Effect of Eleclazine (GS-6615) on Exercise Capacity in Subjects With Symptomatic Hypertrophic Cardiomyopathy (LIBERTY-HCM)

Tracking Information
First Submitted Date ICMJE	November 11, 2014
First Posted Date ICMJE	November 14, 2014
Results First Submitted Date	January 19, 2018
Results First Posted Date	March 22, 2018
Last Update Posted Date	September 24, 2018
Actual Study Start Date ICMJE	February 5, 2015
Actual Primary Completion Date	January 20, 2017 (Final data collection date for primary outcome measure)
Current Primary Outcome Measures ICMJE; (submitted: February 23, 2018)	Change in Peak Oxygen Uptake (VO2) Achieved During Cardiopulmonary Exercise Testing (CPET) From Baseline to Week 24 [ Time Frame: Baseline to Week 24 ]
Original Primary Outcome Measures ICMJE; (submitted: November 11, 2014)	Change in peak oxygen uptake (VO2) from baseline (screening) to Week 24 [ Time Frame: Up to 24 weeks ]
Change History	Complete list of historical versions of study NCT02291237 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures ICMJE; (submitted: February 23, 2018)	Change in Peak Oxygen Uptake (VO2) Achieved During Cardiopulmonary Exercise Testing (CPET) From Baseline to Week 12 [ Time Frame: Baseline to Week 12 ]; Change in Minnesota Living With Heart Failure Questionnaire (MLHFQ) From Baseline to Week 24 [ Time Frame: Baseline to Week 24 ]The MLHFQ is a 21-item quality of life (QoL) questionnaire that measures the effects of symptoms, functional limitations, and psychological distress on an individual. Each item is measured on a 6-point Likert scale (0 to 5) and is scored by summing the responses to all 21 questions. Scores range from 0 to 105, with lower scores indicating a better quality of life.; Change in Minnesota Living With Heart Failure Questionnaire (MLHFQ) From Baseline to Week 12 [ Time Frame: Baseline to Week 12 ]The MLHFQ is a 21-item quality of life (QoL) questionnaire that measures the effects of symptoms, functional limitations, and psychological distress on an individual. Each item is measured on a 6-point Likert scale (0 to 5) and is scored by summing the responses to all 21 questions. Scores range from 0 to 105, with lower scores indicating a better quality of life.; Change in Treadmill Exercise Time From Baseline to Week 24 [ Time Frame: Baseline to Week 24 ]Treadmill exercise time is the time to peak exercise.; Change in Treadmill Exercise Time From Baseline to Week 12 [ Time Frame: Baseline to Week 12 ]Treadmill exercise time is the time to peak exercise.
Original Secondary Outcome Measures ICMJE; (submitted: November 11, 2014)	Change in Minnesota Living With Heart Failure Questionnaire (MLHFQ) From Baseline to Week 24 [ Time Frame: Up to 24 weeks ]; Change in Treadmill Exercise Time From Baseline to Week 24 [ Time Frame: Up to 24 weeks ]; Change in Peak VO2 from baseline to Week 12 [ Time Frame: Up to 12 weeks ]; Change in the MLHFQ from baseline to Week 12 [ Time Frame: Up to 12 weeks ]; Change in Treadmill Exercise Time From Baseline to Week 12 [ Time Frame: Up to 12 weeks ]
Current Other Outcome Measures ICMJE	Not Provided
Original Other Outcome Measures ICMJE	Not Provided
Descriptive Information
Brief Title ICMJE	Effect of Eleclazine (GS-6615) on Exercise Capacity in Subjects With Symptomatic Hypertrophic Cardiomyopathy
Official Title ICMJE	Study Title: A Phase 2/3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Effect of GS-6615 on Exercise Capacity in Subjects With Symptomatic Hypertrophic Cardiomyopathy
Brief Summary	The primary objective of this study was to evaluate the effect of eleclazine (GS-6615) on exercise capacity as measured by Peak oxygen uptake (VO2) achieved during cardiopulmonary exercise testing (CPET), in participants with symptomatic hypertrophic cardiomyopathy (HCM).
Detailed Description	Not Provided
Study Type ICMJE	Interventional
Study Phase	Phase 2; Phase 3
Study Design ICMJE	Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor); Primary Purpose: Treatment
Condition ICMJE	Hypertrophic Cardiomyopathy
Intervention ICMJE	Drug: Eleclazine Tablet (s) administered orally once daily Other Name: GS-6615; Drug: Placebo Placebo to match eleclazine administered orally once daily
Study Arms	Experimental: Eleclazine Eleclazine 30 mg single loading dose followed by 3 mg daily maintenance dose up until Week 12, then 6 mg daily maintenance dose from Week 12 at least Week 24, followed by eleclazine 6 mg in an open-label extension period. Intervention: Drug: Eleclazine; Experimental: Placebo Placebo to match eleclazine until at least Week 24, followed by active eleclazine 6 mg in an open-label extension period. Interventions: Drug: Eleclazine Drug: Placebo
Publications *	Olivotto I, Hellawell JL, Farzaneh-Far R, Blair C, Coppini R, Myers J, Belardinelli L, Maron MS. Novel Approach Targeting the Complex Pathophysiology of Hypertrophic Cardiomyopathy: The Impact of Late Sodium Current Inhibition on Exercise Capacity in Subjects with Symptomatic Hypertrophic Cardiomyopathy (LIBERTY-HCM) Trial. Circ Heart Fail. 2016 Mar;9(3):e002764. doi: 10.1161/CIRCHEARTFAILURE.115.002764.
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
Recruitment Information
Recruitment Status ICMJE	Terminated
Actual Enrollment ICMJE; (submitted: December 23, 2016)	172
Original Estimated Enrollment ICMJE; (submitted: November 11, 2014)	180
Actual Study Completion Date	February 17, 2017
Actual Primary Completion Date	January 20, 2017 (Final data collection date for primary outcome measure)
Eligibility Criteria ICMJE	Key Inclusion Criteria: Established diagnosis of hypertrophic cardiomyopathy defined by standard criteria as a maximal left ventricular wall thickness ≥ 15 mm at initial diagnosis; Exertional symptoms including at least one of the following: New York Heart Association (NYHA) Class ≥ II dyspnea Canadian Cardiovascular Society (CCS) Class ≥ II angina; Screening (baseline) peak VO2 < 80% of predicted for age, sex, and weight; Ability to perform an upright treadmill cardiopulmonary exercise test (CPET) Key Exclusion Criteria: Known aortic valve stenosis (moderate or severe); Known coronary artery disease; Left ventricular systolic dysfunction (ejection fraction < 50%); Recent septal reduction procedure (ie, surgical myectomy or alcohol septal ablation) within six months prior to screening or such a procedure scheduled to occur during the study Note: Other protocol defined Inclusion/Exclusion criteria may apply.
Sex/Gender	Sexes Eligible for Study: All
Ages	18 Years to 65 Years (Adult, Older Adult)
Accepts Healthy Volunteers	No
Contacts ICMJE	Contact information is only displayed when the study is recruiting subjects
Listed Location Countries ICMJE	Australia, France, Germany, Israel, Italy, Netherlands, United Kingdom, United States
Removed Location Countries
Administrative Information
NCT Number ICMJE	NCT02291237
Other Study ID Numbers ICMJE	GS-US-361-1157; 2013-004429-97 ( EudraCT Number )
Has Data Monitoring Committee	Yes
U.S. FDA-regulated Product	Studies a U.S. FDA-regulated Drug Product: Yes Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement	Not Provided
Responsible Party	Gilead Sciences
Study Sponsor ICMJE	Gilead Sciences
Collaborators ICMJE	Not Provided
Investigators ICMJE	Study Director: Gilead Study Director Gilead Sciences
PRS Account	Gilead Sciences
Verification Date	February 2018
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP