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Journal article

Rationale and design of a randomised trial of trientine in patients with hypertrophic cardiomyopathy

Abstract:
Aims Hypertrophic cardiomyopathy (HCM) is characterised by left ventricular hypertrophy (LVH), myocardial fibrosis, enhanced oxidative stress and energy depletion. Unbound/loosely bound tissue copper II ions are powerful catalysts of oxidative stress and inhibitors of antioxidants. Trientine is a highly selective copper II chelator. In preclinical and clinical studies in diabetes, trientine is associated with reduced LVH and fibrosis, and improved mitochondrial function and energy metabolism. Trientine was associated with improvements in cardiac structure and function in an open-label study in patients with HCM.
Methods The Efficacy and Mechanism of Trientine in Patients with Hypertrophic Cardiomyopathy (TEMPEST) trial is a multicentre, double-blind, parallel group, 1:1 randomised, placebo-controlled phase II trial designed to evaluate the efficacy and mechanism of action of trientine in patients with HCM. Patients with a diagnosis of HCM according to the European Society of Cardiology Guidelines and in New York Heart Association classes I–III are randomised to trientine or matching placebo for 52 weeks. Primary outcome is change in left ventricular (LV) mass indexed to body surface area, measured using cardiovascular magnetic resonance. Secondary efficacy objectives will determine whether trientine improves exercise capacity, reduces arrhythmia burden, reduces cardiomyocyte injury, improves LV and atrial function, and reduces LV outflow tract gradient. Mechanistic objectives will determine whether the effects are mediated by cellular or extracellular mass regression and improved myocardial energetics.
Conclusion TEMPEST will determine the efficacy and mechanism of action of trientine in patients with HCM.
Trial registration numbers NCT04706429 and ISRCTN57145331.
Publication status:
Published
Peer review status:
Peer reviewed

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Publisher copy:
10.1136/heartjnl-2022-322271

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Role:
Author
ORCID:
0000-0002-9241-2410

Contributors

Role:
Contributor


Publisher:
BMJ Publishing Group
Journal:
Heart More from this journal
Volume:
109
Issue:
15
Pages:
1175-1182
Place of publication:
England
Publication date:
2023-05-03
Acceptance date:
2023-03-14
DOI:
EISSN:
1468-201X
ISSN:
1355-6037
Pmid:
37137675


Language:
English
Keywords:
Pubs id:
1340565
Local pid:
pubs:1340565
Deposit date:
2024-01-19

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