Pulmonary Arterial HypertensionPhase 2PHANTOMNCT03229499
Pulmonary Hypertension and Anastrozole Trial
A Phase 2 study testing Anastrozole in people with Pulmonary Arterial Hypertension, run by University of Pennsylvania at 7 sites in United States.
Trial summary
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). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Pulmonary Arterial Hypertension
- What is being tested?
- Anastrozole
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 84 participants
- Where
- 7 sitesUnited States
- Study dates
- 7 Dec 2017to 22 Jul 2022
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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:
- > 50 years old and a) have not menstruated during the preceding 12 months or b) have follicle-stimulating hormone (FSH) levels (> 40 IU/L) or
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- < 50 years and FSH (> 40 IU/L) or
- having had a bilateral oophorectomy.
- Informed consent.
Who cannot join
- Age < 18.
- Current treatment with estrogen, hormone therapy, or anti-hormone therapy (tamoxifen, fulvestrant, etc.)
- WHO Class IV functional status.
- History of invasive breast cancer.
- Clinically significant untreated sleep apnea.
- Left-sided valvular disease (more than moderate mitral valve stenosis or insufficiency or aortic stenosis or insufficiency), pulmonary artery or valve stenosis, or ejection fraction < 45% on most recent echocardiography (within 1 year).
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- Initiation of PAH therapy (prostacyclin analogues, endothelin-1 receptor antagonists, phosphodiesterase-5 inhibitors, riociguat, selexipag) within three months of enrollment; the dose must be stable for at least three months prior to Baseline Visit. PAH therapy which is stopped and then restarted or has dose changes which are not related to initiation and uptitration will be allowed within 3 months prior to the Baseline Visit.
- Hospitalized or acutely ill.
- Renal failure (creatinine ≥ 2.0).
- Hypercalcemia.
- Severe osteoporosis: T score -2.5 to -3.4 without bone modifying treatment OR T score = - 3.5 or lower
- Child-Pugh Class C cirrhosis.
- Current or recent (< 3 months) chronic heavy alcohol consumption.
- Enrollment in a clinical trial or concurrent use of another investigational drug or device within 30 days of screening visit.
Trial locations and contacts
7 sites in 1 country.
7 sites in 1 country
United States7 sites
University of Colorado - Denver
Aurora, Colorado, 80045
Johns Hopkins University
Baltimore, Maryland, 21218
Rhode Island Hospital
East Providence, Rhode Island, 02914
Vanderbilt University
Nashville, Tennessee, 37232
University of Pennsylvania
Philadelphia, Pennsylvania, 19104
Washington University
St Louis, Missouri, 63130
Stanford University
Stanford, California, 94305
About this study
Official title: Pulmonary Hypertension and Anastrozole Trial (PHANTOM)
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).
- Conditions
- Pulmonary Arterial Hypertension
- Being tested
- Anastrozole, Placebo Oral Tablet
- Keywords
- Randomized Controlled Trial, clinical trial, anastrozole, pulmonary hypertension, pulmonary arterial hypertension, PAH
- Medical subject headings
- Pulmonary Arterial Hypertension, Hypertension, Pulmonary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AnastrozoleDrug
Also called Arimidex
Anastrozole is an aromatase inhibitor indicated for:
- adjuvant treatment of postmenopausal women with hormone receptor-positive breast cancer
- first-line treatment of postmenopausal women with hormone receptor-positive or hormone receptor unknown locally advanced or metastatic breast cancer
- treatment of advanced breast cancer in postmenopausal women with disease progression following tamoxifen therapy.
Placebo Oral TabletDrug
matching placebo tablet
| Study group | Role | What they receive |
|---|---|---|
| Anastrozole | Experimental | 1mg (1 tablet)taken by mouth once a day for one year Receives: Drug: Anastrozole |
| Placebo | Placebo comparator | 1 tablet taken by mouth once a day for one year Receives: Drug: Placebo Oral Tablet |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Six-minute Walk Distance
Change in the distance walked in six minutes adjusted for baseline value and sex
Measured: 6 months
Secondary outcomes (7)
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
Measured: 6 months
Change in Plasma NT-proBNP
Change in log10 NT-proBNP adjusted for baseline value and sex
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Jul 2017
- Study start7 Dec 2017
- Primary completion22 Jul 2022
- Study completion22 Jul 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Pulmonary Arterial HypertensionStudies of the same condition, open and under way.
- More trials run by University of PennsylvaniaThe 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
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 84 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Pennsylvania
- Sponsor type
- University, hospital or other
- Collaborators
- National Heart, Lung, and Blood Institute (NHLBI), Johns Hopkins University, University of Colorado, Denver, Rhode Island Hospital, Stanford University, Vanderbilt University, Washington University School of Medicine
- Responsible party
- Sponsor
- Investigators
- Steven M Kawut, MD, Principal investigator, University of Pennsylvania
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03229499
- Sponsor's protocol ID
- 827486
- Other IDs
- R01HL134905
- Acronym
- PHANTOM
- Status last verified
- Apr 2024
- First submitted
- 18 Jul 2017
- Last updated
- 1 May 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
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.
PubMed 38747680
Study at a glance
From the registry record, last updated by the sponsor on 1 May 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pulmonary Hypertension and Anastrozole Trial (PHANTOM)
- ClinicalTrials.gov ID
- NCT03229499
- Acronym / sponsor ID
- PHANTOM / 827486
- Conditions
- Pulmonary Arterial Hypertension
- Keywords
- Randomized Controlled Trial, clinical trial, anastrozole, pulmonary hypertension, pulmonary arterial hypertension, PAH
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 84 participants (actual)
- Lead sponsor
- University of PennsylvaniaUniversity, hospital or other
- Collaborators
- National Heart, Lung, and Blood Institute (NHLBI), Johns Hopkins University, University of Colorado, Denver, Rhode Island Hospital, Stanford University, Vanderbilt University, Washington University School of Medicine
- Study start
- 7 Dec 2017 (actual)
- Primary completion
- 22 Jul 2022 (actual)
- Study completion
- 22 Jul 2022 (actual)
- First posted
- 25 Jul 2017 (actual)
- Results
- Posted 1 May 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Anastrozole | Experimental | Drug: Anastrozole1mg (1 tablet)taken by mouth once a day for one year |
| Placebo | Placebo comparator | Drug: Placebo Oral Tablet1 tablet taken by mouth once a day for one year |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- 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:
Key exclusion criteria (14 listed)
- Age < 18.
- Current treatment with estrogen, hormone therapy, or anti-hormone therapy (tamoxifen, fulvestrant, etc.)
- WHO Class IV functional status.
- History of invasive breast cancer.
- Clinically significant untreated sleep apnea.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AnastrozoleAlso: ArimidexAnastrozole is an aromatase inhibitor indicated for: * adjuvant treatment of postmenopausal women with hormone receptor-positive breast cancer * first-line treatment of postmenopausal women with hormone receptor-positive or hormone receptor unknown locally advanced or metastatic breast cancer * treatment of advanced breast cancer in postmenopausal women with disease progression following tamoxifen therapy. | Drug | Anastrozole |
| Placebo Oral Tabletmatching placebo tablet | Drug | Placebo |
Primary outcome
Change in Six-minute Walk Distance
Time frame: 6 months
Change in the distance walked in six minutes adjusted for baseline value and sex
7 secondary outcomes
Change in Right Ventricular Function: Tricuspid Annular Systolic Plane Excursion (TAPSE)
Time frame: 6 months
Change in Plasma NT-proBNP
Time frame: 6months
Change in the Medical Outcomes Study Questionnaire Short Form-36 (SF36) Physical Component Summary (PCS) Score Adjusted for Baseline Value and Sex.
Time frame: 6months
Change in the emPHasis-10 Score Adjusted for Baseline Value and Sex
Time frame: 6months
Change in Actigraphy-measured Physical Activity: Change in 7-day Median Daily Vector Magnitude Count
Time frame: 6months
Number of Participants With a Clinical Worsening Event Between Anastrozole and Placebo Groups
Time frame: 12 months
Change in Bone Mineral Density: Lumbar Spine Between Anastrozole and Placebo Groups
Time frame: 12 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 May 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 38747680Kawut 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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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. This study is not recruiting at the moment.
Steven M Kawut, MD principal investigator
University of Pennsylvania
7 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Anastrozole in people with Pulmonary Arterial Hypertension, run by University of Pennsylvania at 7 sites 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 9 inclusion and 14 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: finished, as of the sponsor's last update on 1 May 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites 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. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 1 May 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in Six-minute Walk Distance (time frame: 6 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03229499, which also lists 1 linked publication.
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 1 May 2024; AzTrials last refreshed its copy on 3 Nov 2026.