Hypertrophic CardiomyopathyPhase 2/3LIBERTY-HCMNCT02291237
Effect of Eleclazine (GS-6615) on Exercise Capacity in Subjects With Symptomatic Hypertrophic Cardiomyopathy
A Phase 2/3 study testing Eleclazine in people with Hypertrophic Cardiomyopathy, run by Gilead Sciences at 46 sites in 8 countries.
Trial 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). Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Hypertrophic Cardiomyopathy
- What is being tested?
- Eleclazine
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 172 participants
- Where
- 46 sitesin 8 countries
- Study dates
- 5 Feb 2015to 17 Feb 2017
- Phase
- Phase 2/3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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)
Who cannot join
- 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.
Trial locations and contacts
46 sites in 8 countries.
46 sites in 8 countries
United States26 sites
Athens Regional Medical Center
Athens, Georgia
St. Luke's University Health Network
Bethlehem, Pennsylvania
Brigham & Women's Hospital and Harvard Medical School
Boston, Massachusetts
Massachusetts General Hospital
Boston, Massachusetts
Tufts Medical Center
Boston, Massachusetts
Northwestern Memorial Hospital
Chicago, Illinois
Duke Health Center at Southpoint
Durham, North Carolina
Houston Methodist Hospital
Houston, Texas
Texas Heart Institute
Houston, Texas
UT Southwestern Medical Center
Houston, Texas
University of Iowa Hospitals and Clinics
Iowa City, Iowa
Cedars-Sinai Heart Institute
Los Angeles, California
University of California Los Angeles
Los Angeles, California
Marshfield Clinic Research Institute
Marshfield, Wisconsin
Medical College of Wisconsin
Milwaukee, Wisconsin
Morristown Medical Center
Morristown, New Jersey
St. Thomas Research Institute
Nashville, Tennessee
Vanderbilt University Medical Center
Nashville, Tennessee
Yale New Haven Hospital
New Haven, Connecticut
Columbia University Medical Center/ New York Presbyterian
New York, New York
NYU School of Medicine Pediatrics
New York, New York
University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania
Oregon Health and Science University
Portland, Oregon
University of Washington
Seattle, Washington
Washington University School of Medicine
St Louis, Missouri
Stanford University
Stanford, California
Italy8 sites
Azienda Ospedaliera Papa Giovanni XXIII
Bergamo
Azienda Ospedaliero Universitaria Di Bologna
Bologna
Azienda Ospedaliera Universitaria Careggi
Florence
Ospedale San Raffaele S.r.l.
Milan
Azienda Ospedaliera Monaldi
Naples
Azienda Ospedaliero Universitaria di Parma
Parma
Azienda Ospedaliera San Camillo Forlanini
Rome
Madonna del Soccorso Hospital
San Benedetto del Tronto, Ascoli Piceno
Israel4 sites
Ein Kerem-Hadassah Medical Organization
Jerusalem
Rabin Medical Center
Petah Tikva
Sheba Medical Center
Ramat Gan
Tel Aviv Sourasky Medical Center
Tel Aviv
Netherlands3 sites
Academisch Medisch Centrum Amsterdam
Amsterdam, North Holland
Erasmus MC
Rotterdam
University Medical Center Utrecht
Utrecht
United Kingdom2 sites
University Hospital of Wales
Cardiff, South Glamergon
Northern General Hospital
Sheffield, Yorkshire
About this study
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
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).
- Conditions
- Hypertrophic Cardiomyopathy
- Being tested
- Eleclazine, Placebo
- 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
- Medical subject headings
- Cardiomyopathy, Hypertrophic, Cardiomyopathies, Hypertrophy, Cardiomyopathy, Hypertrophic, Familial, Heart Failure, Angina Pectoris, Dyspnea
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EleclazineDrug
Also called GS-6615
Tablet (s) administered orally once daily
PlaceboDrug
Placebo to match eleclazine administered orally once daily
| Study group | Role | What they receive |
|---|---|---|
| Eleclazine | Experimental | 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. Receives: Drug: Eleclazine |
| Placebo | Experimental | Placebo to match eleclazine until at least Week 24, followed by active eleclazine 6 mg in an open-label extension period. Receives: Drug: Eleclazine; Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Peak Oxygen Uptake (VO2) Achieved During Cardiopulmonary Exercise Testing (CPET) From Baseline to Week 24
Measured: Baseline to Week 24
Secondary outcomes (5)
Change in Peak Oxygen Uptake (VO2) Achieved During Cardiopulmonary Exercise Testing (CPET) From Baseline to Week 12
Measured: 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.
Measured: Baseline to Week 24
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.
Measured: Baseline to Week 12
Change in Treadmill Exercise Time From Baseline to Week 24
Treadmill exercise time is the time to peak exercise.
Measured: Baseline to Week 24
Change in Treadmill Exercise Time From Baseline to Week 12
Treadmill exercise time is the time to peak exercise.
Measured: Baseline to Week 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Nov 2014 (estimated)
- Study start5 Feb 2015
- Primary completion20 Jan 2017
- Study completion17 Feb 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Hypertrophic CardiomyopathyStudies of the same condition, open and under way.
- More trials run by Gilead SciencesThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 172 participants (actual)
Sponsor and investigators
- Lead sponsor
- Gilead Sciences
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Gilead Study Director, Study director, Gilead Sciences
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02291237
- Sponsor's protocol ID
- GS-US-361-1157
- Other IDs
- 2013-004429-97
- Acronym
- LIBERTY-HCM
- Status last verified
- Feb 2018
- First submitted
- 11 Nov 2014
- Last updated
- 24 Sep 2018
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
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.
PubMed 26915375
Study at a glance
From the registry record, last updated by the sponsor on 24 Sep 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT02291237
- Acronym / sponsor ID
- LIBERTY-HCM / GS-US-361-1157
- Conditions
- Hypertrophic Cardiomyopathy
- Keywords
- hypertrophic cardiomyopathy, hcm, hocm, hypertrophic myocardiopathy, hypertrophic obstructive cardiomyopathies, cardiomyopathy, hypertrophic, familial hypertrophic cardiomyopathy
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Terminated
- Enrolment
- 172 participants (actual)
- Lead sponsor
- Gilead SciencesIndustry (pharma, biotech, devices)
- Study start
- 5 Feb 2015 (actual)
- Primary completion
- 20 Jan 2017 (actual)
- Study completion
- 17 Feb 2017 (actual)
- First posted
- 14 Nov 2014 (estimated)
- Results
- Posted 22 Mar 2018
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Eleclazine | Experimental | Drug: EleclazineEleclazine 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 | Experimental | Drug: Eleclazine, Drug: PlaceboPlacebo to match eleclazine until at least Week 24, followed by active eleclazine 6 mg in an open-label extension period. |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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:
- 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 (5 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| EleclazineAlso: GS-6615Tablet (s) administered orally once daily | Drug | Eleclazine, Placebo |
| PlaceboPlacebo to match eleclazine administered orally once daily | Drug | Placebo |
Primary outcome
Change in Peak Oxygen Uptake (VO2) Achieved During Cardiopulmonary Exercise Testing (CPET) From Baseline to Week 24
Time frame: Baseline to Week 24
5 secondary outcomes
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
Change in Minnesota Living With Heart Failure Questionnaire (MLHFQ) From Baseline to Week 12
Time frame: Baseline to Week 12
Change in Treadmill Exercise Time From Baseline to Week 24
Time frame: Baseline to Week 24
Change in Treadmill Exercise Time From Baseline to Week 12
Time frame: Baseline to Week 12
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Sep 2018.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 26915375Olivotto 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Gilead Study Director study director
Gilead Sciences
46 sites in 8 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Eleclazine in people with Hypertrophic Cardiomyopathy, run by Gilead Sciences at 46 sites in 8 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 24 Sep 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 46 sites in United States, Italy, Israel, Netherlands, United Kingdom and 3 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 22 Mar 2018). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in Peak Oxygen Uptake (VO2) Achieved During Cardiopulmonary Exercise Testing (CPET) From Baseline to Week 24 (time frame: Baseline to Week 24). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02291237, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Sep 2018.