Tideglusib for Arrhythmogenic Cardiomyopathy

(TaRGET Trial)

Recruiting at 26 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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to determine if tideglusib, a medication, can benefit individuals with arrhythmogenic cardiomyopathy, a heart condition affecting muscle and rhythm. Researchers seek to discover if tideglusib can enhance heart health in those with a genetic marker linked to this condition. Participants will receive either tideglusib or a placebo (a pill with no active ingredient) daily. Individuals with a genetic diagnosis of this heart condition for at least six months and who experience frequent extra heartbeats may be suitable for the trial. As a Phase 2 trial, this research measures tideglusib's effectiveness in an initial, smaller group, offering participants a chance to contribute to significant medical advancements.

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.

Is there any evidence suggesting that tideglusib is likely to be safe for humans?

Studies have shown that tideglusib, also known as AMO-02, is generally safe based on research with patients over several years. In a long-term study involving patients with a different condition, the treatment was well-tolerated for almost four years, with few serious side effects. This evidence suggests that tideglusib could be safe for humans, but treatments can affect people differently. It is important to discuss potential risks and benefits with a healthcare provider before joining a trial. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for arrhythmogenic cardiomyopathy?

Tideglusib is unique because it targets the root causes of arrhythmogenic cardiomyopathy by inhibiting an enzyme called GSK-3β, which is different from the way most current treatments work. Current options usually focus on managing symptoms or preventing arrhythmias with medications like beta-blockers or antiarrhythmics. Unlike these traditional treatments, Tideglusib offers a new approach by potentially modifying the disease process itself. Researchers are excited about Tideglusib because it might not only improve symptoms but also slow down disease progression, offering hope for better long-term outcomes.

What evidence suggests that tideglusib might be an effective treatment for arrhythmogenic cardiomyopathy?

Research has shown that tideglusib, which participants in this trial may receive, holds potential for treating arrhythmogenic cardiomyopathy (ACM) based on animal studies. Specifically, tideglusib successfully prevented heart rhythm issues and other heart problems in mice with ACM. This drug blocks a protein called GSK-3β, involved in many cell activities. By targeting this protein, tideglusib might help manage ACM symptoms in people. Although more research in humans is needed, these early findings offer promising prospects for future treatment options. ^{2 3 6 7 8}

Who Is on the Research Team?

Jason D Roberts, MD MAS

Principal Investigator

McMaster University and Population Health Research Institute

Are You a Good Fit for This Trial?

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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+

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT06174220

Study Details | NCT06174220 | Targeted Therapy With ... Driven by promising findings observed for tideglusib in ACM mouse models, we now seek to evaluate its potential efficacy in a randomized clinical trial ...

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12399120/

Evaluation of Tideglusib as a Disease Modifying Therapy in ... Tideglusib has been shown for the first time to prevent the functional and arrhythmia phenotypes in 2 different mouse models of ACM. •.

3. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11885336/

Targeting Canonical Wnt-signaling Through GSK-3β in ... In light of the current TaRGET trial, designed to assess the therapeutic efficacy of the GSK3β inhibitor tideglusib in patients with ACM [5], it ...

4. clinicaltrials.gov clinicaltrials.gov/study/NCT06174220?term=AREA%5BInterventionSearch%5D(NP-031112)%20AND%20AREA%5BStudyType%5D(INTERVENTIONAL)&rank=8

Targeted Therapy With Glycogen Synthase Kinase-3 ... 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 ...

5. phri.ca phri.ca/targettrial/

TaRGET to test new treatment in patients with ACM - News Should Tideglusib be found effective, it will provide compelling data to motivate larger studies designed to evaluate clinical outcomes. Jason ...

6. amo-pharma.com amo-pharma.com/news/Press_Release_250915.pdf

Press Release "We are pleased to share new findings that reflect the experience of patients treated with. AMO-02 for almost four full years in our REACHCDM-X ...

7. prnewswire.com prnewswire.com/news-releases/amo-pharma-reports-long-term-safety-data-from-reachcdm-x-study-of-amo-02-in-treatment-of-congenital-myotonic-dystrophy-type-1-302555592.html

AMO Pharma Reports Long-Term Safety Data from ... -Four years of study data highlight continued favorable safety profile for AMO-02 -FDA meeting planned in Q4 2025 to discuss potential route ...

8. clival.com clival.com/news/amo-pharma-reports-long-term-safety-data-for-amo-02-in-congenital-and-childhood-onset-dm1

AMO Pharma Reports Long-Term Safety Data for AMO-02 ... “We are pleased to share new findings that reflect the experience of patients treated with AMO-02 for almost four full years. The results ...