NCT04706429

The Efficacy and Mechanism of Trientine in Patients With Hypertrophic Cardiomyopathy

A Randomised, Double-blind, Placebo-controlled, Phase 2 Evaluation of the Efficacy and Mechanism of Trientine in Patients With Hypertrophic Cardiomyopathy

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 154 (actual)
• Sponsor: Manchester University NHS Foundation Trust · Other Government
• Sex: All
• Age: 18 Years – 75 Years
• Healthy volunteers: Not accepted

Summary

This research study has been designed to test whether a drug called trientine dihydrochloride (also called Cufence) reduces heart muscle thickening, improves exercise capacity, improves heart function and reduces abnormal heart rhythms in patients with hypertrophic cardiomyopathy (HCM). The study is also assessing how trientine works in HCM. Participants will be prescribed either trientine or placebo, for a period of 12 months.

Detailed description

HCM is the most common inherited cardiovascular disorder. It is characterised by left ventricular (LV) myocardial hypertrophy and fibrosis. Patients can experience symptoms of effort intolerance, progressive heart failure and abnormal heart rhythms. There are currently no treatments that alter the natural history of HCM. Patients and the cardiovascular field have identified a "critical need" for clinical studies of drug therapies that target HCM pathophysiological mechanisms. Trientine dihydrochloride is a copper-chelating agent licensed for Wilson disease, a genetic disorder of copper excretion, in which patients exhibit a cardiac phenotype that mimics HCM. Proof of concept has been established through an MRC-funded study to suggest that use of trientine may also be beneficial in HCM.

Conditions

• Hypertrophic Cardiomyopathy

Interventions

Type	Name	Description
DRUG	Trientine	Trientine dihydrochloride is a white to pale yellow crystalline hygroscopic powder.
DRUG	Placebo	Placebo capsule, manufactured with the exact components of the trientine capsules, without the active ingredient / investigational medicinal product.

Timeline

• Start date: 2020-12-01
• Primary completion: 2024-04-30
• Completion: 2024-07-30
• First posted: 2021-01-12
• Last updated: 2025-09-29

Locations

7 sites across 1 country: United Kingdom