Grant: Operating Grant: Clinical Trials Projects - Indirect Costs

Arrhythmogenic right ventricular cardiomyopathy (ARVC) is a genetic form of heart disease characterized by abnormal replacement of heart tissue with scar and fat. Affected patients are vulnerable to heart failure, dangerous fast heart beats, and sudden death. Current medical therapy for ARVC has been largely adopted from those utilized for more common forms of heart disease and none target the underlying disease process in ARVC. Unfortunately these therapies have modest effectiveness and patients often suffer from poor quality of life and potentially premature sudden death. Through a series of animal experiments, our team has identified a compound that has been able to prevent onset of ARVC in mouse models of the condition. This compound, named tideglusib, is currently in human clinical trials for a condition involving peripheral muscles called myotonic dystrophy. In this context, because it has established safety in humans, we can test its potential efficacy in human ARVC. We endeavour to evaluate the clinical utility of tideglusib in a randomized controlled trial in human ARVC. 120 ARVC patients will be randomized to oral tideglusib 1000mg daily or placebo for a period of 1 year. The planned primary outcome of the study is the change in premature ventricular contractions (extra beats from the lower chambers of the heart) as measured by a 7 day monitor. Multiple clinical outcomes including sudden death, defibrillator shocks, burden of dangerous fast heart beats, and heart pumping function will also be explored. Should tideglusib prove successful at improving heart function in our trial, it carries the potential of revolutionizing the treatment approach for this often devastating condition.