CompletedPhase IIInterventional

The efficacy and mechanism of trientine in patients with hypertrophic cardiomyopathy

Hypertrophic cardiomyopathy (HCM) is an inherited condition that causes the walls of the heart to become thick and stiff. This makes it harder for the heart to pump blood efficiently, leading to breathlessness, chest pain, and tiredness. Currently, treatments can only manage symptoms rather than fixing the underlying heart changes. This phase 2 trial tests a tablet called trientine. Trientine helps the body remove excess copper in urine, which earlier research suggests may help reverse heart muscle thickening. Around 152 adults aged 18 to 75 across the UK will take either trientine or a dummy pill (placebo) for one year. Participants will attend six hospital visits for regular checks, including heart MRI scans, blood tests, and exercise assessments. Researchers hope to learn whether trientine can safely shrink thickened heart muscle, protect against irregular heartbeats, and help patients stay more active.

At a glance

Status

Completed

Phase

Phase II

Sponsor

Manchester University NHS Foundation Trust

Enrolment target

152

Start

29 Mar 2021

Estimated completion

30 Apr 2023

Hypertrophic cardiomyopathy

What is this study about?

Hypertrophic cardiomyopathy (HCM) is the most common inherited heart muscle disease. In people with HCM, parts of the heart wall become abnormally thick and scarred. This extra thickness makes the heart stiff, meaning it cannot fill with or pump blood as easily as it should. Daily activities can become challenging, and people often experience shortness of breath, chest tightness, dizziness, or fainting, especially during physical effort. Although current medicines can help ease these symptoms, none can repair or reverse the muscle thickening itself.

Researchers are investigating whether an imbalance of copper in the body plays a role in this heart thickening. Trientine is an established oral medication that has been used safely for more than 30 years to treat a different condition by helping the body pass extra copper out through urine. Early studies show that balancing copper levels with trientine might actually help reverse heart muscle thickening. This trial aims to find out whether trientine can achieve this in people living with HCM.

Over the course of one year, researchers will closely compare patients taking trientine against those taking an inactive dummy tablet. They will measure whether the medicine shrinks heart thickness, boosts exercise capacity, and reduces the risk of irregular heart rhythms. If the results are positive, this research could lead to the first treatment that directly targets the underlying cause of HCM.

Key takeaways

Tests whether trientine can safely shrink thickened heart muscle in people with HCM.
Open to UK adults aged 18 to 75 with a confirmed HCM diagnosis.
Lasts 12 months and involves six hospital visits for heart checks and scans.
Half of the participants receive the active drug and half receive an inactive dummy pill.
Trientine is already well-studied and has been used safely for over 30 years.

Who may be eligible?

This trial is open to adults aged between 18 and 75 who have a confirmed diagnosis of hypertrophic cardiomyopathy. To qualify, your most recent heart scan (an MRI or echocardiogram) must show that at least one area of your heart muscle wall is 15 millimetres thick or more. You should also have mild to moderate symptoms that affect your daily physical activity, such as breathlessness when walking or climbing stairs.

You will not be able to join if you have had a past heart attack, severe coronary artery disease, or long-standing irregular heartbeats known as persistent atrial fibrillation. People who have had, or are planning to have, surgery or procedures to thin the heart muscle (septal reduction) are also excluded. Finally, you cannot take part if you have very high blood pressure, significantly weakened heart pumping strength, or low iron levels (anaemia).

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

I am aged between 18 and 75.
I have been diagnosed with hypertrophic cardiomyopathy (HCM).
A heart scan shows my heart muscle wall is at least 15 mm thick.
I have not had a heart attack or surgery to thin my heart wall.
I do not have persistent atrial fibrillation or low blood iron (anaemia).

Answer every question to see your result.

What does participation involve?

Taking part in the trial lasts for 12 months. Half of the participants will take trientine capsules each day, while the other half will take a dummy pill (placebo) that looks identical. A computer chooses who receives which treatment at random, and neither you nor your doctor will know which one you are taking. You will need to attend hospital for six visits during the year. These visits involve routine blood and urine tests, heart rhythm traces (ECGs), an exercise bike or treadmill test, wearing a 24-hour heart monitor at home, and MRI scans of your heart to measure wall thickness. Female participants of childbearing age will also have routine pregnancy tests.

Potential risks and benefits

By taking part, you will receive close specialist care, including detailed heart scans that may give you a better understanding of your condition. You will also help researchers find new treatments for HCM. Trientine has a strong 30-year safety record, but like all medicines, it can cause side effects. Between 1 in 10 and 1 in 100 people feel mild nausea when starting the tablets, and between 1 in 100 and 1 in 1,000 people develop a skin rash or lower iron levels. Your blood count and iron levels will be checked regularly, and iron tablets can be provided if needed. Any side effects usually go away if the dose is lowered or stopped. Taking part is entirely your choice, and you can withdraw at any time without your regular NHS care being affected.

Locations (7)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

Liverpool Heart And Chest Hospital NHS Foundation Trust
City only
Liverpool, England
Manchester University NHS Foundation Trust
City only
Manchester, England
Oxford University Hospitals NHSs Foundation Trust
City only
Oxford, England
Royal Brompton & Harefield NHS Foundation Trust
City only
London, England
University Hospitals of Leicester NHS Foundation Trust
City only
Leicester, England
NHS Grampian
Unverified
Aberdeen, Scotland
Northumbria Healthcare NHS Foundation Trust
Unverified
Northumberland, England

Common questions

Will I definitely receive the active medicine?

No. You will have a 50% chance of receiving trientine and a 50% chance of receiving a dummy pill (placebo). This is necessary so researchers can accurately measure if the drug works.

What is trientine normally used for?

Trientine has been prescribed safely for over 30 years to treat Wilson's disease, a rare genetic disorder where copper builds up in the body.

Can I keep taking my normal heart medications?

In most cases, yes. The study team will carefully review your current prescriptions to confirm they are safe to continue alongside the study tablets.

Are the hospital tests painful?

No. Tests like heart traces, wearing a heart monitor, and MRI scans are non-invasive and painless. Blood tests may cause a brief pinch or slight bruising.

What happens if I change my mind during the trial?

You are free to leave the study at any point. You do not need to give a reason, and your usual NHS medical care will carry on as normal.

How to find out more

Chris Miller

christopher.miller@manchester.ac.uk +44 (0)161 291 2034 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.