Trial Outcomes & Findings for Ranolazine and Pulmonary Hypertension (NCT NCT01174173)

NCT ID: NCT01174173

Last Updated: 2018-05-11

Results Overview

Assessed as average improvement in WHO Functional Class. The WHO Functional Class score ranges from 1 to 4, with higher scores indicating more impairment

• Recruitment status: COMPLETED
• Study phase: PHASE3
• Target enrollment: 11 participants
• Primary outcome timeframe: 3 months
• Results posted on: 2018-05-11

Participant Flow

Participant milestones

Measure	Ranolazine 1000 mg PO BID Ranolazine: ranolazine 1000 mg PO BID for 3 months
Overall Study STARTED	11
Overall Study COMPLETED	10
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Measure	Ranolazine 1000 mg PO BID Ranolazine: ranolazine 1000 mg PO BID for 3 months
Overall Study drug-drug interaction	1

Baseline Characteristics

Ranolazine and Pulmonary Hypertension

Baseline characteristics by cohort

Measure	Ranolazine n=11 Participants 1000 mg PO BID Ranolazine: ranolazine 1000 mg PO BID for 3 months
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	10 Participants n=5 Participants
Age, Categorical >=65 years	1 Participants n=5 Participants
Age, Continuous	48 years STANDARD_DEVIATION 13 • n=5 Participants
Sex: Female, Male Female	8 Participants n=5 Participants
Sex: Female, Male Male	3 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	5 Participants n=5 Participants
Race (NIH/OMB) White	5 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	1 Participants n=5 Participants
Region of Enrollment United States	11 participants n=5 Participants
Exercise stress echocardiography with strain	63 Ejection Fraction (%) STANDARD_DEVIATION 5 • n=5 Participants
Right heart catheterization	48 Mean pulmonary artery pressure (mm Hg) STANDARD_DEVIATION 14 • n=5 Participants
6-minute walk test	383 meters STANDARD_DEVIATION 60 • n=5 Participants

PRIMARY outcome

Timeframe: 3 months

Population: Analysis was performed on all participants who completed as average change in WHO Functional class score from baseline to 3 months (Baseline, 3 months)

Assessed as average improvement in WHO Functional Class. The WHO Functional Class score ranges from 1 to 4, with higher scores indicating more impairment

Outcome measures

Measure	Ranolazine n=8 Participants 1000 mg PO BID Ranolazine: ranolazine 1000 mg PO BID for 3 months
Improve Angina Symptoms	1.75 units on a scale Standard Deviation 0.46

PRIMARY outcome

Timeframe: 3 Months

Population: Analysis was performed in all participants who completed the study and had a 6-minute walk test at baseline and at the conclusion of the study.

Improve Exercise Capacity measured by 6-Minute Walk Test

Outcome measures

Measure	Ranolazine n=6 Participants 1000 mg PO BID Ranolazine: ranolazine 1000 mg PO BID for 3 months
6-Minute Walk Test	419 meters Standard Deviation 80

PRIMARY outcome

Timeframe: 3 Months

Population: Analysis was performed on the 8 participants who completed the KCCQ questionnaire at baseline and at the conclusion of the study (month 3)

The Kansas City Cardiomyopathy Questionnaire is a 23-item, self-administered instrument that quantifies physical function, symptoms (frequency, severity and recent change), social function, self-efficacy and knowledge, and quality of life. The scores are averaged and transformed to a range of 0-100, in which higher scores reflect better health status and was performed at the conclusion of the study.

Outcome measures

Measure	Ranolazine n=8 Participants 1000 mg PO BID Ranolazine: ranolazine 1000 mg PO BID for 3 months
Improve Quality of Life Baseline	60.3 KCCQ Summary Score Standard Deviation 19.7
Improve Quality of Life Month 3	64.2 KCCQ Summary Score Standard Deviation 17.5

SECONDARY outcome

Timeframe: 3 months

The majority of patients did not undergo cardiac MRI because it was difficult for the patients to tolerate the imaging study. Therefore, we were not able to assess change in RV perfusion.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 3 months

Population: Analysis was performed on all participants who completed exercise stress echocardiography at baseline and conclusion of the study (month 3)

Change in absolute right ventricular (RV) longitudinal strain as assessed by exercise stress echocardiography with speckle-tracking echocardiography at baseline and conclusion of the study. An increase in exercise-induced change in RV longitudinal strain between baseline to conclusion of the study is indicative of improved RV function. If absolute RV longitudinal strain increases with exercise, that is a sign that the RV is working well. If absolute RV longitudinal strain decreases with exercise, that is a sign that the RV is not working well. Therefore, between baseline and conclusion of the study, if exercise-induced change in RV strain increases that means that the RV is working better at the conclusion of the study.

Outcome measures

Measure	Ranolazine n=8 Participants 1000 mg PO BID Ranolazine: ranolazine 1000 mg PO BID for 3 months
Absolute RV Longitudinal Strain Baseline	-1.4 percentage Standard Deviation 0.7
Absolute RV Longitudinal Strain Month 3	1.0 percentage Standard Deviation 0.5

SECONDARY outcome

Timeframe: 3 months

Population: Analysis was performed on all participants who completed right heart catheterization at the conclusion of the study

Mean pulmonary artery pressure was assessed invasively by right heart catheterization at the conclusion of the study to estimate right ventricular hemodynamics.

Outcome measures

Measure	Ranolazine n=8 Participants 1000 mg PO BID Ranolazine: ranolazine 1000 mg PO BID for 3 months
Right Ventricular Hemodynamics	48 mm Hg Standard Deviation 6

Adverse Events

Ranolazine

Serious events: 1 serious events

Other events: 6 other events

Deaths: 0 deaths

Serious adverse events

Measure	Ranolazine n=11 participants at risk Ranolazine: ranolazine 1000 mg PO BID for 3 months
Nervous system disorders Inpatient hospitalization	9.1% 1/11 • Number of events 1 • 3 months

Other adverse events

Measure	Ranolazine n=11 participants at risk Ranolazine: ranolazine 1000 mg PO BID for 3 months
Musculoskeletal and connective tissue disorders Myalgias	9.1% 1/11 • Number of events 1 • 3 months
Gastrointestinal disorders Constipation	27.3% 3/11 • Number of events 3 • 3 months
Gastrointestinal disorders nausea	18.2% 2/11 • Number of events 2 • 3 months

Additional Information

Sanjiv J. Shah, MD

Northwestern University

Phone: 3129262926

Email: sanjiv.shah@northwestern.edu

Results disclosure agreements

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