Open-Label Study of Perhexiline in Patients With Hypertrophic Cardiomyopathy and Moderate to Severe Heart Failure
NCT ID: NCT02862600
Last Updated: 2017-08-31
Study Results
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View full resultsBasic Information
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TERMINATED
PHASE2
35 participants
INTERVENTIONAL
2016-08-01
2017-05-22
Brief Summary
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Detailed Description
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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.
Conditions
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Keywords
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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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.
Perhexiline
Period 1 (Weeks 1-8) and Period 2 (Weeks 9-16): dose titrated to two different plasma levels of perhexiline
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.
Interventions
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Perhexiline
Period 1 (Weeks 1-8) and Period 2 (Weeks 9-16): dose titrated to two different plasma levels of perhexiline
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.
Eligibility Criteria
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Inclusion Criteria
* 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
Exclusion Criteria
* 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
18 Years
ALL
No
Sponsors
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Heart Metabolics Limited
INDUSTRY
Responsible Party
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Principal Investigators
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Mark Midei, MD
Role: STUDY_CHAIR
Heart Metabolics
Locations
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Stanford, California, United States
Indianapolis, Indiana, United States
Johns Hopkins
Baltimore, Maryland, United States
University of Maryland
Baltimore, Maryland, United States
Detroit, Michigan, United States
Columbus, Ohio, United States
Portland, Oregon, United States
Hershey, Pennsylvania, United States
Germantown, Tennessee, United States
Salt Lake City, Utah, United States
Madison, Wisconsin, United States
Countries
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Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Document Type: Informed Consent Form
Other Identifiers
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HML-PHX-005
Identifier Type: -
Identifier Source: org_study_id