Trial Outcomes & Findings for Estrogen Receptor Antagonist in Patients With Pulmonary Arterial Hypertension (NCT NCT02911844)

NCT ID: NCT02911844

Last Updated: 2019-12-09

Results Overview

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

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 5 participants
• Primary outcome timeframe: Baseline to 9 weeks
• Results posted on: 2019-12-09

Participant Flow

Participant milestones

Measure	Fulvestrant 500 mg administered intramuscularly (as two 5 mL injections) on days 0, 14, 28 and 56 Fulvestrant: 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.
Overall Study STARTED	5
Overall Study COMPLETED	5
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Estrogen Receptor Antagonist in Patients With Pulmonary Arterial Hypertension

Baseline characteristics by cohort

Measure	Fulvestrant n=5 Participants 500 mg administered intramuscularly (as two 5 mL injections) on days 0, 14, 28 and 56 Fulvestrant: 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.
Age, Continuous	55 years n=5 Participants
Sex: Female, Male Female	5 Participants n=5 Participants
Sex: Female, Male Male	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	5 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	2 Participants n=5 Participants
Race (NIH/OMB) White	3 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment United States	5 participants n=5 Participants
Childs-Pugh Class A	5 Participants n=5 Participants
Childs-Pugh Class B or C	0 Participants n=5 Participants
World Health Organization functional class Class I	0 Participants n=5 Participants
World Health Organization functional class Class II	4 Participants n=5 Participants
World Health Organization functional class Class III	1 Participants n=5 Participants

PRIMARY outcome

Timeframe: Baseline to 9 weeks

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

Outcome measures

Measure	Fulvestrant n=5 Participants 500 mg administered intramuscularly (as two 5 mL injections) on days 0, 14, 28 and 56 Fulvestrant: 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.
Change From Baseline of Plasma Estradiol Levels	-1.26 pg/ml Interval -1.7 to -0.26

PRIMARY outcome

Timeframe: Baseline to 9 weeks

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

Outcome measures

Measure	Fulvestrant n=5 Participants 500 mg administered intramuscularly (as two 5 mL injections) on days 0, 14, 28 and 56 Fulvestrant: 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.
Change From Baseline of Tricuspid Annular Plane Systolic Excursion (TAPSE)	2 millimeter Interval -3.0 to 5.5

PRIMARY outcome

Timeframe: Baseline to 9 weeks

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

Outcome measures

Measure	Fulvestrant n=5 Participants 500 mg administered intramuscularly (as two 5 mL injections) on days 0, 14, 28 and 56 Fulvestrant: 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.
Change From Baseline of Six Minute Walk Distance	31 meters Interval 10.0 to 32.0

PRIMARY outcome

Timeframe: Baseline to 9 weeks

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

Outcome measures

Measure	Fulvestrant n=5 Participants 500 mg administered intramuscularly (as two 5 mL injections) on days 0, 14, 28 and 56 Fulvestrant: 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.
Change From Baseline of Plasma NT-proBNP Level	42.1 pg/ml Interval 6.7 to 114.0

Adverse Events

Fulvestrant

Serious events: 0 serious events

Other events: 5 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure	Fulvestrant n=5 participants at risk 500 mg administered intramuscularly (as two 5 mL injections) on days 0, 14, 28 and 56 Fulvestrant: 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.
Gastrointestinal disorders Diarrhea	40.0% 2/5 • Number of events 2 • 12 weeks
General disorders Injection Site Pain	20.0% 1/5 • Number of events 1 • 12 weeks
Nervous system disorders Hot flashes	20.0% 1/5 • Number of events 1 • 12 weeks
General disorders Shoulder pain	20.0% 1/5 • Number of events 1 • 12 weeks
Respiratory, thoracic and mediastinal disorders Worsened dyspnea	20.0% 1/5 • Number of events 1 • 12 weeks
General disorders Joint pain	20.0% 1/5 • Number of events 1 • 12 weeks
Cardiac disorders Worsening hemodynamics	20.0% 1/5 • Number of events 1 • 12 weeks
Cardiac disorders Increased NT-proBNP level	20.0% 1/5 • Number of events 1 • 12 weeks

Additional Information

Dr. Steven Kawut

University of Pennsylvania

Phone: 215-573-0258

Email: kawut@upenn.edu

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place