Efficacy, Safety, and Tolerability of Perhexiline in Subjects With Hypertrophic Cardiomyopathy and Heart Failure
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|ClinicalTrials.gov Identifier: NCT02431221|
Recruitment Status : Withdrawn (Lack of efficacy in a preceding study.)
First Posted : April 30, 2015
Last Update Posted : July 14, 2017
Heart Metabolics Limited
Information provided by (Responsible Party):
Heart Metabolics Limited
A Study on the Efficacy, Safety, and Tolerability of Perhexiline maleate in Subjects with Hypertrophic Cardiomyopathy and Moderate-To-Severe Heart Failure
|Condition or disease||Intervention/treatment||Phase|
|Hypertrophic Cardiomyopathy||Drug: Perhexiline Drug: Placebo||Phase 3|
This study is an international, multi-center, randomized, double-blind, placebo controlled clinical trial of the effects of perhexiline maleate on a rank-ordered, hierarchical variable consisting of outcome and functional measures in 350 subjects with hypertrophic cardiomyopathy and symptoms of moderate to severe congestive heart failure. The study is designed in four parts: 1) an open-label run-in period of 2 weeks, 2) A blinded, randomized phase of variable duration (anticipated between 6 months and 18 months depending on enrollment rate), 3) an open label period of 3 months, and 4) a randomized withdrawal period of 3 months. Subjects who do not experience an adverse outcome (death or resuscitated ventricular arrhythmia, stroke, need for surgical intervention, cardiovascular hospitalization, occurrence of atrial fibrillation, or study dropout) will undergo cardiopulmonary exercise testing in order to determine change from baseline in maximum oxygen consumption.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||0 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|Official Title:||A Study on the Efficacy, Safety, and Tolerability of Perhexiline Maleate in Subjects With Hypertrophic Cardiomyopathy and Moderate-To-Severe Heart Failure|
|Actual Primary Completion Date :||July 2017|
Perhexiline maleate will be supplied in 100 mg and 25 mg tablets. It will be initially administered at a dose of 200 mg once a day in tablet form for at least six months. After initiation of dosing, perhexiline dosing will be determined using plasma level guided dose adjustment. Dosing decisions will be made by an unblinded dose control center.
perhexiline maleate, oral, 25mg or 100mg tablets Whenever initiating PHM therapy, subjects will receive a loading regimen of PHM as indicated in in the Heart Metabolics Dose Justification document. Drug levels will be measured first on Day 4 +/- 1 day, with an initial dose adjustment performed on Day 7 +/- 1 day, as needed. The next sampling will occur on Day 21 +/- 1 day for potential dose adjustment, as advised by the DCC, on or about Day 24. Subsequent dosing will be monitored and advised by a Dose Control Center
Placebo Comparator: Placebo
Placebo will be administered once a day in tablet form for at least six months. Tablets will be identical in size and shape to perhexiline tablets. Adjustments in placebo dosing will be made by an unblinded dose control center to mimic decisions made for subjects in the experimental arm.
Primary Outcome Measures :
- Subject Outcome Variable [ Time Frame: 6 months after randomization ]The subject Outcome variable is a rank-ordered, hierarchical classification of outcome variables followed by a rank ordering of change from baseline in Maximum oxygen consumption
Secondary Outcome Measures :
- Functional Assessment During Randomized Withdrawal Phase [ Time Frame: 6 months after initiation of open label period, and 3 months after randomized withdrawal period initiated ]Change of CPEX-VO2max from the beginning of the Randomized Withdrawal phase to the end of the Randomized Withdrawal phase (Part 4). The change in CPEX-VO2max in the placebo group will be compared to the change in CPEX-VO2max in the continued perhexiline (non-withdrawal) group.
- Functional Assessment During Randomized Phase [ Time Frame: 6 months after Randomization ]Change in six-minute walk test (6MWT) from baseline (pre-Part 1 run-in, prior to receiving any perhexiline) to 6 months after randomization during the Randomized Treatment (Part 2)
- Functional Assessment During Randomized Withdrawal Phase [ Time Frame: 6 months after initiation of open label period, and 3 months after randomized withdrawal period initiated ]Change in six-minute walk test (6MWT) from the beginning of the Randomized Withdrawal (Part 4) to the end of the Randomized Withdrawal (Part 4)
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