Trial Outcomes & Findings for Oral CXA-10 in Pulmonary Arterial Hypertension (NCT NCT04125745)

Results Overview

Numbers of treatment-related adverse events is assessed by CTCAE v4.0

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

1 participants

Primary outcome timeframe

Baseline to 12 Weeks

Results posted on

2022-01-12

Participant Flow

Participant milestones

Participant milestones
Measure	CXA-10 Oral CXA-10 300 mg once daily for 12 weeks CXA-10: Each subject will receive oral CXA-10 at the dose of 300 mg once daily for 12 weeks
Overall Study STARTED	1
Overall Study COMPLETED	1
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Oral CXA-10 in Pulmonary Arterial Hypertension

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	CXA-10 n=1 Participants Oral CXA-10 300 mg once daily for 12 weeks CXA-10: Each subject will receive oral CXA-10 at the dose of 300 mg once daily for 12 weeks
Age, Categorical <=18 years	0 Participants n=99 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=99 Participants
Age, Categorical >=65 years	1 Participants n=99 Participants
Sex: Female, Male Female	1 Participants n=99 Participants
Sex: Female, Male Male	0 Participants n=99 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=99 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	1 Participants n=99 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=99 Participants
Region of Enrollment United States	1 participants n=99 Participants

PRIMARY outcome

Timeframe: Baseline to 12 Weeks

Numbers of treatment-related adverse events is assessed by CTCAE v4.0

Outcome measures

Outcome measures
Measure	CXA-10 n=1 Participants Oral CXA-10 300 mg once daily for 12 weeks CXA-10: Each subject will receive oral CXA-10 at the dose of 300 mg once daily for 12 weeks
Number of Treatment-Related Adverse Events as Assessed by CTCAE v4.0 in 12 Weeks	2 Events

SECONDARY outcome

Timeframe: Baseline and 12 Weeks

Pulmonary vascular resistance (PVR) will be measured by right heart catheterization; results reported as changed from baseline in Woods units. PVR is calculated as (mean pulmonary artery pressure - pulmonary artery wedge pressure)/cardiac output, where pressures are in units of mm Hg and cardiac output is in units of L/min. Pulmonary hypertension is defined as PVR \> 3 Woods units. A negative change implies a decrease in PVR which would be considered an improvement.

Outcome measures

Outcome measures
Measure	CXA-10 n=1 Participants Oral CXA-10 300 mg once daily for 12 weeks CXA-10: Each subject will receive oral CXA-10 at the dose of 300 mg once daily for 12 weeks
Change From Baseline in Pulmonary Vascular Resistance is Measured at Baseline and 12 Weeks by Right Heart Catheterization	-1.2 Woods units

SECONDARY outcome

Timeframe: Baseline and 12 Weeks

Pulmonary Artery mean pressure will be measured by right heart catheterization

Outcome measures

Outcome measures
Measure	CXA-10 n=1 Participants Oral CXA-10 300 mg once daily for 12 weeks CXA-10: Each subject will receive oral CXA-10 at the dose of 300 mg once daily for 12 weeks
Change From Baseline in Mean Pulmonary Artery Pressure is Measured at Baseline and 12 Weeks by Right Heart Catheterization	-5 mm Hg

SECONDARY outcome

Timeframe: at baseline and 12 weeks

Population: Drug and funding withdrawn prior to analysis; data were not collected.

Change from baseline in RV function as measured by tricuspid annular plane systolic excursion (TAPSE) as assessed by echocardiograms at 12 weeks

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: at baseline and 12 weeks

Change from baseline in 6 minute walk distance at 12 weeks

Outcome measures

Outcome measures
Measure	CXA-10 n=1 Participants Oral CXA-10 300 mg once daily for 12 weeks CXA-10: Each subject will receive oral CXA-10 at the dose of 300 mg once daily for 12 weeks
Changes From Baseline in Functional Exercise Capacity by Assessing 6 Minute Walk Distance Test	23.5 meters

SECONDARY outcome

Timeframe: at Baseline and 12 Weeks

Population: Drug and funding withdrawn; data were not collected

Blood samples will be collected for NT-proBNP analysis

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: at Baseline and 12 Weeks

Population: Drug and funding withdrawn prior to analysis; data were not collected

The New York Heart Association Classification of Functional Status of Patients with pulmonary hypertension will be used to classify disease severity in PAH. It places patients in one of four categories based on how much they are limited during physical activity. Measure Description: Class I: No limitation of physical activity. Ordinary physical activity does not cause undue fatigue, palpitation, dyspnea (shortness of breath). Class II: Slight limitation of physical activity. Comfortable at rest. Ordinary physical activity results in fatigue, palpitation, dyspnea (shortness of breath). Class III: Marked limitation of physical activity. Comfortable at rest. Less than ordinary activity causes fatigue, palpitation, or dyspnea. Class IV: Unable to carry on any physical activity without discomfort. Symptoms of dyspnea at rest. If any physical activity is undertaken, discomfort increases.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline and 12 Weeks

Population: Drug and funding withdrawn prior to analysis; data were not collected

Continuous physical activity monitoring will be conducted using a noninvasive accelerometer. The device must be worn for a minimum of 7 days at home prior to receiving the first dose of the study drug and prior to the end of the study drug treatment at week 12.

Outcome measures

Outcome data not reported

Adverse Events

CXA-10

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	CXA-10 n=1 participants at risk Oral CXA-10 300 mg once daily for 12 weeks CXA-10: Each subject will receive oral CXA-10 at the dose of 300 mg once daily for 12 weeks
Gastrointestinal disorders Diarrhea	100.0% 1/1 • Number of events 1 • 12 weeks
Respiratory, thoracic and mediastinal disorders Shortness of Breath	100.0% 1/1 • Number of events 1 • 12 weeks

Additional Information

Marc Simon, MD, Director of Pulmonary Vascular Disease and Pulmonary Hypertension Comprehensive Care

University of California San Francisco

Phone: 415-476-1325

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place