Phase 2 N=18 Randomized Triple-blind Treatment

Anastrozole in Patients With Pulmonary Arterial Hypertension

Pulmonary Arterial Hypertension

Bottom Line

View on ClinicalTrials.gov: NCT01545336 ↗

Enrolled (actual)

Serious AEs

11.1%

Results posted

Aug 2016

Primary outcome: Primary: Plasma Estradiol (E2) Level — -40; -4 % change from baseline

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: Anastrozole (Drug); Placebo (Drug)
Age: Adult, Older Adult · 18+ yrs
Sex: All
Sponsor: University of Pennsylvania
Primary completion: Jun 2015

Outcome Measures

Outcome	Result	p-value
PRIMARY Plasma Estradiol (E2) Level	-40; -4	—
PRIMARY Tricuspid Annular Plane Systolic Excursion (TAPSE)	7; 10	—
SECONDARY Six Minute Walk Distance	8; -2	—

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).

Eligibility Criteria

Inclusion Criteria

Previous documentation of mean pulmonary artery pressure > 25 mm Hg with a pulmonary capillary wedge pressure (or left ventricular end-diastolic pressure) 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
40 IU/L) OR
having had a bilateral oophorectomy
Informed consent

Exclusion Criteria

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 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

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT01545336). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.