Tideglusib for Arrhythmogenic Cardiomyopathy

(TaRGET Trial)

Recruiting at 20 trial locations

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Hamilton Health Sciences Corporation

Must not be taking: Strong CYP3A4 inhibitors

Disqualifiers: Heart failure, Liver disease, Substance use, others

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 6 months

120 Participants Needed

Trial Summary

What is the purpose of this trial?

The TaRGET study 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 β inhibitor, in genotype positive arrhythmogenic cardiomyopathy.

Will I have to stop taking my current medications?

The trial does not specify if you must stop taking your current medications, but you cannot start, stop, or change the dose of certain heart medications (Class I or III anti-arrhythmic drugs) within 3 months before joining. Also, you cannot use certain antibiotics or antifungal medications regularly.

How is the drug Tideglusib unique in treating arrhythmogenic cardiomyopathy?

Tideglusib is unique because it targets the underlying mechanisms of arrhythmogenic cardiomyopathy by modulating protein trafficking at the intercalated discs, which are crucial for heart cell communication and function. This approach differs from other treatments that primarily focus on managing symptoms rather than addressing the root cause of the disease. ^{1 2 3 4 5}

Research Team

Jason D Roberts, MD MAS

Principal Investigator

McMaster University and Population Health Research Institute

Eligibility Criteria

This trial is for individuals with a genetic form of arrhythmogenic cardiomyopathy, which affects the heart muscle and can cause irregular heartbeats. Participants must meet specific criteria that will be detailed by the study organizers.

Inclusion Criteria

I have been diagnosed with ACM or known as a genetic carrier for at least 6 months.

I have a specific genetic mutation linked to heart disease.

Mean ≥ 500 PVCs per 24 hours on a baseline screening 7-day Holter monitor

Exclusion Criteria

Patients unwilling to provide informed consent or comply with follow-up

Hypersensitivity to tideglusib or any components of its formulation, including allergy to strawberry

I have heart scar tissue from blocked heart arteries.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive tideglusib or placebo for 6 months to evaluate its efficacy in genotype positive ACM patients

6 months

Monthly visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• Tideglusib

Trial Overview The TaRGET study tests tideglusib, a drug aimed at inhibiting an enzyme involved in this type of heart disease, against a placebo. It's randomized and double-blind, meaning neither participants nor researchers know who gets the real drug or placebo.

Participant Groups

Group I: Tideglusib Active Control 1 Intervention

Randomization to Tideglusib 1g po daily or matching placebo

Group II: Placebo Placebo Group 1 Intervention

Randomization to matching placebo 1g po daily

Who Is Running the Clinical Trial?

Hamilton Health Sciences Corporation

Lead Sponsor

Trials

380

Recruited

345,000+

Hearts in Rhythm Organization (HiRO)

Collaborator

Trials

1

Recruited

120+

Population Health Research Institute

Collaborator

Trials

165

Recruited

717,000+

AMO Pharma

Collaborator

Trials

1

Recruited

120+

Canadian Institutes of Health Research (CIHR)

Collaborator

Trials

1,417

Recruited

26,550,000+

Canadian SADS

Collaborator

Trials

1

Recruited

120+

References

1. Switzerland pubmed.ncbi.nlm.nih.gov

Phospholamban R14del disease: The past, the present and the future. [2023]

2. England pubmed.ncbi.nlm.nih.gov

Rare non-coding Desmoglein-2 variant contributes to Arrhythmogenic right ventricular cardiomyopathy. [2020]

3. United States pubmed.ncbi.nlm.nih.gov

Arrhythmogenic Right Ventricular Cardiomyopathy Presenting as Clinical Myocarditis in Women. [2021]

4. United States pubmed.ncbi.nlm.nih.gov

Stabilization of desmoglein-2 binding rescues arrhythmia in arrhythmogenic cardiomyopathy. [2021]

5. United States pubmed.ncbi.nlm.nih.gov

Identification of a new modulator of the intercalated disc in a zebrafish model of arrhythmogenic cardiomyopathy. [2022]