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Hypertrophic CardiomyopathyPhase 2TEMPESTNCT04706429

The Efficacy and Mechanism of Trientine in Patients With Hypertrophic Cardiomyopathy

A Phase 2 study testing Trientine in people with Hypertrophic Cardiomyopathy, run by Manchester University NHS Foundation Trust at 7 sites in United Kingdom.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial 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). Read more about this study

Who is it for?
Ages 18 to 75 years
Conditions the study is for
Hypertrophic Cardiomyopathy
What is being tested?
Trientine
Type of treatment
Pill or oral medicine
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
154 participants
Where
7 sitesUnited Kingdom
Study dates
1 Dec 2020to 30 Jul 2024
Phase
Phase 2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 75 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Written informed consent.
  • Age 18-75 inclusive.
  • Hypertrophic cardiomyopathy (HCM), as defined by the European Society of Cardiology HCM guidelines as: "a wall thickness ≥15 mm in one or more LV myocardial segments that is not explained solely by loading conditions". The same definition is applied to first-degree relatives of patients with HCM i.e. all participants are required to have a LV wall thickness ≥15 mm. Wall thickness is as measured on the most recent cardiovascular magnetic resonance (CMR) scan performed prior to the baseline visit. If CMR has not been performed previously, wall thickness measurement should be taken from the most recent echocardiogram performed prior to the baseline visit. (It is recognised that in the European Society of Cardiology guidelines a clinical diagnosis of HCM in first-degree relatives requires a wall thickness that is less than this value, however ≥15 mm is applied here in order to ensure that all participants have an unequivocal phenotype).
  • New York Heart Association class I, II or III at the most recent clinical assessment performed prior to the baseline visit.

Who cannot join

  • Previous or planned septal reduction therapy.
  • Previously documented myocardial infarction or severe coronary artery disease.
  • Uncontrolled hypertension, defined as a systolic blood pressure of >180mmHg or a diastolic blood pressure of > 100mmHg at Visit 1.
  • Known LV EF < 50%, as measured on the most recent CMR scan performed prior to the baseline visit. If CMR has not been performed previously, the most recent echocardiogram performed prior to the baseline visit should be used.
  • Previously documented persistent atrial fibrillation.
  • Anaemia, defined as haemoglobin being below the local site normal reference range, at Visit 1.
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  • Iron deficiency, defined as serum iron being below the local site normal reference range, at Visit 1.
  • Copper deficiency, defined as serum copper being below the normal reference range, at Visit 1.
  • Pacemaker or implantable cardioverter defibrillator.
  • Known severe valvular heart disease, as demonstrated on the most recent heart imaging performed prior to the baseline visit.
  • Previously documented other cardiomyopathic cause of myocardial hypertrophy (e.g. amyloidosis, Fabry disease, mitochondrial disease).
  • History of hypersensitivity to any of the components of the investigational medicinal product (IMP).
  • Known contraindication to MRI scanning.
  • Pregnancy, lactation or planning pregnancy. Women of childbearing capacity are required to have a negative serum pregnancy test before treatment, must agree to pregnancy tests at study visits as defined in the Section 8 and must agree to maintain highly effective contraception as defined in Section 8 during the study.
  • Any medical condition, which in the opinion of the Investigator, may place the patient at higher risk from his/her participation in the study, or is likely to prevent the patient from complying with the requirements of the study or completing the study.

Trial locations and contacts

7 sites in 1 country.

7 sites in 1 country

United Kingdom7 sites

  • NHS Grampian

    Aberdeen, AB25 2ZN

  • University Hospitals of Leicester NHS Foundation Trust

    Leicester, LE3 9QP

  • Liverpool Heart and Chester Hospital NHS Foundation Trust

    Liverpool, L14 3PE

  • Royal Brompton and Harefield NHS Foundation Trust

    London, SW3 6NP

  • Manchester University NHS Foundation Trust

    Manchester, M23 9LT

  • Northumbria Healthcare NHS Foundation Trust

    North Shields, NE29 8NH

  • Oxford University Hospitals NHS Foundation Trust

    Oxford, OX3 9DU

Other trials in United Kingdom

About this study

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

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.

Read the 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.

Being tested
Trientine, Placebo
Medical subject headings
Cardiomyopathy, Hypertrophic

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • TrientineDrug

    Also called Cufence

    Trientine dihydrochloride is a white to pale yellow crystalline hygroscopic powder.

  • PlaceboDrug

    Placebo capsule, manufactured with the exact components of the trientine capsules, without the active ingredient / investigational medicinal product.

Study groups in NCT04706429
Study groupRoleWhat they receive
TrientineActive comparatorTrientine dihydrochloride 1200 mg per day. This shall be taken orally as two Cufence 200mg hard capsules two times per day. The IMP will be dispensed to participants every 13 weeks for the duration of the study period (52 weeks).
Receives: Drug: Trientine
PlaceboPlacebo comparatorThe placebo shall be taken orally as two capsules two times per day. This will be dispensed to participants every 13 weeks for the duration of the study period (52 weeks).
Receives: Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Left ventricular mass indexed to body surface area (LVMi)

    Change in LVMi (g/m2), measured using CMR, from baseline to week 52.

    Measured: 12 months

Secondary outcomes (7)

  1. Urine copper excretion

    Cumulative urine copper excretion, measured using urinary copper, assessed from baseline to weeks 13, 26, 39 and 52.

    Measured: 12 months

  2. Exercise capacity

    Change in exercise capacity, measured using cardiopulmonary exercise testing (CPET), assessed from baseline to week 52.

    Measured: 12 months

  3. Number of non-sinus supraventricular heart beats, presence and amount of atrial fibrillation, number of ventricular-origin beats and presence and amount of non-sustained ventricular tachycardia

    Change in number of non-sinus supraventricular heart beats, presence and amount of atrial fibrillation, number of ventricular-origin beats and presence and amount of non-sustained ventricular tachycardia, in 24 hours, measured using ambulatory heart monitoring, assessed from baseline to week 52.

    Measured: 12 months

  4. Circulating high sensitivity troponin

    Change in circulating high sensitivity troponin, assessed from baseline to week 52.

    Measured: 12 months

  5. LV global longitudinal strain, wall thickness, mass, volumes and ejection fraction (EF)

    Change in LV global longitudinal strain, wall thickness, mass, volumes and ejection fraction (EF) measured using CMR, assessed from baseline to week 52.

    Measured: 12 months

  6. Peak left ventricular outflow

    Change in peak left ventricular outflow tract gradient, measured using CMR, assessed from baseline to week 52.

    Measured: 12 months

  7. Atrial volume and function

    Change in atrial volume and function, measured using CMR, assessed from baseline to week 52.

    Measured: 12 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted12 Jan 2021
  2. Study start1 Dec 2020
  3. Primary completion30 Apr 2024
  4. Study completion30 Jul 2024

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
154 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04706429
Sponsor's protocol ID
B00844
Other IDs
2020-002242-17, ISRCTN57145331, NIHR127575
Acronym
TEMPEST
Status last verified
Sep 2025
First submitted
11 Jan 2021
Last updated
29 Sep 2025
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
Undecided

Publications

  • Farrant JP, Dodd S, Rosala-Hallas A, Vaughan C, Spowart C, Bedson E, Clayton D, Reid AB, Garratt CJ, Raman B, Mahmod M, Akhtar M, Valkovic L, Ashkir Z, Cooper R, Singh A, Prasad S, Green T, Dawson D, Moon JC, Baroja O, Duran B, Naish J, Harrington CF, Garg S, Graham C, Mohiddin S, Dweck MR, Greenlaw N, Plein S, Berry C, Schmitt M, Neubauer S, Miller CA. Trientine for hypertrophic cardiomyopathy: a phase 2 trial. Eur Heart J. 2026 Jul 15:ehag512. doi: 10.1093/eurheartj/ehag512. Online ahead of print.

    PubMed 42455121

  • Farrant J, Dodd S, Vaughan C, Reid A, Schmitt M, Garratt C, Akhtar M, Mahmod M, Neubauer S, Cooper RM, Prasad SK, Singh A, Valkovic L, Raman B, Ashkir Z, Clayton D, Baroja O, Duran B, Spowart C, Bedson E, Naish JH, Harrington C, Miller CA; TEMPEST investigators. Rationale and design of a randomised trial of trientine in patients with hypertrophic cardiomyopathy. Heart. 2023 Jul 12;109(15):1175-1182. doi: 10.1136/heartjnl-2022-322271.

    PubMed 37137675

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Trientine in people with Hypertrophic Cardiomyopathy, run by Manchester University NHS Foundation Trust at 7 sites in United Kingdom.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 4 inclusion and 15 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 29 Sep 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 7 sites in United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Left ventricular mass indexed to body surface area (LVMi) (time frame: 12 months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04706429, which also lists 2 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Sep 2025.

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