Open-Label Study of Perhexiline in Patients With Hypertrophic Cardiomyopathy and Moderate to Severe Heart Failure
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ClinicalTrials.gov Identifier: NCT02862600 |
Recruitment Status :
Terminated
(Lack of Efficacy)
First Posted : August 11, 2016
Results First Posted : August 31, 2017
Last Update Posted : August 31, 2017
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Condition or disease | Intervention/treatment | Phase |
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Cardiomyopathy, Hypertrophic Cardiomyopathy, Hypertrophic, Familial | Drug: Perhexiline Device: Use of bioanalytical assay to monitor plasma levels of perhexiline | Phase 2 |
Patients with hypertrophic cardiomyopathy and symptoms without severe outflow obstruction will be eligible to participate. Enrollment will be limited to subjects who are unable to attain 75% of their maximum predicted MVO2 at cardiopulmonary exercise testing. Subjects with genetic evidence of CYP2D6 poor metabolizer status will be excluded.
Subjects will undergo functional testing at baseline with CPEX testing and 6 minute walk distance testing. They will begin perhexiline orally, and the dose will be adjusted according to plasma level testing. For the first 8 week period, the target therapeutic range will be 100-300 ng/mL, and for the second 8 week period, the range will be 300-500 ng/mL. Functional testing will be repeated at the end of both periods.
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 35 participants |
Allocation: | N/A |
Intervention Model: | Single Group Assignment |
Intervention Model Description: | Open-label, 2 period, dose escalation study of perhexiline in symptomatic patients with hypertrophic cardiomyopathy |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | A Phase 2, Multi-Center, Open-Label, Ascending Dose Study on the Efficacy, Safety and Tolerability of Perhexiline in Patients With Hypertrophic Cardiomyopathy and Moderate to Severe Heart Failure With Preserved Left Ventricular Function |
Actual Study Start Date : | August 1, 2016 |
Actual Primary Completion Date : | April 28, 2017 |
Actual Study Completion Date : | May 22, 2017 |
Arm | Intervention/treatment |
---|---|
Experimental: Perhexiline
Perhexiline will be administered orally. Dosing will be determined based on plasma level monitoring. For the first 8 week period, the target range will be 100-300 ng/mL, for the second 8 week period, the target range will be 300-500 ng/mL.
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Drug: Perhexiline
Period 1 (Weeks 1-8) and Period 2 (Weeks 9-16): dose titrated to two different plasma levels of perhexiline Device: Use of bioanalytical assay to monitor plasma levels of perhexiline The bioanalytical assay is the device under investigation. It will be used to monitor plasma levels of perhexiline. The data obtained from this analysis will be used to guide dose adjustments of perhexiline. |
- Change From Baseline of VO2MAX at 16 Weeks [ Time Frame: end of Period 2 (Week 16) ]At the conclusion of 16 weeks of perhexiline treatment, MVO2 was measured using CPEX and compared to MVO2 measured at baseline.
- Change From Baseline of VO2MAX at End of Period 1 [ Time Frame: end of Period 1 (Week 8) ]At the conclusion of 8 weeks of perhexiline treatment, MVO2 was measured using CPEX and compared to MVO2 measured at baseline.
- Change From Baseline in the Six-minute Walk Test at the End of Period 2 [ Time Frame: end of Period 2 (Week 16) ]At the conclusion of 16 weeks of perhexiline treatment, 6MWD was measured and compared to 6MWD measured at baseline.
- Change From Baseline in the Six-minute Walk Test at the End of Period 1 [ Time Frame: end of Period 1 (Week 8) ]At the conclusion of 8 weeks of perhexiline treatment, 6MWD was measured and compared to 6MWD measured at baseline.
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Key Inclusion Criteria:
- Hypertrophic cardiomyopathy with symptoms of moderate-to-severe heart failure
- Left ventricular hypertrophy with maximum LV wall thickness ≥ 15 mm
- Left ventricular ejection fraction ≥ 50%
- Able to perform exercise testing but unable to exceed 75% of the predicted age-adjusted maximum level
Key Exclusion Criteria:
- CYP2D6 Poor Metabolizer (PM) status
- History of a known chronic liver disease
- ALT, AST, alkaline phosphatase, or LDH > 1.5 x upper limit of normal
- Total Bilirubin > 2.0 x upper limit of normal
- Severe LV outflow obstruction
- Asymptomatic patients or cardiomyopathy-related criteria as per protocol
- QT interval related criteria as per protocol
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02862600
United States, California | |
Stanford, California, United States | |
United States, Indiana | |
Indianapolis, Indiana, United States | |
United States, Maryland | |
Johns Hopkins | |
Baltimore, Maryland, United States | |
University of Maryland | |
Baltimore, Maryland, United States | |
United States, Michigan | |
Detroit, Michigan, United States | |
United States, Ohio | |
Columbus, Ohio, United States | |
United States, Oregon | |
Portland, Oregon, United States | |
United States, Pennsylvania | |
Hershey, Pennsylvania, United States | |
United States, Tennessee | |
Germantown, Tennessee, United States | |
United States, Utah | |
Salt Lake City, Utah, United States | |
United States, Wisconsin | |
Madison, Wisconsin, United States |
Study Chair: | Mark Midei, MD | Heart Metabolics |
Documents provided by Heart Metabolics Limited:
Responsible Party: | Heart Metabolics Limited |
ClinicalTrials.gov Identifier: | NCT02862600 |
Other Study ID Numbers: |
HML-PHX-005 |
First Posted: | August 11, 2016 Key Record Dates |
Results First Posted: | August 31, 2017 |
Last Update Posted: | August 31, 2017 |
Last Verified: | August 2017 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | No |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | Yes |
Device Product Not Approved or Cleared by U.S. FDA: | Yes |
hypertrophic cardiomyopathy heart failure cardiopulmonary exercise testing 6 minute walk test perhexiline |
carnitine palmitoyltransferase mixed ion channel effects late sodium current inhibitor calcium channel inhibition |
Heart Failure Cardiomyopathies Cardiomyopathy, Hypertrophic Cardiomyopathy, Hypertrophic, Familial Hypertrophy Heart Diseases Cardiovascular Diseases Pathological Conditions, Anatomical Aortic Stenosis, Subvalvular Aortic Valve Stenosis |
Aortic Valve Disease Heart Valve Diseases Genetic Diseases, Inborn Perhexiline Calcium Channel Blockers Membrane Transport Modulators Molecular Mechanisms of Pharmacological Action Calcium-Regulating Hormones and Agents Physiological Effects of Drugs Vasodilator Agents |