free precession (SSFP) imaging was obtained from the base of the heart through the apex. The endocardial and epicardial borders of both ventricles are be traced manually on short axis cine images at end-diastole and end-systole with exclusion of the papillary muscles and trabeculae. EDV and ESV are calculated using Simpson's rule by summation of areas on each slice multiplied by the sum of slice thickness and image gap. | 18 Weeks, 40 Weeks |
| Change in RV Ejection Fraction Measured by Cardiac MRI | Change in RV ejection fraction measured by Cardiac Magnetic Resonance Imaging (MRI), % Change in strain from baseline to 18 weeks (treatment period 1) and 18 week to 40 weeks (treatment period 2) in both groups: DHEA to Placebo (DHEA taken first, followed by washout and then Placebo) and Placebo to DHEA (placebo taken first, followed by washout and then DHEA). Short-axis steady state free precession (SSFP) imaging from the base of the heart through the apex. Ventricular EFs are calculated by dividing respective stroke volumes (EDV-ESV) by EDVs. | Baseline to 18 weeks (treatment period 1), 18 weeks to 40 weeks (treatment period 2) |
| RVESV, mL | Change in right ventricular end-systolic volume from baseline to 18 weeks (treatment period 1) and 18 week to 40 weeks (treatment period 2) in both groups: DHEA to Placebo (DHEA taken first, followed by washout and then Placebo) and Placebo to DHEA (placebo taken first, followed by washout and then DHEA). Short-axis steady state free precession (SSFP) imaging was obtained from the base of the heart through the apex. The endocardial and epicardial borders of both ventricles are be traced manually on short axis cine images at end-diastole and end-systole with exclusion of the papillary muscles and trabeculae. EDV and ESV are calculated using Simpson's rule by summation of areas on each slice multiplied by the sum of slice thickness and image gap. | Baseline to 18 weeks (treatment period 1), 18 weeks to 40 weeks (treatment period 2) |
| RV Stroke Volume, mL | Change in Right ventricular stroke volume by cardiac MRI from baseline to 18 weeks (treatment period 1) and 18 week to 40 weeks (treatment period 2) in both groups: DHEA to Placebo (DHEA taken first, followed by washout and then Placebo) and Placebo to DHEA (placebo taken first, followed by washout and then DHEA). Stroke volume is calculated through breath-hold through-plane phase contrast imaging with a velocity encoding gradient (VENC) of <120 cm/s (larger if aliasing present) in the main PA ~2-3 cm above the pulmonary valve plane, with imaging plane oriented orthogonal to the main PA. Free-breathing phase contrast imaging in the same plane and VENC with averaging over 4 respiratory cycles. | Baseline to 18 weeks (treatment period 1), 18 weeks to 40 weeks (treatment period 2) |
| RV Mass, g | Right ventricular mass, Change in RV mass from baseline to 18 weeks (treatment period 1) and 18 week to 40 weeks (treatment period 2) in both groups: DHEA to Placebo (DHEA taken first, followed by washout and then Placebo) and Placebo to DHEA (placebo taken first, followed by washout and then DHEA). Short-axis steady state free precession (SSFP) imaging from the base of the heart through the apex. RV mass is determined at the end-diastole phase as the difference between end-diastolic epicardial and endocardial volumes X heart specific gravity (1.05 g/cm3). | 18 weeks, 40 weeks |
Change in WHO Functional Class (I - IV with IV indicating worse symptoms) between DHEA and placebo. |
| Baseline to 18 weeks (treatment period 1), 18 weeks to 40 weeks (treatment period 2) |
| Change in Short Form-36 Summary Scores for Physical and Mental Components | Change in Short Form-36 summary scores for physical and mental components (range 0 - 100, higher scores indicating better quality of life). | Baseline to 18 weeks (treatment period 1), 18 weeks to 40 weeks (treatment period 2) |
| Change in emPHasis-10 | Change in emPHasis-10 score (range 0 - 50, higher scores indicating worse quality of life) between DHEA and placebo. | Baseline to 18 weeks (treatment period 1), 18 weeks to 40 weeks (treatment period 2) |
| Change in NT-proBNP Between DHEA and Placebo | Change in serum level of NT-proBNP between DHEA and placebo. | Baseline to 18 weeks (treatment period 1), 18 weeks to 40 weeks (treatment period 2) |
| Change in DHEA-S (ug/dL) | Change in DHEA-S from baseline to 18 weeks (treatment period 1) and 18 week to 40 weeks (treatment period 2) in both groups. | Baseline to 18 weeks (treatment period 1), 18 weeks to 40 weeks (treatment period 2) |
| Change in Estradiol, pg/mL | Change in Estradiol, pg/mL from baseline to 18 weeks (treatment period 1) and 18 week to 40 weeks (treatment period 2) in both groups. | Baseline to 18 weeks (treatment period 1), 18 weeks to 40 weeks (treatment period 2) |
| Change in Testosterone, ng/dL | Change in Testosterone, ng/dL from baseline to 18 weeks (treatment period 1) and 18 week to 40 weeks (treatment period 2) in both groups. | Baseline to 18 weeks (treatment period 1), 18 weeks to 40 weeks (treatment period 2) |
| Change in Progesterone, ng/mL | Change in Progesterone, ng/mL from baseline to 18 weeks (treatment period 1) and 18 week to 40 weeks (treatment period 2) in both groups. | Baseline to 18 weeks (treatment period 1), 18 weeks to 40 weeks (treatment period 2) |
| Change in Follicle Stimulating Hormone (FSH), mIU/mL | Change in FSH, mIU/mL from baseline to 18 weeks (treatment period 1) and 18 week to 40 weeks (treatment period 2) in both groups. | Baseline to 18 weeks (treatment period 1), 18 weeks to 40 weeks (treatment period 2) |
| Change in Sex Hormone Binding Globulin (SHBG), Nmol/L | Change in SHBG, nmol/L from baseline to 18 weeks (treatment period 1) and 18 week to 40 weeks (treatment period 2) in both groups. | Baseline to 18 weeks (treatment period 1), 18 weeks to 40 weeks (treatment period 2) |
| Change in Luteinizing Hormone, mIU/mL | Change in Luteinizing hormone, mIU/mL from baseline to 18 weeks (treatment period 1) and 18 week to 40 weeks (treatment period 2) in both groups. | Baseline to 18 weeks (treatment period 1), 18 weeks to 40 weeks (treatment period 2) |
| Change in Prolactin, μIU/mL | Change in Prolactin, μIU/mL from baseline to 18 weeks (treatment period 1) and 18 week to 40 weeks (treatment period 2) in both groups. | Baseline to 18 weeks (treatment period 1), 18 weeks to 40 weeks (treatment period 2) |
| Change in C-peptide, ng/mL | Change in C-peptide, ng/mL from baseline to 18 weeks (treatment period 1) and 18 week to 40 weeks (treatment period 2) in both groups. | Baseline to 18 weeks (treatment period 1), 18 weeks to 40 weeks (treatment period 2) |
| Change in Insulin, μU/mL | Change in Insulin, μU/mL from baseline to 18 weeks (treatment period 1) and 18 week to 40 weeks (treatment period 2) in both groups. | Baseline to 18 weeks (treatment period 1), 18 weeks to 40 weeks (treatment period 2) |
| Cortisol, μg/dL^2 | Change in Cortisol, μg/dL^2 (micrograms per deciliter squared) from baseline to 18 weeks (treatment period 1) and 18 week to 40 weeks (treatment period 2) in both groups. | Baseline to 18 weeks (treatment period 1), 18 weeks to 40 weeks (treatment period 2) |
| Left Ventricular Ejection Fraction, % | Change in left ventricular ejection fraction (%) measured by Cardiac Magnetic Resonance Imaging (MRI). Change in LVEG from baseline to 18 weeks (treatment period 1) and 18 week to 40 weeks (treatment period 2) in both groups: DHEA to Placebo (DHEA taken first, followed by washout and then Placebo) and Placebo to DHEA (placebo taken first, followed by washout and then DHEA). Short-axis steady state free precession (SSFP) imaging from the base of the heart through the apex was used.. Ventricular EFs are calculated by dividing respective stroke volumes (EDV-ESV) by EDVs. Normal Values: 56-78% for both males and females. | Baseline to 18 weeks (treatment period 1), 18 weeks to 40 weeks (treatment period 2) |
| Left Ventricular End-diastolic Volume (LVEDV) (mL) | Change in left ventricular LVEDV measured by Cardiac Magnetic Resonance Imaging (MRI). Change in LVEDV from baseline to 18 weeks (treatment period 1) and 18 week to 40 weeks (treatment period 2) in both groups: DHEA to Placebo (DHEA taken first, followed by washout and then Placebo) and Placebo to DHEA (placebo taken first, followed by washout and then DHEA). Short-axis steady state free precession (SSFP) imaging was obtained from the base of the heart through the apex. The endocardial and epicardial borders of both ventricles are be traced manually on short axis cine images at end-diastole and end-systole with exclusion of the papillary muscles and trabeculae. EDV and ESV are calculated using Simpson's rule by summation of areas on each slice multiplied by the sum of slice thickness and image gap. Normal values (Male):77-195 ml. Normal values (female):58-154 ml. | Baseline to 18 weeks (treatment period 1), 18 weeks to 40 weeks (treatment period 2) |
| Left Ventricular End-systolic Volume (LVESV) (mL) | Change in LVESV measured by Cardiac Magnetic Resonance Imaging (MRI). Change in LVEDV from baseline to 18 weeks (treatment period 1) and 18 week to 40 weeks (treatment period 2) in both groups: DHEA to Placebo (DHEA taken first, followed by washout and then Placebo) and Placebo to DHEA (placebo taken first, followed by washout and then DHEA). Short-axis steady state free precession (SSFP) imaging was obtained from the base of the heart through the apex. The endocardial and epicardial borders of both ventricles are be traced manually on short axis cine images at end-diastole and end-systole with exclusion of the papillary muscles and trabeculae. EDV and ESV are calculated using Simpson's rule by summation of areas on each slice multiplied by the sum of slice thickness and image gap. Normal Values (Female):13-51 ml. Normal values (Male):19-72 ml | Baseline to 18 weeks (treatment period 1), 18 weeks to 40 weeks (treatment period 2) |
| Left Ventricular Stroke Volume (mL) | Change in Left ventricular stroke volume measured by Cardiac Magnetic Resonance Imaging (MRI). Change in Left ventricular stroke volume from baseline to 18 weeks (treatment period 1) and 18 week to 40 weeks (treatment period 2) in both groups: DHEA to Placebo (DHEA taken first, followed by washout and then Placebo) and Placebo to DHEA (placebo taken first, followed by washout and then DHEA). Stroke volume is calculated through breath-hold through-plane phase contrast imaging with a velocity encoding gradient (VENC) of <120 cm/s (larger if aliasing present) in the main PA ~2-3 cm above the pulmonary valve plane, with imaging plane oriented orthogonal to the main PA. Free-breathing phase contrast imaging in the same plane and VENC with averaging over 4 respiratory cycles. | Baseline to 18 weeks (treatment period 1), 18 weeks to 40 weeks (treatment period 2) |
| Left Ventricular End Diastolic Mass, g | Change in Left ventricular end diastolic mass, g measured by Cardiac Magnetic Resonance Imaging (MRI). Change in Left ventricular end diastolic mass, g from baseline to 18 weeks (treatment period 1) and 18 week to 40 weeks (treatment period 2) in both groups: DHEA to Placebo (DHEA taken first, followed by washout and then Placebo) and Placebo to DHEA (placebo taken first, followed by washout and then DHEA). Short-axis steady state free precession (SSFP) imaging from the base of the heart through the apex. LV mass is determined at the end-diastole phase as the difference between end-diastolic epicardial and endocardial volumes X heart specific gravity (1.05 g/cm3). | Baseline to 18 weeks (treatment period 1), 18 weeks to 40 weeks (treatment period 2) |
| Treatment-related Side Effects and Adverse Events | Treatment-related side effects and adverse events (as assessed by CTCAE v4.0). All participants in this trial received both DHEA and Placebo during the trial, therefore, side-effects and adverse events are listed according to the treatment or washout period in which they occurred. | 18 weeks, 40 weeks |
1 placebo tablet taken by mouth once a day for 18 weeks Placebo: 1 placebo tablet taken by mouth once a day for 18 weeks then DHEA tablet: DHEA tablet (50 mg) taken by mouth once a day for 18 weeks. |
| BG002 | Total | Total of all reporting groups |
| Participants |
|
| Age, Continuous | Median | Inter-Quartile Range | years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
| BMI | Median | Inter-Quartile Range | kg/m2 |
|
| 6MWD | Median | Inter-Quartile Range | meters |
|
| Functional Class | Functional classification drives initial choice of therapy and is a major treatment goal in PAH. WHO class is derived from the New York Heart Association classification: Class I is no limitations; Class II is slight limitations in physical activity and no symptoms at rest; Class III is symptoms with minimal activity; Class IV is symptoms at rest. Clinicians will assess functional class at each study visit. | Count of Participants | Participants |
|
| PAH Diagnosis | Count of Participants | Participants |
|
| EmPHasis10 | The EmPHasis-10 score interpretation in pulmonary hypertension (PH) is a numerical value from 0 to 50, where a higher score indicates a poorer health status and quality of life. This score is generated from a 10-item questionnaire that assesses how PH affects a patient's life, and it serves as a tool for risk stratification and monitoring patient well-being, with improved scores correlating to better exercise capacity and survival outcomes. | Median | Inter-Quartile Range | units on a scale |
|
| Mean PAP | Hemodynamics from prior right and left heart catheterizations were reviewed for inclusion. Hemodynamic criteria were met within a single catheterization procedure at any time before study enrollment. | Median | Inter-Quartile Range | mmHg |
|
| N-terminal proBNP, pg/mL | Median | Inter-Quartile Range | pg/mL |
|
| Right atrial pressure, mmHg | Hemodynamics from prior right and left heart catheterizations were reviewed for inclusion. Hemodynamic criteria were met within a single catheterization procedure at any time before study enrollment. | Median | Inter-Quartile Range | mmHg |
|
| PAOP, mm Hg | Pulmonary artery occlusion pressure. Hemodynamics from prior right and left heart catheterizations were reviewed for inclusion. Hemodynamic criteria were met within a single catheterization procedure at any time before study enrollment. | Median | Inter-Quartile Range | mmHg |
|
| PVR, Wood units | Hemodynamics from prior right and left heart catheterizations were reviewed for inclusion. Hemodynamic criteria were met within a single catheterization procedure at any time before study enrollment. Pulmonary vascular resistance (PVR) in a hemodynamic measure that must be above 3 woods units to qualify subject for enrollment. | Median | Inter-Quartile Range | woods units |
|
| Cardiac Index L/min/m^2 | Hemodynamics from prior right and left heart catheterizations were reviewed for inclusion. Hemodynamic criteria were met within a single catheterization procedure at any time before study enrollment. | Median | Inter-Quartile Range | L/min/m^2 |
|
| Short-Form 36 PCS | The SF-36 measures health-related quality of life by assessing eight domains: physical functioning, role limitations (physical and emotional), bodily pain, general health, vitality, social functioning, and mental health. SF-36 PCS refers to the physical component. To interpret, scores above 50 indicate better-than-average health-related quality of life, and scores below 50 indicate below-average health. | Median | Inter-Quartile Range | units on a scale |
|
| Short-Form 36 MCS | The SF-36 measures health-related quality of life by assessing eight domains: physical functioning, role limitations (physical and emotional), bodily pain, general health, vitality, social functioning, and mental health. SF-36 MCS refers to mental component summary. Scores above 50 indicate better-than-average health-related quality of life, and scores below 50 indicate below-average health | Median | Inter-Quartile Range | units on a scale |
|
| RV Longitudinal Strain, % | Study MRIs were performed at a core MRI facility used for research grade imaging. Measurements were made using CVI42, Circle Cardiovascular Imaging. | Median | Inter-Quartile Range | % |
|
| RV peak global radial strain, % | RV peak global radial strain, %. Study MRIs were performed at a core MRI facility regularly used for research grade imaging. All MRI measurements were made using commercially available cardiac MRI software (CVI42, Circle Cardiovascular Imaging). | Median | Inter-Quartile Range | % |
|
| RV circumferential strain, % | Study MRIs were performed at a core MRI facility regularly used for research grade imaging. All MRI measurements were made using commercially available cardiac MRI software (CVI42, Circle Cardiovascular Imaging). Strain can be either positive, which indicates lengthening, or negative, indicating shortening. Values for normal circumferential strain are negative numbers. | Median | Inter-Quartile Range | % |
|
| †RV radial strain, % | Right ventricular radial strain. Study MRIs were performed at a core MRI facility on a single Siemens 1.5T Aera with full Advanced Cardiac Package and XQ Gradients (45 mT/m @ 200 T/m/s) regularly used for research grade imaging. All MRI measurements were made using commercially available cardiac MRI software (CVI42, Circle Cardiovascular Imaging). By convention strain can be either positive, which indicates lengthening, or negative, which indicates shortening. Values for normal circumferential and longitudinal strain are negative numbers, whereas those for radial strain are positive. | Median | Inter-Quartile Range | % |
|
| RVEF, % | Right ventricular ejection fraction. A normal RVEF is 40% to 50% or higher. Lower RVEF indicates right heart dysfunction. Values below 40-45% are often considered reduced, with RVEF below 20% having a particularly strong association with worse outcomes. Study MRIs were performed at a core MRI facility on a single Siemens 1.5T Aera with full Advanced Cardiac Package. All MRI measurements were made using commercially available cardiac MRI software (CVI42, Circle Cardiovascular Imaging). Normal Values (Female): RV EF%: 47-80%. Normal values (Male):RV EF%: 47-74% | Median | Inter-Quartile Range | % |
|
| RVEDV, mL | RVEDV (Right Ventricular End-Diastolic Volume) represents the volume of blood in the right ventricle at the end of its filling phase (end-diastole).Study MRIs were performed at a core MRI facility regularly used for research grade imaging. All MRI measurements were made using commercially available cardiac MRI software (CVI42, Circle Cardiovascular Imaging). Normal values (female) RVEDV: 58-154 ml (48-87 ml/m2), Normal values (Male) RV EDV: 88-227 ml (55-105 ml/m2) | Median | Inter-Quartile Range | mL |
|
| RVESV, mL | Right Ventricular End-Systolic Volume. Study MRIs were performed at a core MRI facility on a single Siemens 1.5T Aera with full Advanced Cardiac Package and XQ Gradients (45 mT/m @ 200 T/m/s) regularly used for research grade imaging. All MRI measurements were made using commercially available cardiac MRI software (CVI42, Circle Cardiovascular Imaging). Normal Values (Female) RV ESV: 12-68 ml (11.0-27.6 ml/m2). Normal value (Male): RV ESV: 23-103 ml (15.4-42.9 ml/m2) | Median | Inter-Quartile Range | mL |
|
| RV stroke volume, mL | Right ventricular stroke volume. Study MRIs were performed at a core MRI facility on a single Siemens 1.5T Aera with full Advanced Cardiac Package and XQ Gradients (45 mT/m @ 200 T/m/s) regularly used for research grade imaging. All MRI measurements were made using commercially available cardiac MRI software (CVI42, Circle Cardiovascular Imaging). | Median | Inter-Quartile Range | mL |
|
| RV mass, g | Right ventricular mass. Study MRIs were performed at a core MRI facility on a single Siemens 1.5T Aera with full Advanced Cardiac Package and XQ Gradients (45 mT/m @ 200 T/m/s) regularly used for research grade imaging. All MRI measurements were made using commercially available cardiac MRI software (CVI42, Circle Cardiovascular Imaging). | Median | Inter-Quartile Range | grams |
|
| ID |
|---|
| Title |
|---|
| Description |
|---|
| OG000 | DHEA to Placebo, Baseline | DHEA to Placebo group, Baseline |
| OG001 | Placebo to DHEA, Baseline | Placebo to DHEA group, Baseline |
| OG002 | DHEA to placebo, 18 Weeks | Treatment period 1: DHEA to Placebo group, measurement of % change at 18 Weeks |
| OG003 | Placebo to DHEA, 18 Weeks | Treatment period 1: Placebo to DHEA group, % change at 18 Weeks |
| OG004 | DHEA to Placebo, 40 Weeks | Treatment period 2: DHEA to Placebo group, % change between 18 and 40 Weeks |
| OG005 | Placebo to DHEA, 40 weeks | Treatment period 2: Placebo to DHEA group, % change between 18 and 40 Weeks |
|
|
|
| Primary | Change in Right Ventricular (RV) †RV Radial Strain, %, | †Short axis, RV radial strain, % by Cardiac Magnetic Resonance Imaging (MRI). Study MRIs were performed at a core MRI facility on a single Siemens 1.5T Aera with full Advanced Cardiac Package and XQ Gradients (45 mT/m @ 200 T/m/s) used for research grade imaging. Measurements were made using CVI42, Circle Cardiovascular Imaging. 2D RV longitudinal strain: Measured from RV free wall of 4-chamber view cine CMR image. RV wall is divided into 6 equal segments. Strain can be either positive, which indicates lengthening, or negative, which indicates shortening. Normal circumferential and longitudinal strain are negative numbers, those for radial strain are positive. | Posted | Median | 95% Confidence Interval | % Change in RV radial strain | Baseline to 18 weeks (treatment period 1), 18 weeks to 40 weeks (treatment period 2) |
|
|
|
|
| Primary | †RV Circumferential Strain, % | Circumferential strain is determined using standard cine imaging and Tissue Tracking (Strain) software (Tissue Tracking plugin, Circle Cardiovascular Imaging). Cardiac Study MRIs were performed at a core MRI facility on a single Siemens 1.5T Aera with full Advanced Cardiac Package and XQ Gradients (45 mT/m @ 200 T/m/s) used for research grade imaging. Measurements were made using CVI42, Circle Cardiovascular Imaging. 2D RV circumferential strain: Measured from RV free wall of 4-chamber view cine CMR image. RV wall is divided into 6 equal segments. Strain can be either positive, which indicates lengthening, or negative, which indicates shortening. Normal circumferential and longitudinal strain are negative numbers, those for radial strain are positive. | Posted | Median | 95% Confidence Interval | % Change in RV circumferential strain | Baseline to 18 weeks (treatment period 1), 18 weeks to 40 weeks (treatment period 2) |
|
|
|
|
| Primary | RV End Diastolic Volume (RVEDV), mL | % Change in RV End Diastolic Volume (RVEDV), mL by Cardiac Magnetic Resonance Imaging (MRI), strain from baseline to 18 weeks (treatment period 1) and 18 week to 40 weeks (treatment period 2) in both groups: DHEA to Placebo (DHEA taken first, followed by washout and then Placebo) and Placebo to DHEA (placebo taken first, followed by washout and then DHEA). Short-axis steady state free precession (SSFP) imaging was obtained from the base of the heart through the apex. The endocardial and epicardial borders of both ventricles are be traced manually on short axis cine images at end-diastole and end-systole with exclusion of the papillary muscles and trabeculae. EDV and ESV are calculated using Simpson's rule by summation of areas on each slice multiplied by the sum of slice thickness and image gap. | Posted | Median | 95% Confidence Interval | mL | 18 Weeks, 40 Weeks |
|
|
|
|
| Primary | Change in RV Ejection Fraction Measured by Cardiac MRI | Change in RV ejection fraction measured by Cardiac Magnetic Resonance Imaging (MRI), % Change in strain from baseline to 18 weeks (treatment period 1) and 18 week to 40 weeks (treatment period 2) in both groups: DHEA to Placebo (DHEA taken first, followed by washout and then Placebo) and Placebo to DHEA (placebo taken first, followed by washout and then DHEA). Short-axis steady state free precession (SSFP) imaging from the base of the heart through the apex. Ventricular EFs are calculated by dividing respective stroke volumes (EDV-ESV) by EDVs. | Posted | Median | 95% Confidence Interval | % Change in RV ejection fraction | Baseline to 18 weeks (treatment period 1), 18 weeks to 40 weeks (treatment period 2) |
|
|
|
|
| Primary | RVESV, mL | Change in right ventricular end-systolic volume from baseline to 18 weeks (treatment period 1) and 18 week to 40 weeks (treatment period 2) in both groups: DHEA to Placebo (DHEA taken first, followed by washout and then Placebo) and Placebo to DHEA (placebo taken first, followed by washout and then DHEA). Short-axis steady state free precession (SSFP) imaging was obtained from the base of the heart through the apex. The endocardial and epicardial borders of both ventricles are be traced manually on short axis cine images at end-diastole and end-systole with exclusion of the papillary muscles and trabeculae. EDV and ESV are calculated using Simpson's rule by summation of areas on each slice multiplied by the sum of slice thickness and image gap. | Posted | Median | 95% Confidence Interval | mL | Baseline to 18 weeks (treatment period 1), 18 weeks to 40 weeks (treatment period 2) |
|
|
|
|
| Primary | RV Stroke Volume, mL | Change in Right ventricular stroke volume by cardiac MRI from baseline to 18 weeks (treatment period 1) and 18 week to 40 weeks (treatment period 2) in both groups: DHEA to Placebo (DHEA taken first, followed by washout and then Placebo) and Placebo to DHEA (placebo taken first, followed by washout and then DHEA). Stroke volume is calculated through breath-hold through-plane phase contrast imaging with a velocity encoding gradient (VENC) of <120 cm/s (larger if aliasing present) in the main PA ~2-3 cm above the pulmonary valve plane, with imaging plane oriented orthogonal to the main PA. Free-breathing phase contrast imaging in the same plane and VENC with averaging over 4 respiratory cycles. | Posted | Median | 95% Confidence Interval | mL | Baseline to 18 weeks (treatment period 1), 18 weeks to 40 weeks (treatment period 2) |
|
|
|
|
| Primary | RV Mass, g | Right ventricular mass, Change in RV mass from baseline to 18 weeks (treatment period 1) and 18 week to 40 weeks (treatment period 2) in both groups: DHEA to Placebo (DHEA taken first, followed by washout and then Placebo) and Placebo to DHEA (placebo taken first, followed by washout and then DHEA). Short-axis steady state free precession (SSFP) imaging from the base of the heart through the apex. RV mass is determined at the end-diastole phase as the difference between end-diastolic epicardial and endocardial volumes X heart specific gravity (1.05 g/cm3). | Posted | Median | 95% Confidence Interval | g | 18 weeks, 40 weeks |
|
|
|
|
| Secondary | Change in Six Minute Walk Distance (6MWD) Between DHEA and Placebo | Change in 6MWD from baseline to 18 weeks (treatment period 1) and 18 week to 40 weeks (treatment period 2) in both groups. | Posted | Median | 95% Confidence Interval | meters | Baseline to 18 weeks (treatment period 1), 18 weeks to 40 weeks (treatment period 2) |
|
|
|
|
| Secondary | Change in World Health Organization (WHO) Functional Class | Change in WHO Functional Class (I - IV with IV indicating worse symptoms) between DHEA and placebo. | Posted | Median | Inter-Quartile Range | score on a scale | Baseline to 18 weeks (treatment period 1), 18 weeks to 40 weeks (treatment period 2) |
|
|
|
|
| Secondary | Change in Short Form-36 Summary Scores for Physical and Mental Components | Change in Short Form-36 summary scores for physical and mental components (range 0 - 100, higher scores indicating better quality of life). | Posted | Median | 95% Confidence Interval | score on a scale | Baseline to 18 weeks (treatment period 1), 18 weeks to 40 weeks (treatment period 2) |
|
|
|
|
| Secondary | Change in emPHasis-10 | Change in emPHasis-10 score (range 0 - 50, higher scores indicating worse quality of life) between DHEA and placebo. | Posted | Median | 95% Confidence Interval | score on a scale | Baseline to 18 weeks (treatment period 1), 18 weeks to 40 weeks (treatment period 2) |
|
|
|
|
| Secondary | Change in NT-proBNP Between DHEA and Placebo | Change in serum level of NT-proBNP between DHEA and placebo. | Posted | Median | 95% Confidence Interval | pg/mL | Baseline to 18 weeks (treatment period 1), 18 weeks to 40 weeks (treatment period 2) |
|
|
|
|
| Secondary | Change in DHEA-S (ug/dL) | Change in DHEA-S from baseline to 18 weeks (treatment period 1) and 18 week to 40 weeks (treatment period 2) in both groups. | Posted | Median | 95% Confidence Interval | ug/dL | Baseline to 18 weeks (treatment period 1), 18 weeks to 40 weeks (treatment period 2) |
|
|
|
|
| Secondary | Change in Estradiol, pg/mL | Change in Estradiol, pg/mL from baseline to 18 weeks (treatment period 1) and 18 week to 40 weeks (treatment period 2) in both groups. | Posted | Median | 95% Confidence Interval | pg/mL | Baseline to 18 weeks (treatment period 1), 18 weeks to 40 weeks (treatment period 2) |
|
|
|
|
| Secondary | Change in Testosterone, ng/dL | Change in Testosterone, ng/dL from baseline to 18 weeks (treatment period 1) and 18 week to 40 weeks (treatment period 2) in both groups. | Posted | Median | 95% Confidence Interval | ng/dL | Baseline to 18 weeks (treatment period 1), 18 weeks to 40 weeks (treatment period 2) |
|
|
|
|
| Secondary | Change in Progesterone, ng/mL | Change in Progesterone, ng/mL from baseline to 18 weeks (treatment period 1) and 18 week to 40 weeks (treatment period 2) in both groups. | Posted | Median | 95% Confidence Interval | ng/mL | Baseline to 18 weeks (treatment period 1), 18 weeks to 40 weeks (treatment period 2) |
|
|
|
|
| Secondary | Change in Follicle Stimulating Hormone (FSH), mIU/mL | Change in FSH, mIU/mL from baseline to 18 weeks (treatment period 1) and 18 week to 40 weeks (treatment period 2) in both groups. | Posted | Median | 95% Confidence Interval | mIU/mL | Baseline to 18 weeks (treatment period 1), 18 weeks to 40 weeks (treatment period 2) |
|
|
|
|
| Secondary | Change in Sex Hormone Binding Globulin (SHBG), Nmol/L | Change in SHBG, nmol/L from baseline to 18 weeks (treatment period 1) and 18 week to 40 weeks (treatment period 2) in both groups. | Posted | Median | 95% Confidence Interval | nmol/L | Baseline to 18 weeks (treatment period 1), 18 weeks to 40 weeks (treatment period 2) |
|
|
|
|
| Secondary | Change in Luteinizing Hormone, mIU/mL | Change in Luteinizing hormone, mIU/mL from baseline to 18 weeks (treatment period 1) and 18 week to 40 weeks (treatment period 2) in both groups. | Posted | Median | 95% Confidence Interval | mIU/mL | Baseline to 18 weeks (treatment period 1), 18 weeks to 40 weeks (treatment period 2) |
|
|
|
|
| Secondary | Change in Prolactin, μIU/mL | Change in Prolactin, μIU/mL from baseline to 18 weeks (treatment period 1) and 18 week to 40 weeks (treatment period 2) in both groups. | Posted | Median | 95% Confidence Interval | μIU/mL | Baseline to 18 weeks (treatment period 1), 18 weeks to 40 weeks (treatment period 2) |
|
|
|
|
| Secondary | Change in C-peptide, ng/mL | Change in C-peptide, ng/mL from baseline to 18 weeks (treatment period 1) and 18 week to 40 weeks (treatment period 2) in both groups. | Posted | Median | 95% Confidence Interval | ng/mL | Baseline to 18 weeks (treatment period 1), 18 weeks to 40 weeks (treatment period 2) |
|
|
|
|
| Secondary | Change in Insulin, μU/mL | Change in Insulin, μU/mL from baseline to 18 weeks (treatment period 1) and 18 week to 40 weeks (treatment period 2) in both groups. | Posted | Median | 95% Confidence Interval | μU/mL | Baseline to 18 weeks (treatment period 1), 18 weeks to 40 weeks (treatment period 2) |
|
|
|
|
| Secondary | Cortisol, μg/dL^2 | Change in Cortisol, μg/dL^2 (micrograms per deciliter squared) from baseline to 18 weeks (treatment period 1) and 18 week to 40 weeks (treatment period 2) in both groups. | Posted | Median | 95% Confidence Interval | μg/dL^2 | Baseline to 18 weeks (treatment period 1), 18 weeks to 40 weeks (treatment period 2) |
|
|
|
|
| Secondary | Left Ventricular Ejection Fraction, % | Change in left ventricular ejection fraction (%) measured by Cardiac Magnetic Resonance Imaging (MRI). Change in LVEG from baseline to 18 weeks (treatment period 1) and 18 week to 40 weeks (treatment period 2) in both groups: DHEA to Placebo (DHEA taken first, followed by washout and then Placebo) and Placebo to DHEA (placebo taken first, followed by washout and then DHEA). Short-axis steady state free precession (SSFP) imaging from the base of the heart through the apex was used.. Ventricular EFs are calculated by dividing respective stroke volumes (EDV-ESV) by EDVs. Normal Values: 56-78% for both males and females. | Posted | Median | Inter-Quartile Range | % Change in LVEF | Baseline to 18 weeks (treatment period 1), 18 weeks to 40 weeks (treatment period 2) |
|
|
|
|
| Secondary | Left Ventricular End-diastolic Volume (LVEDV) (mL) | Change in left ventricular LVEDV measured by Cardiac Magnetic Resonance Imaging (MRI). Change in LVEDV from baseline to 18 weeks (treatment period 1) and 18 week to 40 weeks (treatment period 2) in both groups: DHEA to Placebo (DHEA taken first, followed by washout and then Placebo) and Placebo to DHEA (placebo taken first, followed by washout and then DHEA). Short-axis steady state free precession (SSFP) imaging was obtained from the base of the heart through the apex. The endocardial and epicardial borders of both ventricles are be traced manually on short axis cine images at end-diastole and end-systole with exclusion of the papillary muscles and trabeculae. EDV and ESV are calculated using Simpson's rule by summation of areas on each slice multiplied by the sum of slice thickness and image gap. Normal values (Male):77-195 ml. Normal values (female):58-154 ml. | Posted | Median | Inter-Quartile Range | mL | Baseline to 18 weeks (treatment period 1), 18 weeks to 40 weeks (treatment period 2) |
|
|
|
|
| Secondary | Left Ventricular End-systolic Volume (LVESV) (mL) | Change in LVESV measured by Cardiac Magnetic Resonance Imaging (MRI). Change in LVEDV from baseline to 18 weeks (treatment period 1) and 18 week to 40 weeks (treatment period 2) in both groups: DHEA to Placebo (DHEA taken first, followed by washout and then Placebo) and Placebo to DHEA (placebo taken first, followed by washout and then DHEA). Short-axis steady state free precession (SSFP) imaging was obtained from the base of the heart through the apex. The endocardial and epicardial borders of both ventricles are be traced manually on short axis cine images at end-diastole and end-systole with exclusion of the papillary muscles and trabeculae. EDV and ESV are calculated using Simpson's rule by summation of areas on each slice multiplied by the sum of slice thickness and image gap. Normal Values (Female):13-51 ml. Normal values (Male):19-72 ml | Posted | Median | Inter-Quartile Range | mL | Baseline to 18 weeks (treatment period 1), 18 weeks to 40 weeks (treatment period 2) |
|
|
|
|
| Secondary | Left Ventricular Stroke Volume (mL) | Change in Left ventricular stroke volume measured by Cardiac Magnetic Resonance Imaging (MRI). Change in Left ventricular stroke volume from baseline to 18 weeks (treatment period 1) and 18 week to 40 weeks (treatment period 2) in both groups: DHEA to Placebo (DHEA taken first, followed by washout and then Placebo) and Placebo to DHEA (placebo taken first, followed by washout and then DHEA). Stroke volume is calculated through breath-hold through-plane phase contrast imaging with a velocity encoding gradient (VENC) of <120 cm/s (larger if aliasing present) in the main PA ~2-3 cm above the pulmonary valve plane, with imaging plane oriented orthogonal to the main PA. Free-breathing phase contrast imaging in the same plane and VENC with averaging over 4 respiratory cycles. | Posted | Median | Inter-Quartile Range | mL | Baseline to 18 weeks (treatment period 1), 18 weeks to 40 weeks (treatment period 2) |
|
|
|
|
| Secondary | Left Ventricular End Diastolic Mass, g | Change in Left ventricular end diastolic mass, g measured by Cardiac Magnetic Resonance Imaging (MRI). Change in Left ventricular end diastolic mass, g from baseline to 18 weeks (treatment period 1) and 18 week to 40 weeks (treatment period 2) in both groups: DHEA to Placebo (DHEA taken first, followed by washout and then Placebo) and Placebo to DHEA (placebo taken first, followed by washout and then DHEA). Short-axis steady state free precession (SSFP) imaging from the base of the heart through the apex. LV mass is determined at the end-diastole phase as the difference between end-diastolic epicardial and endocardial volumes X heart specific gravity (1.05 g/cm3). | Posted | Median | Inter-Quartile Range | g | Baseline to 18 weeks (treatment period 1), 18 weeks to 40 weeks (treatment period 2) |
|
|
|
|
| Secondary | Treatment-related Side Effects and Adverse Events | Treatment-related side effects and adverse events (as assessed by CTCAE v4.0). All participants in this trial received both DHEA and Placebo during the trial, therefore, side-effects and adverse events are listed according to the treatment or washout period in which they occurred. | Data shown as n (%) of all adverse events during DHEA, placebo or the washout period. Includes only events that occurred ≥ 2 times during the study. *Specifically queried at each study visit. | Posted | Number | participants | 18 weeks, 40 weeks |
|
|
|
| 0 |
| 26 |
| 2 |
| 26 |
| 16 |
| 26 |
| EG001 | Placebo | 1 placebo tablet taken by mouth once a day for 18 weeks Placebo: 1 placebo tablet taken by mouth once a day for 18 weeks | 0 | 26 | 10 | 26 | 16 | 26 |
| EG002 | DHEA Washout | Washout Period between Placebo and DHEA | 0 | 26 | 1 | 26 | 2 | 26 |
| EG003 | Placebo Washout | Washout Period DHEA and Placebo | 0 | 26 | 2 | 26 | 6 | 26 |
|
| 10053206 | Musculoskeletal and connective tissue disorders | MEDRA | Systematic Assessment | Fracture displacement |
|
| 10073974 | Musculoskeletal and connective tissue disorders | MEDRA | Systematic Assessment | Device dislocation |
|
| 10056871 | Skin and subcutaneous tissue disorders | MEDRA | Systematic Assessment | Device Failure |
|
| 10060842 | Respiratory, thoracic and mediastinal disorders | MEDRA | Systematic Assessment | Hypercapnic Respiratory failure |
|
| 10040047 | Blood and lymphatic system disorders | MEDRA | Systematic Assessment | Sepsis |
|
| 10020850 | Endocrine disorders | MEDRA | Systematic Assessment | Hyperthyroidism |
|
| 10002272 | Blood and lymphatic system disorders | MEDRA | Systematic Assessment | Anaemia |
|
| 10007810 | Product Issues | MEDRA | Systematic Assessment | Device related infection |
|
| 10038695 | Respiratory, thoracic and mediastinal disorders | MEDRA | Systematic Assessment | Respiratory failure |
|
| 10000081 | Gastrointestinal disorders | MEDRA | Systematic Assessment | Abdominal pain |
|
| 10084268 | Infections and infestations | MEDRA | Systematic Assessment | COVID-19 |
|
| 10042772 | General disorders | MEDRA | Systematic Assessment | syncope |
|
| 10002034 | Blood and lymphatic system disorders | MEDRA | Systematic Assessment | Hospitalization due to anemia |
|
| 10039911 | Nervous system disorders | MEDRA | Systematic Assessment | Hospitalization due to seizure |
|
|
| 10000496 | Skin and subcutaneous tissue disorders | MEDRA | Systematic Assessment | Acne |
|
| 10001760 | Skin and subcutaneous tissue disorders | MEDRA | Systematic Assessment | Alopecia |
|
| 10002856 | Psychiatric disorders | MEDRA | Systematic Assessment | Anxiety Attack |
|
| 10003664 | Cardiac disorders | MEDRA | Systematic Assessment | Atrial septal defect |
|
| 10003875 | Immune system disorders | MEDRA | Systematic Assessment | Axillary Lymph node |
|
| 10003988 | Musculoskeletal and connective tissue disorders | MEDRA | Systematic Assessment | Back pain |
|
| 10006373 | Skin and subcutaneous tissue disorders | MEDRA | Systematic Assessment | Onychoclasis |
|
| 10007810 | Infections and infestations | MEDRA | Systematic Assessment | Device related infection |
|
| 10007909 | Skin and subcutaneous tissue disorders | MEDRA | Systematic Assessment | Cellulitis |
|
| 10009851 | Infections and infestations | MEDRA | Systematic Assessment | Cold |
|
| 10012727 | Gastrointestinal disorders | MEDRA | Systematic Assessment | Diarrhea |
|
| 10013036 | Eye disorders | MEDRA | Systematic Assessment | Diplopia |
|
| 10013573 | Ear and labyrinth disorders | MEDRA | Systematic Assessment | Dizziness |
|
| 10013582 | Ear and labyrinth disorders | MEDRA | Systematic Assessment | Dizziness |
|
| 10013774 | Eye disorders | MEDRA | Systematic Assessment | Dry eye |
|
| 10013786 | Skin and subcutaneous tissue disorders | MEDRA | Systematic Assessment | Dry skin |
|
| 10013877 | Vascular disorders | MEDRA | Systematic Assessment | Deep vein thrombosis |
|
| 10014542 | Gastrointestinal disorders | MEDRA | Systematic Assessment | Emesis |
|
| 10015573 | Skin and subcutaneous tissue disorders | MEDRA | Systematic Assessment | Acne |
|
| 10015988 | Eye disorders | MEDRA | Systematic Assessment | Eyelid infection |
|
| 10016791 | Infections and infestations | MEDRA | Systematic Assessment | Influenza like illness |
|
| 10016952 | Gastrointestinal disorders | MEDRA | Systematic Assessment | Food poisoning |
|
| 10017853 | Gastrointestinal disorders | MEDRA | Systematic Assessment | Gastritis |
|
| 10019044 | Skin and subcutaneous tissue disorders | MEDRA | Systematic Assessment | Hair Growth on face |
|
| 10019045 | Skin and subcutaneous tissue disorders | MEDRA | Systematic Assessment | Hair loss |
|
| 10019211 | Nervous system disorders | MEDRA | Systematic Assessment | Headache |
|
| 10019428 | Blood and lymphatic system disorders | MEDRA | Systematic Assessment | Hematoma |
|
| 10019523 | Respiratory, thoracic and mediastinal disorders | MEDRA | Systematic Assessment | Hemoptysis |
|
| 10021018 | Blood and lymphatic system disorders | MEDRA | Systematic Assessment | hypokalemia |
|
| 10021143 | Respiratory, thoracic and mediastinal disorders | MEDRA | Systematic Assessment | Hypoxia |
|
| 10021282 | Nervous system disorders | MEDRA | Systematic Assessment | IIIrd nerve paralysis |
|
| 10021680 | Respiratory, thoracic and mediastinal disorders | MEDRA | Systematic Assessment | Dyspnoea |
|
| 10022437 | Psychiatric disorders | MEDRA | Systematic Assessment | Insomnia |
|
| 10023086 | Skin and subcutaneous tissue disorders | MEDRA | Systematic Assessment | Pruritus |
|
| 10023090 | Eye disorders | MEDRA | Systematic Assessment | Eye pruritus |
|
| 10023222 | Musculoskeletal and connective tissue disorders | MEDRA | Systematic Assessment | joint pain |
|
| 10027192 | Nervous system disorders | MEDRA | Systematic Assessment | Meningioma benign |
|
| 10028050 | General disorders | MEDRA | Systematic Assessment | Abnormal MRI |
|
| 10029410 | General disorders | MEDRA | Systematic Assessment | Night sweats |
|
| 10033411 | Musculoskeletal and connective tissue disorders | MEDRA | Systematic Assessment | Arthralgia |
|
| 10033432 | Musculoskeletal and connective tissue disorders | MEDRA | Systematic Assessment | Pain in hip |
|
| 10033557 | Cardiac disorders | MEDRA | Systematic Assessment | Palpitations |
|
| 10033733 | Skin and subcutaneous tissue disorders | MEDRA | Systematic Assessment | Papule (on chin) |
|
| 10034474 | Cardiac disorders | MEDRA | Systematic Assessment | Pericardial Effusion |
|
| 10036653 | General disorders | MEDRA | Systematic Assessment | Presyncope |
|
| 10037402 | Respiratory, thoracic and mediastinal disorders | MEDRA | Systematic Assessment | PH worsening |
|
| 10037844 | Skin and subcutaneous tissue disorders | MEDRA | Systematic Assessment | Rash |
|
| 10039792 | Skin and subcutaneous tissue disorders | MEDRA | Systematic Assessment | Seborrhoea (oily hair) |
|
| 10040604 | Respiratory, thoracic and mediastinal disorders | MEDRA | Systematic Assessment | Shortness of breath |
|
| 10042702 | General disorders | MEDRA | Systematic Assessment | Peripheral swelling |
|
| 10046300 | Infections and infestations | MEDRA | Systematic Assessment | Upper respiratory tract infection |
|
| 10046901 | Reproductive system and breast disorders | MEDRA | Systematic Assessment | Vaginal discharge |
|
| 10047115 | Vascular disorders | MEDRA | Systematic Assessment | Vasculitis |
|
| 10049201 | Skin and subcutaneous tissue disorders | MEDRA | Systematic Assessment | Rash |
|
| 10049498 | Respiratory, thoracic and mediastinal disorders | MEDRA | Systematic Assessment | Pulmonary mass |
|
| 10051626 | Eye disorders | MEDRA | Systematic Assessment | Conjunctivitis bacterial |
|
| 10051792 | Product Issues | MEDRA | Systematic Assessment | Device Failure |
|
| 10056739 | Blood and lymphatic system disorders | MEDRA | Systematic Assessment | WBC increased |
|
| 10059294 | Skin and subcutaneous tissue disorders | MEDRA | Systematic Assessment | Seborrhoea |
|
| 10059295 | Skin and subcutaneous tissue disorders | MEDRA | Systematic Assessment | Dry/Itchy Scalp |
|
| 10073974 | Product Issues | MEDRA | Systematic Assessment | Device dislocation |
|
| 10077730 | Respiratory, thoracic and mediastinal disorders | MEDRA | Systematic Assessment | Clinical Worsening - Change in WHO functional class |
|
| 10080818 | General disorders | MEDRA | Systematic Assessment | Peripheral swelling |
|
Not provided
Not provided
Not provided
| Fused-Ring Compounds |
| D011083 | Polycyclic Compounds |
| D015068 | 17-Ketosteroids |
| D007664 | Ketosteroids |
| D000305 | Adrenal Cortex Hormones |
| D006728 | Hormones |
| D006730 | Hormones, Hormone Substitutes, and Hormone Antagonists |
| D045165 | Testosterone Congeners |
| D012739 | Gonadal Steroid Hormones |
| D042341 | Gonadal Hormones |
Treatment period 2: DHEA to Placebo group, % change between 18 and 40 Weeks Treatment period 2: Placebo to DHEA group, % change between 18 and 40 Weeks
| generalized estimating equations (GEE) |
with sandwich estimation to correct for model misspecification. |
| 0.03 |
| Superiority |
Treatment period 2: DHEA to Placebo group, % change between 18 and 40 Weeks Treatment period 2: Placebo to DHEA group, % change between 18 and 40 Weeks
| generalized estimating equations (GEE) |
| 0.052 |
| Superiority |
Treatment period 2: DHEA to Placebo group, % change between 18 and 40 Weeks Treatment period 2: Placebo to DHEA group, % change between 18 and 40 Weeks
| generalized estimating equations (GEE) |
| 0.96 |
| Superiority |
Treatment period 2: DHEA to Placebo group, % change between 18 and 40 Weeks Treatment period 2: Placebo to DHEA group, % change between 18 and 40 Weeks
| generalized estimating equations (GEE) w |
| 0.61 |
| Superiority |
Treatment period 2: DHEA to Placebo group, % change between 18 and 40 Weeks Treatment period 2: Placebo to DHEA group, % change between 18 and 40 Weeks
| generalized estimating equations |
| 0.84 |
| Superiority |
Treatment period 2: DHEA to Placebo group, % change between 18 and 40 Weeks Treatment period 2: Placebo to DHEA group, % change between 18 and 40 Weeks
| generalized estimating equations |
| 0.71 |
| Superiority |
Treatment period 2: DHEA to Placebo group, % change between 18 and 40 Weeks Treatment period 2: Placebo to DHEA group, % change between 18 and 40 Weeks
| generalized estimating equations |
| 0.44 |
| Superiority |
Treatment period 2: DHEA to Placebo group, change between 18 and 40 Weeks Treatment period 2: Placebo to DHEA group, change between 18 and 40 Weeks
| generalized estimating equations (GEE) |
| 0.34 |
| Superiority |
Treatment period 2: DHEA to Placebo group, change between 18 and 40 Weeks Treatment period 2: Placebo to DHEA group, change between 18 and 40 Weeks
| generalized estimating equations (GEE) w |
| 0.99 |
| Superiority |
| SF-36, MCS |
|
SF-36 PCS |
| generalized estimating equations (GEE) |
| 0.044 |
| Superiority |
Treatment period 2: DHEA to Placebo group, change between 18 and 40 Weeks Treatment period 2: Placebo to DHEA group, change between 18 and 40 Weeks |
| SF-36 MCS | generalized estimating equations (GEE) | 0.77 | Superiority | Treatment period 1: DHEA to Placebo group, change at 18 Weeks Treatment period 1: Placebo to DHEA group, change at 18 Weeks |
| SF-MCS | generalized estimating equations (GEE) | 0.98 | Superiority | Treatment period 2: DHEA to Placebo group, change between 18 and 40 Weeks Treatment period 2: Placebo to DHEA group, change between 18 and 40 Weeks |
Treatment period 2: DHEA to Placebo group, change between 18 and 40 Weeks Treatment period 2: Placebo to DHEA group, change between 18 and 40 Weeks
| generalized estimating equations (GEE) |
| 0.037 |
| Superiority |
Treatment period 2: DHEA to Placebo group, % change between 18 and 40 Weeks Treatment period 2: Placebo to DHEA group, % change between 18 and 40 Weeks
| generalized estimating equations (GEE) |
| 0.9 |
| Superiority |
Treatment period 2: DHEA to Placebo group, % change between 18 and 40 Weeks Treatment period 2: Placebo to DHEA group, % change between 18 and 40 Weeks
| generalized estimating equations (GEE) |
| 0.034 |
| Superiority |
Treatment period 2: DHEA to Placebo group, change between 18 and 40 Weeks Treatment period 2: Placebo to DHEA group, change between 18 and 40 Weeks
| generalized estimating equations (GEE) |
| 0.23 |
| Superiority |
Treatment period 2: DHEA to Placebo group, change between 18 and 40 Weeks Treatment period 2: Placebo to DHEA group, change between 18 and 40 Weeks
| generalized estimating equations (GEE) |
| <0.0001 |
| Superiority |
Treatment period 2: DHEA to Placebo group, change between 18 and 40 Weeks Treatment period 2: Placebo to DHEA group, change between 18 and 40 Weeks
| generalized estimating equations (GEE) |
| 0.28 |
| Superiority |
Treatment period 2: DHEA to Placebo group, change between 18 and 40 Weeks Treatment period 2: Placebo to DHEA group, change between 18 and 40 Weeks
| generalized estimating equations (GEE) |
| 0.65 |
| Superiority |
Treatment period 2: DHEA to Placebo group, change between 18 and 40 Weeks Treatment period 2: Placebo to DHEA group, change between 18 and 40 Weeks
| generalized estimating equations (GEE) |
| <0.01 |
| Superiority |
Treatment period 2: DHEA to Placebo group, change between 18 and 40 Weeks Treatment period 2: Placebo to DHEA group, change between 18 and 40 Weeks
| generalized estimating equations (GEE) |
| 0.26 |
| Superiority |
Treatment period 2: DHEA to Placebo group, change between 18 and 40 Weeks Treatment period 2: Placebo to DHEA group, change between 18 and 40 Weeks
| generalized estimating equations (GEE) |
| 0.94 |
| Superiority |
Treatment period 2: DHEA to Placebo group, change between 18 and 40 Weeks Treatment period 2: Placebo to DHEA group, change between 18 and 40 Weeks
| generalized estimating equations (GEE) |
| 0.83 |
| Superiority |
Treatment period 2: DHEA to Placebo group, change between 18 and 40 Weeks Treatment period 2: Placebo to DHEA group, change between 18 and 40 Weeks
| generalized estimating equations (GEE) |
| 0.74 |
| Superiority |
| generalized estimating equations (GEE) |
| 0.05 |
| Superiority |
Treatment period 2: DHEA to Placebo group, change between 18 and 40 Weeks Treatment period 2: Placebo to DHEA group, change between 18 and 40 Weeks
| generalized estimating equations |
| 0.23 |
| Superiority |
Treatment period 2: DHEA to Placebo group, change between 18 and 40 Weeks Treatment period 2: Placebo to DHEA group, change between 18 and 40 Weeks
| generalized estimating equations (GEE) |
| 0.94 |
| Superiority |
Treatment period 2: DHEA to Placebo group, change between 18 and 40 Weeks Treatment period 2: Placebo to DHEA group, change between 18 and 40 Weeks
| generalized estimating equations (GEE) |
| 0.39 |
| Superiority |
Treatment period 2: DHEA to Placebo group, change between 18 and 40 Weeks Treatment period 2: Placebo to DHEA group, change between 18 and 40 Weeks
| generalized estimating equations (GEE) |
| 0.88 |
| Superiority |
Treatment period 2: DHEA to Placebo group, change between 18 and 40 Weeks Treatment period 2: Placebo to DHEA group, change between 18 and 40 Weeks
| generalized estimating equations (GEE) |
| 1.00 |
| Superiority |
| Title | Measurements |
|---|---|
|
| Seborrhoea |
|
| Hair Growth |
|
| Hair Loss |
|
| Headache |
|
| Diarrhea |
|
| Dizziness |
|
| Dry skin |
|
| Hypokalemia |
|
| Peripheral swelling |
|
| Pruritis |
|
| Rash |
|
| Sepsis |
|