Trial Outcomes & Findings for Targeting Right Ventricle in Pulmonary Hypertension Gilead (NCT NCT02829034)

NCT ID: NCT02829034

Last Updated: 2019-02-26

Results Overview

Change in right ventricle ejection fraction as assessed by MRI

• Recruitment status: COMPLETED
• Study phase: PHASE4
• Target enrollment: 22 participants
• Primary outcome timeframe: 26 weeks
• Results posted on: 2019-02-26

Participant Flow

Participant milestones

Measure	Ranolazine Ranolazine 500mg by mouth twice per day and after two weeks increase to 1000mg by mouth twice per day Ranolazine: Ranolazine 500mg by mouth twice per day and after two weeks increases to 1000mg by mouth twice per day and continue for a total of 26 weeks.	Placebo Placebo by mouth twice per day Placebo: Placebo by mouth twice per day for a total of 26 weeks
Overall Study STARTED	14	8
Overall Study COMPLETED	9	6
Overall Study NOT COMPLETED	5	2

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Targeting Right Ventricle in Pulmonary Hypertension Gilead

Baseline characteristics by cohort

Measure	Ranolazine n=14 Participants Ranolazine 500mg by mouth twice per day and after two weeks increase to 1000mg by mouth twice per day Ranolazine: Ranolazine 500mg by mouth twice per day and after two weeks increases to 1000mg by mouth twice per day and continue for a total of 26 weeks.	Placebo n=8 Participants Placebo by mouth twice per day Placebo: Placebo by mouth twice per day for a total of 26 weeks	Total n=22 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	13 Participants n=5 Participants	7 Participants n=7 Participants	20 Participants n=5 Participants
Age, Categorical >=65 years	1 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants
Age, Continuous	55.4 years STANDARD_DEVIATION 16.9 • n=5 Participants	53.6 years STANDARD_DEVIATION 14.1 • n=7 Participants	54.8 years STANDARD_DEVIATION 16.0 • n=5 Participants
Sex: Female, Male Female	8 Participants n=5 Participants	5 Participants n=7 Participants	13 Participants n=5 Participants
Sex: Female, Male Male	6 Participants n=5 Participants	3 Participants n=7 Participants	9 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	5 Participants n=5 Participants	2 Participants n=7 Participants	7 Participants n=5 Participants
Race (NIH/OMB) White	9 Participants n=5 Participants	6 Participants n=7 Participants	15 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Region of Enrollment United States	14 participants n=5 Participants	8 participants n=7 Participants	22 participants n=5 Participants

PRIMARY outcome

Timeframe: 26 weeks

Population: participants who completed follow up study at 6 months.

Change in right ventricle ejection fraction as assessed by MRI

Outcome measures

Measure	Ranolazine n=9 Participants Ranolazine 500mg by mouth twice per day and after two weeks increase to 1000mg by mouth twice per day Ranolazine: Ranolazine 500mg by mouth twice per day and after two weeks increases to 1000mg by mouth twice per day and continue for a total of 26 weeks.	Placebo n=6 Participants Placebo by mouth twice per day Placebo: Placebo by mouth twice per day for a total of 26 weeks
Absolute Change From Baseline Right Ventricular Ejection Fraction (the Unit is Percentage)	7.56 percentage Standard Error 1.72	-3.99 percentage Standard Error 2.23

SECONDARY outcome

Timeframe: 6 months

Population: complete and recorded data

6-minute walk test

Outcome measures

Measure	Ranolazine n=8 Participants Ranolazine 500mg by mouth twice per day and after two weeks increase to 1000mg by mouth twice per day Ranolazine: Ranolazine 500mg by mouth twice per day and after two weeks increases to 1000mg by mouth twice per day and continue for a total of 26 weeks.	Placebo n=5 Participants Placebo by mouth twice per day Placebo: Placebo by mouth twice per day for a total of 26 weeks
Percent Change in 6min-walk-test Distance	-0.09 percentage of distance walked Standard Error 0.095	-0.06 percentage of distance walked Standard Error 0.125

SECONDARY outcome

Timeframe: 6 months

Population: completers

NT-proBNP measured at 6-months compared to baseline

Outcome measures

Measure	Ranolazine n=9 Participants Ranolazine 500mg by mouth twice per day and after two weeks increase to 1000mg by mouth twice per day Ranolazine: Ranolazine 500mg by mouth twice per day and after two weeks increases to 1000mg by mouth twice per day and continue for a total of 26 weeks.	Placebo n=6 Participants Placebo by mouth twice per day Placebo: Placebo by mouth twice per day for a total of 26 weeks
Change in N-terminal Pro B-type Natriuretic Peptide (NT-proBNP)	-119.72 pg/mL Standard Error 276.42	-287.75 pg/mL Standard Error 353.81

Adverse Events

Ranolazine

Serious events: 3 serious events

Other events: 11 other events

Deaths: 0 deaths

Placebo

Serious events: 2 serious events

Other events: 4 other events

Deaths: 1 deaths

Serious adverse events

Measure	Ranolazine n=14 participants at risk Ranolazine 500mg by mouth twice per day and after two weeks increase to 1000mg by mouth twice per day Ranolazine: Ranolazine 500mg by mouth twice per day and after two weeks increases to 1000mg by mouth twice per day and continue for a total of 26 weeks.	Placebo n=8 participants at risk Placebo by mouth twice per day Placebo: Placebo by mouth twice per day for a total of 26 weeks
Cardiac disorders heart failure	21.4% 3/14 • Number of events 3 • 7 months including 6 months of drug treatment and 1 month post treatment	25.0% 2/8 • Number of events 2 • 7 months including 6 months of drug treatment and 1 month post treatment

Other adverse events

Measure	Ranolazine n=14 participants at risk Ranolazine 500mg by mouth twice per day and after two weeks increase to 1000mg by mouth twice per day Ranolazine: Ranolazine 500mg by mouth twice per day and after two weeks increases to 1000mg by mouth twice per day and continue for a total of 26 weeks.	Placebo n=8 participants at risk Placebo by mouth twice per day Placebo: Placebo by mouth twice per day for a total of 26 weeks
Gastrointestinal disorders nausea	14.3% 2/14 • Number of events 2 • 7 months including 6 months of drug treatment and 1 month post treatment	0.00% 0/8 • 7 months including 6 months of drug treatment and 1 month post treatment
Renal and urinary disorders dark urine	7.1% 1/14 • Number of events 1 • 7 months including 6 months of drug treatment and 1 month post treatment	0.00% 0/8 • 7 months including 6 months of drug treatment and 1 month post treatment
Gastrointestinal disorders dyspepsia	0.00% 0/14 • 7 months including 6 months of drug treatment and 1 month post treatment	12.5% 1/8 • Number of events 1 • 7 months including 6 months of drug treatment and 1 month post treatment
Musculoskeletal and connective tissue disorders leg weakness	7.1% 1/14 • Number of events 1 • 7 months including 6 months of drug treatment and 1 month post treatment	0.00% 0/8 • 7 months including 6 months of drug treatment and 1 month post treatment
Gastrointestinal disorders loss of appetite	7.1% 1/14 • Number of events 1 • 7 months including 6 months of drug treatment and 1 month post treatment	0.00% 0/8 • 7 months including 6 months of drug treatment and 1 month post treatment
Skin and subcutaneous tissue disorders hair loss	0.00% 0/14 • 7 months including 6 months of drug treatment and 1 month post treatment	12.5% 1/8 • Number of events 1 • 7 months including 6 months of drug treatment and 1 month post treatment
Musculoskeletal and connective tissue disorders muscle spasm	7.1% 1/14 • Number of events 1 • 7 months including 6 months of drug treatment and 1 month post treatment	0.00% 0/8 • 7 months including 6 months of drug treatment and 1 month post treatment
Nervous system disorders restless sleep	7.1% 1/14 • Number of events 1 • 7 months including 6 months of drug treatment and 1 month post treatment	0.00% 0/8 • 7 months including 6 months of drug treatment and 1 month post treatment
Respiratory, thoracic and mediastinal disorders dryness on CPAP	0.00% 0/14 • 7 months including 6 months of drug treatment and 1 month post treatment	12.5% 1/8 • Number of events 1 • 7 months including 6 months of drug treatment and 1 month post treatment
Musculoskeletal and connective tissue disorders TENOSYNOVITIS,	7.1% 1/14 • Number of events 1 • 7 months including 6 months of drug treatment and 1 month post treatment	0.00% 0/8 • 7 months including 6 months of drug treatment and 1 month post treatment
Respiratory, thoracic and mediastinal disorders cold	0.00% 0/14 • 7 months including 6 months of drug treatment and 1 month post treatment	12.5% 1/8 • Number of events 1 • 7 months including 6 months of drug treatment and 1 month post treatment
General disorders fatique	7.1% 1/14 • Number of events 1 • 7 months including 6 months of drug treatment and 1 month post treatment	0.00% 0/8 • 7 months including 6 months of drug treatment and 1 month post treatment
Gastrointestinal disorders bloating	7.1% 1/14 • Number of events 1 • 7 months including 6 months of drug treatment and 1 month post treatment	0.00% 0/8 • 7 months including 6 months of drug treatment and 1 month post treatment
Gastrointestinal disorders dry mouth	7.1% 1/14 • Number of events 1 • 7 months including 6 months of drug treatment and 1 month post treatment	0.00% 0/8 • 7 months including 6 months of drug treatment and 1 month post treatment

Additional Information

Yuchi Han, PI of the study

University of Pennsylvania

Phone: 215-615-3417

Email: yuchi.han@uphs.upenn.edu

Results disclosure agreements

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