Table 4.
Summary of the clinical trials investigating the use of perhexiline in the management of heart failure.
| Study | Year | Study Design | No. of Patients | Patient Cohort | Results |
|---|---|---|---|---|---|
| Beadle et al. [50] | 2015 | Randomised double-blind placebo-controlled trial, parallel-group study | 47 | Patients with systolic heart failure of non-ischemic etiology with NYHA class of II–IV | Treatment of with perhexiline compared to placebo in heart failure resulted in: 30% increase in PCr/ATP ratio (p < 0.001) (no change in placebo group) 52% of treated patients improved by 1 NHYA class compared to 20% improving in the placebo group (p = 0.02) No change in LVEF (p = 0.68) No change in BNP levels |
| Abozguia et al. [51] | 2010 | Randomised, double-blind, placebo-controlled, parallel-group trial | 46 | Patients with symptomatic exercise limitation caused by non-obstructive hypertrophic cardiomyopathy | Treatment of patients with hypertrophic cardiomyopathy with perhexiline compared to placebo resulted in: Improved exercise capacity (VO2 increased by 2.1 mL/kg/min, p = 0.003) Reportedly less symptoms (MLHFQ score improved by 8, p < 0.001) Improved NYHA classification in more patients (67% of treated patients compared to 30% of control) No significant difference in ejection fraction |
| Phan et al. [52] | 2009 | 151 | Patients with chronic heart failure (LVEF < 40% with NYHA class > IIb) or refractory angina | Treatment of patients with angina or heart failure with perhexiline resulted in the majority of patients reporting subjective symptom reduction (58.9%) | |
| Lee et al. [53] | 2005 | Randomised double-blind placebo-controlled trial | 56 | Patient with chronic heart failure with EF < 40% and NYHA Class II or III already on optimal treatment | Treatment of with perhexiline compared to placebo in heart failure resulted in: Increased VO2 max by 17% (p < 0.001) (no change in placebo group) Increased LVEF by 10% (p < 0.001) with no change in the placebo group Reduced symptoms of heart failure (MLHFQ score reduced by 24%, p = 0.04) while placebo group score was unchanged Mean NYHA classification improved by 21% (p = 0.02) with no change in the placebo group |
PCr/ATP = phosphocreatine/adenosine triphosphate; NHYA = New York Heart Association; LVEF = left ventricular ejection fraction; BNP = brain natriuretic peptide; VO2 max = peak exercise oxygen consumption; MLHFQ = Minnesota Living with Heart Failure Questionnaire.