Evaluating the role of perhexiline (new medication treatment) in patients with abnormally thickened heart muscle (hypertrophic cardiomyopathy)

Phase 2

• Conditions: Chest pain; Shortness of breath; Abnormally thickened heart muscle; Cardiovascular - Other cardiovascular diseases; Human Genetics and Inherited Disorders - Other human genetics and inherited disorders

• Registration Number: ACTRN12620000785909
• Lead Sponsor: Flinders Medical Centre

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2020-08-03
• Last Update Posted: 10 August 2020

Recruitment & Eligibility

• Status: ot yet recruiting
• Sex: All
• Target Recruitment: 60

Inclusion Criteria

a) LVEF =/> 55% by echocardiography or CMR during the screening period or within 6 months prior to study entry
b) Current / prior symptom(s) of HCM (New York Heart Association [NYHA] functional class II or class III, Canadian Cardiovascular Society [CCS] grade II or grade III) and requiring treatment with ß-blockers and /or non-dihydropyridine calcium antagonists and / or disopyramide for at least 30 days prior to study entry
c) Structural heart disease as evidenced by interventricular septal thickness of (=/> 15 mm) on echocardiography or CMR in the absence of abnormal loading conditions
d) Elevated NT-proBNP (>125 pg/ml)

Exclusion Criteria

a) Any prior echocardiographic or CMR measurement of LVEF <55%
b) Current acute decompensated heart failure requiring hospitalisation and / or augmented medical therapy
c) Cardiac surgery or catheter-based septal reduction therapy planned or having occurred within the past 1 year
d) Patients with a non-CMR conditional pacemaker / implantable cardioverter-defibrillator device
e) History of a known chronic liver disease, peripheral neuropathy, recurrent hypoglycemia
f) Presence of an additional diagnosis that in the opinion of investigator could account for patient's symptoms (e.g. significant pulmonary disease)
g) Serum bilirubin, alanine aminotransferase, aspartate aminotransferase, alkaline phosphatase, or lactate dehydrogenase > 2.0 times upper limit of normal
h) Previous adverse reaction to perhexiline at therapeutic plasma levels of the drug
i) Concomitant use of amiodarone, ranolazine or trimetazidine
j) Life-threatening or uncontrolled dysrhythmia
k) Contraindications to CMR, gadolinium, adenosine

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Change in left ventricular hypertrophy (septal thickness) in symptomatic HCM patients at 12 months following perhexiline therapy as assessed by CMR [At 12 months post baseline]