TEMPEST
Research type
Research Study
Full title
A randomised, double-blind, placebo-controlled, phase 2 evaluation of the efficacy and mechanism of trientine in patients with hypertrophic cardiomyopathy.
IRAS ID
283265
Contact name
Christopher Miller
Contact email
Sponsor organisation
Manchester University NHS Foundation Trust
Eudract number
2020-002242-17
Clinicaltrials.gov Identifier
Duration of Study in the UK
3 years, 0 months, 1 days
Research summary
Hypertrophic cardiomyopathy (HCM) is the most common inherited heart condition. It leads to abnormal thickening and scarring of the walls of the heart. As a result, the heart can have trouble pumping blood as well as it should. This causes patients to develop breathing difficulties, chest pain and fainting, which often get worse with exercise, and therefore can limit physical activity. Current treatments only aim to relieve the symptoms. There are no treatments that correct the underlying damage to the heart. Patients, caregivers and researchers have identified a “critical need” for trials of medicines that target the HCM disease process.
Several studies in other diseases have shown that copper imbalance is associated with heart thickening similar to HCM, and that treatment with trientine, an oral medicine that increases copper removal in urine, can reverse this thickening.
This study will investigate whether trientine reduces heart muscle thickening, improves exercise capacity, improves heart function and reduces abnormal heart rhythms in patients with HCM. The study will also assess how trientine works in HCM.
This research study aims to recruit 172 patients with HCM aged 18-70 years in the UK. Participants in the study will be prescribed either trientine or placebo for 1 year to compare the difference.
Results Summary:
Hypertrophic cardiomyopathy is the most common inherited heart condition. It causes thickening and scarring of the heart muscle, and reduces the heart's ability to produce energy efficiently.This study investigated whether trientine, a drug that affects how the body handles copper, could help people with hypertrophic cardiomyopathy. Copper is an essential mineral that plays an important role in energy production within the heart and helps protect heart cells from damage and scarring. Low copper levels inside heart cells can impair energy production and contribute to thickening of the heart muscle. Excess copper outside heart cells may promote the formation of scar tissue. Trientine is thought to improve these processes by restoring a healthier balance of copper in the heart.
Over the course of a year, people with hypertrophic cardiomyopathy who received trientine experienced a greater reduction in heart muscle thickness than those who received a placebo. Trientine was generally well tolerated, with few side effects.
Overall, the findings suggest that trientine may help reverse some of the abnormal heart muscle growth seen in hypertrophic cardiomyopathy. While the results are encouraging, the study was not designed to determine whether the drug improves symptoms, daily functioning, or long-term clinical outcomes. Further research is needed to see whether these improvements translate into meaningful benefits for patients.
REC name
North West - Greater Manchester South Research Ethics Committee
REC reference
20/NW/0275
Date of REC Opinion
21 Jul 2020
REC opinion
Further Information Favourable Opinion