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

Primary Purpose

Hypertrophic Cardiomyopathy

Status
Terminated
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
Eleclazine
Placebo
Sponsored by
Gilead Sciences
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Hypertrophic Cardiomyopathy focused on measuring hypertrophic cardiomyopathy, hcm, hocm, hypertrophic myocardiopathy, hypertrophic obstructive cardiomyopathies, cardiomyopathy, hypertrophic, familial hypertrophic cardiomyopathy, genetic heart disease, echocardiography, cardiopulmonary exercise testing, exercise Capacity, heart failure, angina, dyspnea, diastolic dysfunction, microvascular ischemia, late sodium current inhibitor, GS-6615, late INA

Eligibility Criteria

18 Years - 65 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • Cedars-Sinai Heart Institute
  • University of California Los Angeles
  • Stanford University
  • Yale New Haven Hospital
  • Athens Regional Medical Center
  • Northwestern Memorial Hospital
  • University of Iowa Hospitals and Clinics
  • Brigham & Women's Hospital and Harvard Medical School
  • Massachusetts General Hospital
  • Tufts Medical Center
  • Washington University School of Medicine
  • Morristown Medical Center
  • Columbia University Medical Center/ New York Presbyterian
  • NYU School of Medicine Pediatrics
  • Duke Health Center at Southpoint
  • Oregon Health and Science University
  • St. Luke's University Health Network
  • University of Pittsburgh Medical Center
  • St. Thomas Research Institute
  • Vanderbilt University Medical Center
  • Houston Methodist Hospital
  • Texas Heart Institute
  • UT Southwestern Medical Center
  • University of Washington
  • Marshfield Clinic Research Institute
  • Medical College of Wisconsin
  • The Alfred Hospital
  • Hôpital Européen Georges Pompidou
  • Universitätsklinikum Hamburg Eppendorf
  • Ein Kerem-Hadassah Medical Organization
  • Rabin Medical Center
  • Sheba Medical Center
  • Tel Aviv Sourasky Medical Center
  • Madonna del Soccorso Hospital
  • Azienda Ospedaliera Papa Giovanni XXIII
  • Azienda Ospedaliero Universitaria Di Bologna
  • Azienda Ospedaliera Universitaria Careggi
  • Ospedale San Raffaele S.r.l.
  • Azienda Ospedaliera Monaldi
  • Azienda Ospedaliero Universitaria di Parma
  • Azienda Ospedaliera San Camillo Forlanini
  • Academisch Medisch Centrum Amsterdam
  • Erasmus MC
  • University Medical Center Utrecht
  • University Hospital of Wales
  • Northern General Hospital

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Experimental

Arm Label

Eleclazine

Placebo

Arm Description

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.

Placebo to match eleclazine until at least Week 24, followed by active eleclazine 6 mg in an open-label extension period.

Outcomes

Primary Outcome Measures

Change in Peak Oxygen Uptake (VO2) Achieved During Cardiopulmonary Exercise Testing (CPET) From Baseline to Week 24

Secondary Outcome Measures

Change in Peak Oxygen Uptake (VO2) Achieved During Cardiopulmonary Exercise Testing (CPET) From Baseline to Week 12
Change in Minnesota Living With Heart Failure Questionnaire (MLHFQ) From 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
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
Treadmill exercise time is the time to peak exercise.
Change in Treadmill Exercise Time From Baseline to Week 12
Treadmill exercise time is the time to peak exercise.

Full Information

First Posted
November 11, 2014
Last Updated
August 24, 2018
Sponsor
Gilead Sciences
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1. Study Identification

Unique Protocol Identification Number
NCT02291237
Brief Title
Effect of Eleclazine (GS-6615) on Exercise Capacity in Subjects With Symptomatic Hypertrophic Cardiomyopathy
Acronym
LIBERTY-HCM
Official Title
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
Study Type
Interventional

2. Study Status

Record Verification Date
February 2018
Overall Recruitment Status
Terminated
Study Start Date
February 5, 2015 (Actual)
Primary Completion Date
January 20, 2017 (Actual)
Study Completion Date
February 17, 2017 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Gilead Sciences

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

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).

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hypertrophic Cardiomyopathy
Keywords
hypertrophic cardiomyopathy, hcm, hocm, hypertrophic myocardiopathy, hypertrophic obstructive cardiomyopathies, cardiomyopathy, hypertrophic, familial hypertrophic cardiomyopathy, genetic heart disease, echocardiography, cardiopulmonary exercise testing, exercise Capacity, heart failure, angina, dyspnea, diastolic dysfunction, microvascular ischemia, late sodium current inhibitor, GS-6615, late INA

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2, Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
172 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Eleclazine
Arm Type
Experimental
Arm Description
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.
Arm Title
Placebo
Arm Type
Experimental
Arm Description
Placebo to match eleclazine until at least Week 24, followed by active eleclazine 6 mg in an open-label extension period.
Intervention Type
Drug
Intervention Name(s)
Eleclazine
Other Intervention Name(s)
GS-6615
Intervention Description
Tablet (s) administered orally once daily
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Placebo to match eleclazine administered orally once daily
Primary Outcome Measure Information:
Title
Change in Peak Oxygen Uptake (VO2) Achieved During Cardiopulmonary Exercise Testing (CPET) From Baseline to Week 24
Time Frame
Baseline to Week 24
Secondary Outcome Measure Information:
Title
Change in Peak Oxygen Uptake (VO2) Achieved During Cardiopulmonary Exercise Testing (CPET) From Baseline to Week 12
Time Frame
Baseline to Week 12
Title
Change in Minnesota Living With Heart Failure Questionnaire (MLHFQ) From Baseline to Week 24
Description
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.
Time Frame
Baseline to Week 24
Title
Change in Minnesota Living With Heart Failure Questionnaire (MLHFQ) From Baseline to Week 12
Description
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.
Time Frame
Baseline to Week 12
Title
Change in Treadmill Exercise Time From Baseline to Week 24
Description
Treadmill exercise time is the time to peak exercise.
Time Frame
Baseline to Week 24
Title
Change in Treadmill Exercise Time From Baseline to Week 12
Description
Treadmill exercise time is the time to peak exercise.
Time Frame
Baseline to Week 12

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Gilead Study Director
Organizational Affiliation
Gilead Sciences
Official's Role
Study Director
Facility Information:
Facility Name
Cedars-Sinai Heart Institute
City
Los Angeles
State/Province
California
Country
United States
Facility Name
University of California Los Angeles
City
Los Angeles
State/Province
California
Country
United States
Facility Name
Stanford University
City
Stanford
State/Province
California
Country
United States
Facility Name
Yale New Haven Hospital
City
New Haven
State/Province
Connecticut
Country
United States
Facility Name
Athens Regional Medical Center
City
Athens
State/Province
Georgia
Country
United States
Facility Name
Northwestern Memorial Hospital
City
Chicago
State/Province
Illinois
Country
United States
Facility Name
University of Iowa Hospitals and Clinics
City
Iowa City
State/Province
Iowa
Country
United States
Facility Name
Brigham & Women's Hospital and Harvard Medical School
City
Boston
State/Province
Massachusetts
Country
United States
Facility Name
Massachusetts General Hospital
City
Boston
State/Province
Massachusetts
Country
United States
Facility Name
Tufts Medical Center
City
Boston
State/Province
Massachusetts
Country
United States
Facility Name
Washington University School of Medicine
City
Saint Louis
State/Province
Missouri
Country
United States
Facility Name
Morristown Medical Center
City
Morristown
State/Province
New Jersey
Country
United States
Facility Name
Columbia University Medical Center/ New York Presbyterian
City
New York
State/Province
New York
Country
United States
Facility Name
NYU School of Medicine Pediatrics
City
New York
State/Province
New York
Country
United States
Facility Name
Duke Health Center at Southpoint
City
Durham
State/Province
North Carolina
Country
United States
Facility Name
Oregon Health and Science University
City
Portland
State/Province
Oregon
Country
United States
Facility Name
St. Luke's University Health Network
City
Bethlehem
State/Province
Pennsylvania
Country
United States
Facility Name
University of Pittsburgh Medical Center
City
Pittsburgh
State/Province
Pennsylvania
Country
United States
Facility Name
St. Thomas Research Institute
City
Nashville
State/Province
Tennessee
Country
United States
Facility Name
Vanderbilt University Medical Center
City
Nashville
State/Province
Tennessee
Country
United States
Facility Name
Houston Methodist Hospital
City
Houston
State/Province
Texas
Country
United States
Facility Name
Texas Heart Institute
City
Houston
State/Province
Texas
Country
United States
Facility Name
UT Southwestern Medical Center
City
Houston
State/Province
Texas
Country
United States
Facility Name
University of Washington
City
Seattle
State/Province
Washington
Country
United States
Facility Name
Marshfield Clinic Research Institute
City
Marshfield
State/Province
Wisconsin
Country
United States
Facility Name
Medical College of Wisconsin
City
Milwaukee
State/Province
Wisconsin
Country
United States
Facility Name
The Alfred Hospital
City
Melbourne
State/Province
Victoria
Country
Australia
Facility Name
Hôpital Européen Georges Pompidou
City
Paris
Country
France
Facility Name
Universitätsklinikum Hamburg Eppendorf
City
Hamburg
Country
Germany
Facility Name
Ein Kerem-Hadassah Medical Organization
City
Jerusalem
Country
Israel
Facility Name
Rabin Medical Center
City
Petah Tikva
Country
Israel
Facility Name
Sheba Medical Center
City
Ramat-Gan
Country
Israel
Facility Name
Tel Aviv Sourasky Medical Center
City
Tel Aviv
Country
Israel
Facility Name
Madonna del Soccorso Hospital
City
San Benedetto del Tronto
State/Province
Ascoli Piceno
Country
Italy
Facility Name
Azienda Ospedaliera Papa Giovanni XXIII
City
Bergamo
Country
Italy
Facility Name
Azienda Ospedaliero Universitaria Di Bologna
City
Bologna
Country
Italy
Facility Name
Azienda Ospedaliera Universitaria Careggi
City
Florence
Country
Italy
Facility Name
Ospedale San Raffaele S.r.l.
City
Milan
Country
Italy
Facility Name
Azienda Ospedaliera Monaldi
City
Naples
Country
Italy
Facility Name
Azienda Ospedaliero Universitaria di Parma
City
Parma
Country
Italy
Facility Name
Azienda Ospedaliera San Camillo Forlanini
City
Rome
Country
Italy
Facility Name
Academisch Medisch Centrum Amsterdam
City
Amsterdam
State/Province
Noord-Holland
Country
Netherlands
Facility Name
Erasmus MC
City
Rotterdam
Country
Netherlands
Facility Name
University Medical Center Utrecht
City
Utrecht
Country
Netherlands
Facility Name
University Hospital of Wales
City
Cardiff
State/Province
South Glamergon
Country
United Kingdom
Facility Name
Northern General Hospital
City
Sheffield
State/Province
Yorkshire
Country
United Kingdom

12. IPD Sharing Statement

Citations:
PubMed Identifier
26915375
Citation
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.
Results Reference
background

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

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