Open-Label Study of Perhexiline in Patients With Hypertrophic Cardiomyopathy and Moderate to Severe Heart Failure

August 2, 2017 updated by: Heart Metabolics Limited

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

The purpose of this study is to evaluate the effect of perhexiline on exercise performance (efficacy) and safety in patients with hypertrophic cardiomyopathy and moderate-to-severe heart failure following dosing for 16 weeks.

Study Overview

Status

Terminated

Conditions

Intervention / Treatment

Detailed Description

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

Enrollment (Actual)

35

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • California
      • Stanford, California, United States
    • Indiana
      • Indianapolis, Indiana, United States
    • Maryland
      • Baltimore, Maryland, United States
        • Johns Hopkins
      • Baltimore, Maryland, United States
        • University of Maryland
    • Michigan
      • Detroit, Michigan, United States
    • Ohio
      • Columbus, Ohio, United States
    • Oregon
      • Portland, Oregon, United States
    • Pennsylvania
      • Hershey, Pennsylvania, United States
    • Tennessee
      • Germantown, Tennessee, United States
    • Utah
      • Salt Lake City, Utah, United States
    • Wisconsin
      • Madison, Wisconsin, United States

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (ADULT, OLDER_ADULT)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: TREATMENT
  • Allocation: NA
  • Interventional Model: SINGLE_GROUP
  • Masking: NONE

Arms and Interventions

Participant Group / 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.
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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
end of Period 2 (Week 16)

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
end of Period 1 (Week 8)
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.
end of Period 2 (Week 16)
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.
end of Period 1 (Week 8)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Heart Metabolics Limited

Investigators

  • Study Chair: Mark Midei, MD, Heart Metabolics

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (ACTUAL)

August 1, 2016

Primary Completion (ACTUAL)

April 28, 2017

Study Completion (ACTUAL)

May 22, 2017

Study Registration Dates

First Submitted

August 8, 2016

First Submitted That Met QC Criteria

August 8, 2016

First Posted (ESTIMATE)

August 11, 2016

Study Record Updates

Last Update Posted (ACTUAL)

August 31, 2017

Last Update Submitted That Met QC Criteria

August 2, 2017

Last Verified

August 1, 2017

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • HML-PHX-005

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

Yes

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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