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| ID | Type | Description | Link |
|---|---|---|---|
| R01HL134905 | U.S. NIH Grant/Contract | View source |
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| Name | Class |
|---|---|
| National Heart, Lung, and Blood Institute (NHLBI) | NIH |
| Johns Hopkins University | OTHER |
| University of Colorado, Denver | OTHER |
| Rhode Island Hospital |
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The primary objectives of this study are to determine whether the study drug, anastrozole may improve six minute walk distance at six months compared to placebo and to assess safety and side effects up to twelve months in pulmonary arterial hypertension (PAH).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Anastrozole | Experimental | 1mg (1 tablet)taken by mouth once a day for one year |
|
| Placebo | Placebo Comparator | 1 tablet taken by mouth once a day for one year |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Anastrozole | Drug | Anastrozole is an aromatase inhibitor indicated for:
|
| Measure | Description | Time Frame |
|---|---|---|
| Change in Six-minute Walk Distance | Change in the distance walked in six minutes adjusted for baseline value and sex | 6 months |
| Measure | Description | Time Frame |
|---|---|---|
| Change in Right Ventricular Function: Tricuspid Annular Systolic Plane Excursion (TAPSE) | Change in tricuspid annular systolic plane excursion (TAPSE) adjusted for baseline value and sex. TAPSE is a measure of right ventricular function that is measured via cardiac echography | 6 months |
| Change in Plasma NT-proBNP |
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Inclusion Criteria:
Previous documentation of mean pulmonary artery pressure > 25 mm Hg with a pulmonary capillary wedge pressure (or left ventricular end-diastolic pressure) < 16 mm Hg and PVR > 3 WU at any time before study entry.
Diagnosis of PAH which is idiopathic, heritable, drug- or toxin-induced, or associated with connective tissue disease, congenital heart disease, portal hypertension, or HIV infection and receiving treatment for PAH.
Most recent pulmonary function tests with FEV1/FVC >50% AND either a) total lung capacity > 70% predicted or b) total lung capacity between 60% and 70% predicted with no more than mild interstitial lung disease on computerized tomography scan of the chest.
Ability to perform six minute walk testing without significant limitations in musculoskeletal function or coordination.
If female, post-menopausal state, defined as:
Informed consent.
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Steven M Kawut, MD | University of Pennsylvania | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Stanford University | Stanford | California | 94305 | United States | ||
| University of Colorado - Denver |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 38747680 | Derived | Kawut SM, Feng R, Ellenberg SS, Zamanian R, Bull T, Chakinala M, Mathai SC, Hemnes A, Lin G, Doyle M, Andrew R, MacLean M, Stasinopoulos I, Austin E, DeMichele A, Shou H, Minhas J, Song N, Moutchia J, Ventetuolo CE. Pulmonary Hypertension and Anastrozole (PHANTOM): A Randomized, Double-Blind, Placebo-Controlled Trial. Am J Respir Crit Care Med. 2024 Nov 1;210(9):1143-1151. doi: 10.1164/rccm.202402-0371OC. |
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| ID | Title | Description |
|---|---|---|
| FG000 | Anastrozole | 1mg (1 tablet)taken by mouth once a day for one year Anastrozole: Anastrozole is an aromatase inhibitor indicated for:
|
| FG001 | Placebo | 1 tablet taken by mouth once a day for one year Placebo Oral Tablet: matching placebo tablet |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Anastrozole | 1mg (1 tablet)taken by mouth once a day for one year Anastrozole: Anastrozole is an aromatase inhibitor indicated for:
|
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| 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 Six-minute Walk Distance | Change in the distance walked in six minutes adjusted for baseline value and sex | Some participants are missing data at follow-up and the Analysis Population includes only participants with non-missing data. Includes non-missing six-minute walk distance at 6 months | Posted | Least Squares Mean | 95% Confidence Interval | meters | 6 months |
|
12 months
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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 | Anastrozole | 1mg (1 tablet)taken by mouth once a day for one year Anastrozole: Anastrozole is an aromatase inhibitor indicated for:
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Catheter placement | Surgical and medical procedures | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Upper respiratory infection | Infections and infestations | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Dr. Steven M Kawut, MD, MS | Center for Clinical Epidemiology and Biostatistics, Perelman School of Medicine | 646-352-1654 | kawut@upenn.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 | Jun 17, 2020 | Feb 19, 2024 | Prot_SAP_000.pdf |
| ICF | No | No | Yes | Informed Consent Form | Jun 4, 2020 | Feb 19, 2024 | ICF_001.pdf |
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| ID | Term |
|---|---|
| D000081029 | Pulmonary Arterial Hypertension |
| D006976 | Hypertension, Pulmonary |
| ID | Term |
|---|---|
| D008171 | Lung Diseases |
| D012140 | Respiratory Tract Diseases |
| D006973 | Hypertension |
| D014652 | Vascular Diseases |
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| ID | Term |
|---|---|
| D000077384 | Anastrozole |
| ID | Term |
|---|---|
| D009570 | Nitriles |
| D009930 | Organic Chemicals |
| D014230 | Triazoles |
| D001393 | Azoles |
| D006573 |
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| OTHER |
| Stanford University | OTHER |
| Vanderbilt University | OTHER |
| Washington University School of Medicine | OTHER |
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|
|
| Placebo Oral Tablet | Drug | matching placebo tablet |
|
Change in log10 NT-proBNP adjusted for baseline value and sex |
| 6months |
| Change in the Medical Outcomes Study Questionnaire Short Form-36 (SF36) Physical Component Summary (PCS) Score Adjusted for Baseline Value and Sex. | Change in the Medical Outcomes Study Questionnaire Short Form-36 (SF36) physical component summary (PCS) score adjusted for baseline value and sex. SF36 PCS score is a subjective measure of physical health status, with scores ranging from 0 to 100. Higher scores represent better health status. | 6months |
| Change in the emPHasis-10 Score Adjusted for Baseline Value and Sex | Change in the emPHasis-10 score adjusted for baseline value and sex. EmPHasis-10 is a pulmonary hypertension-specific measure of quality of life which is scored from 0-50, with higher scores indicating worse quality of life. | 6months |
| Change in Actigraphy-measured Physical Activity: Change in 7-day Median Daily Vector Magnitude Count | Actigraphy-measured vector magnitude count is an estimate of physical activity intensity. The total vector magnitude counts per day was measured over 7 days using an actigraphy device, and the median value over the 7 days was obtained. This outcome measure is the change in the 7-day median value from baseline to follow-up, adjusted for the baseline value, sex, and actigraphy device wear time. | 6months |
| Number of Participants With a Clinical Worsening Event Between Anastrozole and Placebo Groups | Number of participants with a clinical worsening event. Clinical worsening was defined as the addition of new PAH therapies or dose increases in previously stable PAH therapy for increased symptoms, hospitalization for PAH progression and/or right-sided heart failure, lung transplantation, atrial septostomy, or all-cause death.' Unit of Measure is retained as 'Participants. | 12 months |
| Change in Bone Mineral Density: Lumbar Spine Between Anastrozole and Placebo Groups | Change in lumbar spine bone mineral density adjusted for baseline value and sex between anastrozole and placebo groups | 12 months |
| Aurora |
| Colorado |
| 80045 |
| United States |
| Johns Hopkins University | Baltimore | Maryland | 21218 | United States |
| Washington University | St Louis | Missouri | 63130 | United States |
| University of Pennsylvania | Philadelphia | Pennsylvania | 19104 | United States |
| Rhode Island Hospital | East Providence | Rhode Island | 02914 | United States |
| Vanderbilt University | Nashville | Tennessee | 37232 | United States |
| BG001 | Placebo | 1 tablet taken by mouth once a day for one year Placebo Oral Tablet: matching placebo tablet |
| BG002 | Total | Total of all reporting groups |
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Six-minute walk distance | Mean | Standard Deviation | meters |
|
| OG001 | Placebo | 1 tablet taken by mouth once a day for one year Placebo Oral Tablet: matching placebo tablet |
|
|
| Secondary | Change in Right Ventricular Function: Tricuspid Annular Systolic Plane Excursion (TAPSE) | Change in tricuspid annular systolic plane excursion (TAPSE) adjusted for baseline value and sex. TAPSE is a measure of right ventricular function that is measured via cardiac echography | Some participants are missing data at follow-up and the Analysis Population includes only participants with non-missing data. | Posted | Least Squares Mean | 95% Confidence Interval | millimeters | 6 months |
|
|
|
| Secondary | Change in Plasma NT-proBNP | Change in log10 NT-proBNP adjusted for baseline value and sex | Please note that some participants are missing data at follow-up and the Analysis Population includes only participants with non-missing data | Posted | Least Squares Mean | 95% Confidence Interval | log10(pg/ml) | 6months |
|
|
|
| Secondary | Change in the Medical Outcomes Study Questionnaire Short Form-36 (SF36) Physical Component Summary (PCS) Score Adjusted for Baseline Value and Sex. | Change in the Medical Outcomes Study Questionnaire Short Form-36 (SF36) physical component summary (PCS) score adjusted for baseline value and sex. SF36 PCS score is a subjective measure of physical health status, with scores ranging from 0 to 100. Higher scores represent better health status. | Please note that some participants are missing data at follow-up and the Analysis Population includes only participants with non-missing data. | Posted | Least Squares Mean | 95% Confidence Interval | units on a scale | 6months |
|
|
|
| Secondary | Change in the emPHasis-10 Score Adjusted for Baseline Value and Sex | Change in the emPHasis-10 score adjusted for baseline value and sex. EmPHasis-10 is a pulmonary hypertension-specific measure of quality of life which is scored from 0-50, with higher scores indicating worse quality of life. | Please note that some participants are missing data at follow-up and the Analysis Population includes only participants with non-missing data. | Posted | Least Squares Mean | 95% Confidence Interval | units on a scale | 6months |
|
|
|
| Secondary | Change in Actigraphy-measured Physical Activity: Change in 7-day Median Daily Vector Magnitude Count | Actigraphy-measured vector magnitude count is an estimate of physical activity intensity. The total vector magnitude counts per day was measured over 7 days using an actigraphy device, and the median value over the 7 days was obtained. This outcome measure is the change in the 7-day median value from baseline to follow-up, adjusted for the baseline value, sex, and actigraphy device wear time. | Please note that some participants are missing data at follow-up and the Analysis Population includes only participants with non-missing data. | Posted | Least Squares Mean | 95% Confidence Interval | counts per day(x10^5) | 6months |
|
|
|
| Secondary | Number of Participants With a Clinical Worsening Event Between Anastrozole and Placebo Groups | Number of participants with a clinical worsening event. Clinical worsening was defined as the addition of new PAH therapies or dose increases in previously stable PAH therapy for increased symptoms, hospitalization for PAH progression and/or right-sided heart failure, lung transplantation, atrial septostomy, or all-cause death.' Unit of Measure is retained as 'Participants. | Please note that some participants are missing data at follow-up and the Analysis Population includes only participants with non-missing data | Posted | Count of Participants | Participants | 12 months |
|
|
|
| Secondary | Change in Bone Mineral Density: Lumbar Spine Between Anastrozole and Placebo Groups | Change in lumbar spine bone mineral density adjusted for baseline value and sex between anastrozole and placebo groups | Please note that some participants are missing data at follow-up and the Analysis Population includes only participants with non-missing data | Posted | Least Squares Mean | 95% Confidence Interval | g/cm^2 | 12 months |
|
|
|
| 0 |
| 43 |
| 16 |
| 43 |
| 38 |
| 43 |
| EG001 | Placebo | 1 tablet taken by mouth once a day for one year Placebo Oral Tablet: matching placebo tablet | 3 | 41 | 10 | 41 | 38 | 41 |
| Dyspnea | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
|
| Heart Failure | Cardiac disorders | Systematic Assessment |
|
| Hypoxia | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
|
| Anaphylaxis | Immune system disorders | Systematic Assessment |
|
| Anemia | Blood and lymphatic system disorders | Systematic Assessment |
|
| Carcinoma hepatocellular | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | Systematic Assessment |
|
| Cardiac arrest | Cardiac disorders | Systematic Assessment |
|
| Chest pain - cardiac | Cardiac disorders | Systematic Assessment |
|
| Chronic leg ulcer | Skin and subcutaneous tissue disorders | Systematic Assessment |
|
| Coronavirus infection | Infections and infestations | Systematic Assessment |
|
| Diarrhea | Gastrointestinal disorders | Systematic Assessment |
|
| Disease progression | General disorders | Systematic Assessment |
|
| Esophagitis | Gastrointestinal disorders | Systematic Assessment |
|
| Fluid overload | Metabolism and nutrition disorders | Systematic Assessment |
|
| Fluid retention in tissues | Metabolism and nutrition disorders | Systematic Assessment |
|
| Hypotension | Vascular disorders | Systematic Assessment |
|
| Mallory Weiss tear | Gastrointestinal disorders | Systematic Assessment |
|
| Paresthesia | Nervous system disorders | Systematic Assessment |
|
| Pelvic mass | General disorders | Systematic Assessment |
|
| Pneumonia | Infections and infestations | Systematic Assessment |
|
| Pneumonitis | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
|
| Pulmonary hypertension | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
|
| Pyoderma gangrenosum | Skin and subcutaneous tissue disorders | Systematic Assessment |
|
| Respiratory failure | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
|
| Seizure | Nervous system disorders | Systematic Assessment |
|
| Small intestinal obstruction | Gastrointestinal disorders | Systematic Assessment |
|
| Subdural hematoma | Injury, poisoning and procedural complications | Systematic Assessment |
|
| Supraventricular tachycardia | Cardiac disorders | Systematic Assessment |
|
| Tenosynovitis | Musculoskeletal and connective tissue disorders | Systematic Assessment |
|
| Arthralgia | Musculoskeletal and connective tissue disorders | Systematic Assessment |
|
| Edema limbs | General disorders | Systematic Assessment |
|
| Hot flashes | Vascular disorders | Systematic Assessment |
|
| Dyspnea | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
|
| Headache | Nervous system disorders | Systematic Assessment |
|
| Depression | Psychiatric disorders | Systematic Assessment |
|
| Nausea | Gastrointestinal disorders | Systematic Assessment |
|
| Pain in extremity | Musculoskeletal and connective tissue disorders | Systematic Assessment |
|
| Back pain | Musculoskeletal and connective tissue disorders | Systematic Assessment |
|
| Diarrhea | Gastrointestinal disorders | Systematic Assessment |
|
| Fatigue | General disorders | Systematic Assessment |
|
| Hypotension | Vascular disorders | Systematic Assessment |
|
| Cough | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
|
| Myalgia | Musculoskeletal and connective tissue disorders | Systematic Assessment |
|
| Pruritus | Skin and subcutaneous tissue disorders | Systematic Assessment |
|
| Vaginal dryness | Reproductive system and breast disorders | Systematic Assessment |
|
| Anemia | Blood and lymphatic system disorders | Systematic Assessment |
|
| Joint range of motion decreased | Musculoskeletal and connective tissue disorders | Systematic Assessment |
|
| Anxiety | Psychiatric disorders | Systematic Assessment |
|
| Palpitations | Cardiac disorders | Systematic Assessment |
|
| Presyncope | Nervous system disorders | Systematic Assessment |
|
| Vertigo | Ear and labyrinth disorders | Systematic Assessment |
|
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| D002318 |
| Cardiovascular Diseases |
| Heterocyclic Compounds, 1-Ring |
| D006571 | Heterocyclic Compounds |