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CompletedNCT02911844ERA-PAHUpdated Dec 9, 2019Results posted

Estrogen Receptor Antagonist in Patients With Pulmonary Arterial Hypertension

A Phase 2 interventional study of Fulvestrant in Pulmonary Arterial Hypertension, sponsored by University of Pennsylvania. Completed at 1 site in United States. Open to female participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2019-12-09.

Sponsored by University of Pennsylvania · Phase 2, Interventional, and Treatment

Phase
Phase 2
Study type
Interventional
Enrollment
5
Allocation
Not applicable
Ages
18 Years and older
Sex
Female
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Study summary

The main purpose of this clinical trial is to examine the feasibility and effects of fulvestrant in post-menopausal women with pulmonary arterial hypertension (PAH). The study will evaluate how well the drug is tolerated. The study will evaluate changes in circulating hematopoietic progenitor cells, plasma hormone levels, NT-proBNP, and other plasma biomarkers after the administration of fulvestrant. Changes in tricuspid annular plane systolic excursion, stroke volume index, right ventricular fractional area change, and other echo parameters after fulvestrant administration will be evaluated as well as changes in distance walked in six minutes.

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Conditions studied

  • Pulmonary Arterial Hypertension
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In context

Pulmonary Arterial Hypertension

761 studies on the registry are indexed under Pulmonary Arterial Hypertension; 142 are open to participants now.

This study's enrollment of 5 is below the median of 38 across 509 interventional studies indexed under Pulmonary Arterial Hypertension.

Browse Pulmonary Arterial Hypertension studies →

Lead sponsor

University of Pennsylvania is the lead sponsor of 1,635 studies on the registry; 239 are open to participants now.

Of its 154 completed or terminated interventional studies of FDA-regulated products, 104 (68%) have results posted.

Counted across the registry records on this site, refreshed daily.

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Who can participate

Ages eligible
18 Years and older
Sexes eligible
Female
Accepts healthy volunteers
No

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 pulmonary vascular resistance > 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.
  • 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.

Exclusion criteria

Exclusion Criteria:

  • Age \< 18.
  • Treatment with estrogen or anti-hormone therapy (tamoxifen, anastrozole, 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 or receptor agonists, endothelin-1 receptor antagonists, phosphodiesterase-5 inhibitors, soluble guanylate cyclase stimulators) within three months of enrollment; the dose must be stable for at least 3 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.
  • Hormone therapy.
  • Use of warfarin or other anticoagulant (use of aspirin is permitted).
  • Platelet count \<100,000.
  • 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.
05

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
5 participants (actual)

Study arms

  • Other
    Fulvestrant

    500 mg administered intramuscularly (as two 5 mL injections) on days 0, 14, 28 and 56

    Drug: Fulvestrant

Interventions

  • DrugFulvestrant

    Fulvestrant 500 mg administered intramuscularly into the buttocks slowly as two 5 mL injections, one in each buttock, on days 0, 14, 28 and 56. Fulvestrant 250 mg (one 5 mL injection) will be used in patients with Child-Pugh Class B liver disease.

    Also known as: Faslodex

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What researchers measure

Primary outcomes

  1. Change From Baseline of Plasma Estradiol Levels

    Plasma estradiol levels are obtained and measured from blood samples collected from each participant. Difference in change from baseline to 9 weeks in plasma estradiol levels

    Time frame: Baseline to 9 weeks

  2. Change From Baseline of Tricuspid Annular Plane Systolic Excursion (TAPSE)

    Measure obtained from echocardiogram completed on participants Difference in change from baseline to 9 weeks in TAPSE

    Time frame: Baseline to 9 weeks

  3. Change From Baseline of Six Minute Walk Distance

    Measure obtained from six minute walk test completed by participants Difference in change from baseline to 9 weeks in the six minute walk test distance

    Time frame: Baseline to 9 weeks

  4. Change From Baseline of Plasma NT-proBNP Level

    Plasma NT-proBNP levels are obtained and measured from blood samples collected from each participant. Difference in change from baseline to 9 weeks in plasma NT-proBNP levels

    Time frame: Baseline to 9 weeks

07

Results

Posted Dec 9, 2019
Limitations and caveats
Small size Open-label and uncontrolled design, making it difficult to directly attribute the findings to the study intervention Short duration of the study may have affected the results

Participant flow

Participant flow — Overall Study
MilestoneFulvestrant
Started5
Completed5
Not completed0

Outcome measures

PrimaryChange From Baseline of Plasma Estradiol Levels

Plasma estradiol levels are obtained and measured from blood samples collected from each participant. Difference in change from baseline to 9 weeks in plasma estradiol levels

Time frame:
Baseline to 9 weeks
Reported as:
Median · pg/ml
Change From Baseline of Plasma Estradiol Levels
pg/mlFulvestrant
Change From Baseline of Plasma Estradiol Levels-1.26 (-1.7 to -0.26)
PrimaryChange From Baseline of Tricuspid Annular Plane Systolic Excursion (TAPSE)

Measure obtained from echocardiogram completed on participants Difference in change from baseline to 9 weeks in TAPSE

Time frame:
Baseline to 9 weeks
Reported as:
Median · millimeter
Change From Baseline of Tricuspid Annular Plane Systolic Excursion (TAPSE)
millimeterFulvestrant
Change From Baseline of Tricuspid Annular Plane Systolic Excursion (TAPSE)2 (-3 to 5.5)
PrimaryChange From Baseline of Six Minute Walk Distance

Measure obtained from six minute walk test completed by participants Difference in change from baseline to 9 weeks in the six minute walk test distance

Time frame:
Baseline to 9 weeks
Reported as:
Median · meters
Change From Baseline of Six Minute Walk Distance
metersFulvestrant
Change From Baseline of Six Minute Walk Distance31 (10 to 32)
PrimaryChange From Baseline of Plasma NT-proBNP Level

Plasma NT-proBNP levels are obtained and measured from blood samples collected from each participant. Difference in change from baseline to 9 weeks in plasma NT-proBNP levels

Time frame:
Baseline to 9 weeks
Reported as:
Median · pg/ml
Change From Baseline of Plasma NT-proBNP Level
pg/mlFulvestrant
Change From Baseline of Plasma NT-proBNP Level42.1 (6.7 to 114)

Adverse events

Collected over 12 weeks. Non-serious events are listed at a 0% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Fulvestrant0/5 (0%)0/5 (0%)5/5 (100%)
Most frequent other events
Most frequent other events
EventFulvestrant
DiarrheaGastrointestinal disorders2/5
Injection Site PainGeneral disorders1/5
Hot flashesNervous system disorders1/5
Shoulder painGeneral disorders1/5
Worsened dyspneaRespiratory, thoracic and mediastinal disorders1/5
Joint painGeneral disorders1/5
Worsening hemodynamicsCardiac disorders1/5
Increased NT-proBNP levelCardiac disorders1/5

Baseline characteristics

Age, Continuous
Age, Continuous(years)Fulvestrant
Median55 (52 to 66)
Sex: Female, Male
Sex: Female, Male(Participants)Fulvestrant
Female5
Male0
Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)Fulvestrant
Hispanic or Latino0
Not Hispanic or Latino5
Unknown or Not Reported0
Race (NIH/OMB)
Race (NIH/OMB)(Participants)Fulvestrant
American Indian or Alaska Native0
Asian0
Native Hawaiian or Other Pacific Islander0
Black or African American2
White3
More than one race0
Unknown or Not Reported0
Region of Enrollment
Region of Enrollment(participants)Fulvestrant
United States5
Childs-Pugh Class
Childs-Pugh Class(Participants)Fulvestrant
A5
B or C0
World Health Organization functional class
World Health Organization functional class(Participants)Fulvestrant
Class I0
Class II4
Class III1
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Study locations

1 site
  • University of Pennsylvania
    Philadelphia, Pennsylvania 19104, United States
09

References and documents

Publications

  • Kawut SM, Pinder D, Al-Naamani N, McCormick A, Palevsky HI, Fritz J, Smith KA, Mazurek JA, Doyle MF, MacLean MR, DeMichele A, Mankoff DA. Fulvestrant for the Treatment of Pulmonary Arterial Hypertension. Ann Am Thorac Soc. 2019 Nov;16(11):1456-1459. doi: 10.1513/AnnalsATS.201904-328RL. No abstract available. PubMed 31314997 ↗

Study documents

  • Protocol and statistical analysis plan · Jul 18, 2016

Documents are hosted by the registry — open the source record to download them.

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Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Dec 9, 2019, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
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Registry details

Key details

Study ID
NCT02911844
Lead sponsor
University of Pennsylvania
Responsible party
Sponsor
First posted
Sep 22, 2016
Start date
Apr 10, 2017
Primary completion
Dec 5, 2018
Completion
Dec 5, 2018
Results posted
Dec 9, 2019
Last update
Dec 9, 2019

Study contacts

Steven M Kawut, MD
principal investigator · University of Pennsylvania

Oversight

Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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