Perhexiline Therapy in Heart Failure With Preserved Ejection Fraction Syndrome

November 4, 2015 updated by: University of Aberdeen

Randomised Double Blind Placebo Controlled Trial of Perhexiline in Heart Failure With Preserved Ejection Fraction Syndrome (HFpEF)

Up to half of all patients with clinical features of heart failure are found to have normal heart pumping function. Recently the investigators have shown that a drug called perhexiline markedly improved exercise capacity and symptoms in patients with heart failure associated with impaired cardiac pump function. In this proposal the investigators will assess whether perhexiline has beneficial effects in patients with heart failure and a normal heart pumping function.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

Up to 50% of patients with symptoms of heart failure have a preserved left ventricular ejection fraction (HFpEF syndrome). Current therapy for systolic heart failure is targeted at inducing vasodilation and counteracting neuro-endocrine activation. There is a lack of a corresponding evidence base for the treatment of HEpEF. We have previously shown that perhexiline, an agent that increases the efficiency of energy production by shifting substrate utilization from free fatty acids towards glucose, was highly effective in improving exercise capacity, symptoms and cardiac function in patients with systolic heart failure. We have also recently shown that energy deficiency plays a major role in the pathophysiology of HFpEF. In this proposal we therefore aim to investigate the effectiveness of perhexiline in 70 HFpEF patients, in a 3 month randomised, double-blind, controlled trial. An interim analysis is planned after 20 patients.

Study Type

Interventional

Enrollment (Anticipated)

70

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 Kingdom
      • Aberdeen, United Kingdom, AB25 2ZD
        • University of Aberdeen

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

16 years to 90 years (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • HFpEF will be defined as:

    • Clinical features consistent with heart failure
    • LVEF ≥ 50%, with no evidence of significant valvular disease
    • No hypertrophic cardiomyopathy, and no evidence of pericardial constriction
    • Peak VO2 < 80% predicted, with RER>1 and with a pattern of gas exchange on metabolic exercise testing indicating a cardiac cause for limitation)
  • Patients recruited will be in sinus rhythm

Exclusion Criteria:

  • BMI >35
  • Objective evidence of lung disease on formal lung function testing
  • Reversible myocardial ischaemia on contrast-enhanced myocardial stress Echocardiography, and no evidence of exercise-induced mitral regurgitation (>2+)
  • Impaired hepatic function; known hypersensitivity to perhexiline

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: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Triple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Perhexiline
perhexiline 100mg o bd for 3 months
Drug: Perhexiline
100mg o bd for 3 months
Other Names:
  • Pexsig
Placebo Comparator: Placebo
Placebo one tablet bd for 3 months
Drug: Placebo
Placebo one tablet bd for 3 months
Other Names:
  • Starch Placebo

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Change in Peak oxygen consumption (Vo2max)
Time Frame: 3 months
3 months

Secondary Outcome Measures

Outcome Measure
Time Frame
Symptomatic Status (Modified Minnesota Living with Heart Failure Questionnaire)
Time Frame: 3 Months
3 Months
Resting myocardial energetics by cardiac MR spectroscopy (MRS)
Time Frame: 3 months
3 months
Resting and exercise diastolic function (nuclear studies)
Time Frame: 3 months
3 months
Indirect measures of resting LVEDP (tissue Doppler E/Ea)
Time Frame: 3 months
3 months
Global LV Ejection Fraction (MRI / nuclear studies)
Time Frame: 3 months
3 months

Collaborators and Investigators

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

Sponsor

University of Aberdeen

Investigators

  • Principal Investigator: Michael P Frenneaux, MBBS MD, University of Aberdeen

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

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

March 1, 2009

Primary Completion (Actual)

February 1, 2014

Study Completion (Actual)

February 1, 2014

Study Registration Dates

First Submitted

February 6, 2009

First Submitted That Met QC Criteria

February 6, 2009

First Posted (Estimate)

February 9, 2009

Study Record Updates

Last Update Posted (Estimate)

November 5, 2015

Last Update Submitted That Met QC Criteria

November 4, 2015

Last Verified

November 1, 2015

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • RRK 3147 (Other Identifier: Research and Development)
  • MREC 08/H1207/84 (Other Identifier: Ethics)
  • EudraCT 2006-001109-28 (Registry Identifier: European Clinical Trials)

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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