NCT01545336

Brief 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. 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).

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
18

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Oct 2012

Typical duration for phase_2

Geographic Reach
1 country

3 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

February 29, 2012

Completed
6 days until next milestone

First Posted

Study publicly available on registry

March 6, 2012

Completed
7 months until next milestone

Study Start

First participant enrolled

October 1, 2012

Completed
2.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 1, 2015

Completed
3 months until next milestone

Study Completion

Last participant's last visit for all outcomes

September 1, 2015

Completed
11 months until next milestone

Results Posted

Study results publicly available

August 9, 2016

Completed
Last Updated

October 27, 2016

Status Verified

September 1, 2016

Enrollment Period

2.7 years

First QC Date

February 29, 2012

Results QC Date

June 29, 2016

Last Update Submit

September 20, 2016

Conditions

Outcome Measures

Primary Outcomes (2)

  • Plasma Estradiol (E2) Level

    Baseline, 3 months

  • Tricuspid Annular Plane Systolic Excursion (TAPSE)

    Baseline, 3 months

Secondary Outcomes (1)

  • Six Minute Walk Distance

    Baseline, 3 months

Study Arms (2)

Anastrozole

EXPERIMENTAL

1 mg tablet by mouth once daily for 3 months

Drug: Anastrozole

Placebo

PLACEBO COMPARATOR

Placebo tablet by mouth once daily for 3 months

Drug: Placebo

Interventions

1 mg tablet to be taken 1 time daily

Also known as: Arimidex
Anastrozole

1 mg tablet to be taken 1 time daily

Also known as: Sugar pill manufactured to mimic Anastrozole 1 mg tablet
Placebo

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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
  • 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

You may not qualify if:

  • 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
  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (3)

University of Pennsylvania - Penn Presbyterian

Philadelphia, Pennsylvania, 19104, United States

Location

University of Pennsylvania - Perelman Center

Philadelphia, Pennsylvania, 19104, United States

Location

Brown University - Rhode Island Hospital

Providence, Rhode Island, 02903, United States

Location

Related Publications (1)

  • 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.

MeSH Terms

Conditions

Pulmonary Arterial Hypertension

Interventions

AnastrozoleSugarsTablets

Condition Hierarchy (Ancestors)

Hypertension, PulmonaryLung DiseasesRespiratory Tract Diseases

Intervention Hierarchy (Ancestors)

NitrilesOrganic ChemicalsTriazolesAzolesHeterocyclic Compounds, 1-RingHeterocyclic CompoundsCarbohydratesDosage FormsPharmaceutical Preparations

Limitations and Caveats

* Small sample size * No changes in echo parameters * Unclear mechanism for impact on 6MWD

Results Point of Contact

Title
Dr. Steven M. Kawut
Organization
University of Pennsylvania

Study Officials

  • Steven M Kawut, MD, MS

    University of Pennsylvania

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
No
Restrictive Agreement
No

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
TRIPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

February 29, 2012

First Posted

March 6, 2012

Study Start

October 1, 2012

Primary Completion

June 1, 2015

Study Completion

September 1, 2015

Last Updated

October 27, 2016

Results First Posted

August 9, 2016

Record last verified: 2016-09

Locations