The TaRGET trial is a multi-centre, prospective, randomized, double-blind, placebo-controlled trial designed to evaluate the potential therapeutic efficacy of tideglusib, a glycogen synthase kinase-3 beta (GSK3β) inhibitor, in genotype positive arrhythmogenic cardiomyopathy (ACM).

ACM is a heritable form of structural heart disease characterized by myocardial fibrosis that confers vulnerability to malignant ventricular arrhythmias and sudden cardiac death (SCD). A subgroup of cases preferentially involves the right ventricle and is termed arrhythmogenic right ventricular cardiomyopathy (ARVC). Insight into its pathophysiology remains modest. A lack of understanding of its operative biological pathways has rendered development of tailored treatments challenging, leading to approaches to medical therapy being largely adopted from those utilized for more common forms of cardiomyopathy.

In 2014, a high-throughput screen of a library of bioactive compounds against a zebrafish model of ACM identified a small molecule classified as a GSK3 inhibitor that successfully prevented and rescued the phenotype, findings that have subsequently been reproduced in a series of ACM murine models. GSK3 is an enzyme that modulates the activity of a broad spectrum of intracellular signaling pathways, including the canonical Wnt/β-catenin pathway, whose suppression has been suggested to exert an important role in ACM pathogenesis.

Tideglusib is an oral GSK3β inhibitor with an established safety profile in humans. Driven by promising findings observed for tideglusib in ACM mouse models, we now seek to evaluate its potential efficacy in a randomized clinical trial involving genotype positive ACM patients.

Primary outcome:

Change in mean PVC count per 24 hours on 7-day Holter between baseline and 6 months.

Secondary outcomes:

  • Change in ventricular strain on echocardiography between baseline and 6 months
  • Number of Implantable cardioverter-defibrillator (ICD) therapies (shock or anti-tachycardia pacing) between baseline and 6 months
  • Number of sustained ventricular tachycardia events (VT; defined as symptomatic or duration > 30 seconds and > 100bpm) between baseline and 6 months
Learn about the TaRGET trial here
Study Type

Interventional - Drug

Study Design

Double blinded, placebo-controlled

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Population Health Research Institute

Hearts in Rhythm Organization (HiRO)

The Canadian Sudden Arrhythmia Death Syndromes (SADS) Foundation

Canadian Institutes of Health Research (CIHR)

AMO Pharma

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