Pulmonary Arterial HypertensionPhase 2AIPHNCT01545336
Anastrozole in Patients With Pulmonary Arterial Hypertension
A Phase 2 study testing Anastrozole in people with Pulmonary Arterial Hypertension, run by University of Pennsylvania at 3 sites in United States.
Trial summary
The main purpose of this clinical trial is to study the safety and efficacy of anastrozole in adults diagnosed with pulmonary arterial hypertension (PAH). The study will evaluate how well the drug is tolerated. 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
- 18 participants
- Where
- 3 sitesUnited States
- Study dates
- Oct 2012to Sep 2015
- 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.
- 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 limitations in musculoskeletal function or coordination.
- If female, post-menopausal state, defined as:
- > 50 years old AND
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- a) have not menstruated during the preceding 12 months OR
- b) have follicle-stimulating hormone (FSH) levels (> 40 IU/L) OR
- < 50 years and FSH (> 40 IU/L) OR
- having had a bilateral oophorectomy
- Informed consent
Who cannot join
- Treatment with estrogen or anti-hormone therapy (tamoxifen, fulvestrant, etc.)
- WHO Class IV functional status
- History of 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 echocardiography
- Initiation of PAH therapy (prostacyclin analogues, endothelin-1 receptor antagonists, phosphodiesterase-5 inhibitors) within three months of enrollment; the dose must be stable for at least 3 months prior to Baseline Visit
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- Hormone therapy
- Hospitalized or acutely ill
- Renal failure (creatinine > 2.0)
- Child-Pugh Class C cirrhosis
- Current or recent (< 6 months) chronic heavy alcohol consumption
- Current use of another investigational drug (non-FDA approved) for PAH
- Enrollment in a clinical trial within one month of screening
- Age < 18
Trial locations and contacts
3 sites in 1 country.
United States3 sites
University of Pennsylvania - Penn Presbyterian
Philadelphia, Pennsylvania, 19104
University of Pennsylvania - Perelman Center
Philadelphia, Pennsylvania, 19104
Brown University - Rhode Island Hospital
Providence, Rhode Island, 02903
About this study
Official title: A Double-blind, Placebo-controlled Phase II Study of Anastrozole in Patients With Pulmonary Arterial Hypertension
The main purpose of this clinical trial is to study the safety and efficacy of anastrozole in adults diagnosed with pulmonary arterial hypertension (PAH). The study will evaluate how well the drug is tolerated. The study will also evaluate if anastrozole effects estradiol (E2) hormone levels, a sex hormone, and improves the function of the lower right chamber of the heart (right ventricle).
- Conditions
- Pulmonary Arterial Hypertension
- Being tested
- Anastrozole, Placebo
- Medical subject headings
- Pulmonary Arterial Hypertension
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AnastrozoleDrug
Also called Arimidex
1 mg tablet to be taken 1 time daily
PlaceboDrug
Also called Sugar pill manufactured to mimic Anastrozole 1 mg tablet
1 mg tablet to be taken 1 time daily
| Study group | Role | What they receive |
|---|---|---|
| Anastrozole | Experimental | 1 mg tablet by mouth once daily for 3 months Receives: Drug: Anastrozole |
| Placebo | Placebo comparator | Placebo tablet by mouth once daily for 3 months Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Plasma Estradiol (E2) Level
Measured: Baseline, 3 months
Tricuspid Annular Plane Systolic Excursion (TAPSE)
Measured: Baseline, 3 months
Secondary outcomes (1)
Six Minute Walk Distance
Measured: Baseline, 3 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Mar 2012 (estimated)
- Study startOct 2012
- Primary completionJun 2015
- Study completionSep 2015
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
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 18 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Pennsylvania
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Steven M Kawut, MD, MS, Principal investigator, University of Pennsylvania
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01545336
- Sponsor's protocol ID
- 815035
- Acronym
- AIPH
- Status last verified
- Sep 2016
- First submitted
- 29 Feb 2012
- Last updated
- 27 Oct 2016
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Kawut SM, Archer-Chicko CL, DeMichele A, Fritz JS, Klinger JR, Ky B, Palevsky HI, Palmisciano AJ, Patel M, Pinder D, Propert KJ, Smith KA, Stanczyk F, Tracy R, Vaidya A, Whittenhall ME, Ventetuolo CE. Anastrozole in Pulmonary Arterial Hypertension. A Randomized, Double-Blind, Placebo-controlled Trial. Am J Respir Crit Care Med. 2017 Feb 1;195(3):360-368. doi: 10.1164/rccm.201605-1024OC.
PubMed 27602993
Study at a glance
From the registry record, last updated by the sponsor on 27 Oct 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Double-blind, Placebo-controlled Phase II Study of Anastrozole in Patients With Pulmonary Arterial Hypertension
- ClinicalTrials.gov ID
- NCT01545336
- Acronym / sponsor ID
- AIPH / 815035
- Conditions
- Pulmonary Arterial Hypertension
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 18 participants (actual)
- Lead sponsor
- University of PennsylvaniaUniversity, hospital or other
- Study start
- Oct 2012
- Primary completion
- Jun 2015 (actual)
- Study completion
- Sep 2015 (actual)
- First posted
- 6 Mar 2012 (estimated)
- Results
- Posted 9 Aug 2016
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The main purpose of this clinical trial is to study the safety and efficacy of anastrozole in adults diagnosed with pulmonary arterial hypertension (PAH). The study will evaluate how well the drug is tolerated. The study will also evaluate if anastrozole effects estradiol (E2) hormone levels, a sex hormone, and improves the function of the lower right chamber of the heart (right ventricle).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Anastrozole | Experimental | Drug: Anastrozole1 mg tablet by mouth once daily for 3 months |
| Placebo | Placebo comparator | Drug: PlaceboPlacebo tablet by mouth once daily for 3 months |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (11 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.
- 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 limitations in musculoskeletal function or coordination.
- If female, post-menopausal state, defined as:
Key exclusion criteria (14 listed)
- Treatment with estrogen or anti-hormone therapy (tamoxifen, fulvestrant, etc.)
- WHO Class IV functional status
- History of 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 echocardiography
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: Arimidex1 mg tablet to be taken 1 time daily | Drug | Anastrozole |
| PlaceboAlso: Sugar pill manufactured to mimic Anastrozole 1 mg tablet1 mg tablet to be taken 1 time daily | Drug | Placebo |
Primary outcome
Plasma Estradiol (E2) Level
Time frame: Baseline, 3 months
Tricuspid Annular Plane Systolic Excursion (TAPSE)
Time frame: Baseline, 3 months
1 secondary outcome
Six Minute Walk Distance
Time frame: Baseline, 3 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Oct 2016.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Results publication: PubMed 27602993Kawut SM, Archer-Chicko CL, DeMichele A, Fritz JS, Klinger JR, Ky B, Palevsky HI, Palmisciano AJ, Patel M, Pinder D, Propert KJ, Smith KA, Stanczyk F, Tracy R, Vaidya A, Whittenhall ME, Ventetuolo CE. Anastrozole in Pulmonary Arterial Hypertension. A Randomized, Double-Blind, Placebo-controlled Trial. Am J Respir Crit Care Med. 2017 Feb 1;195(3):360-368. doi: 10.1164/rccm.201605-1024OC.
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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, MS principal investigator
University of Pennsylvania
3 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 3 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 11 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 27 Oct 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 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 9 Aug 2016). 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?
Plasma Estradiol (E2) Level (time frame: Baseline, 3 months). The study lists 2 primary outcomes in all. 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 NCT01545336, 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 27 Oct 2016.