MedPath

The efficacy and mechanism of trientine in patients with hypertrophic cardiomyopathy

Phase 2
Conditions
Hypertrophic cardiomyopathy
Circulatory System
Registration Number
ISRCTN57145331
Lead Sponsor
Manchester University NHS Foundation Trust
Brief Summary

2023 Protocol article in https://pubmed.ncbi.nlm.nih.gov/37137675/ (added 04/05/2023)

Detailed Description

Not available

Recruitment & Eligibility

Status
Ongoing
Sex
All
Target Recruitment
152
Inclusion Criteria

1. Written informed consent given
2. Aged between 18 and 75 years inclusive (Updated 07/11/2023: previously between 18 and 70 years inclusive)
3. 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).
4. New York Heart Association class I, II or III at the most recent clinical assessment performed prior to the baseline visit

Exclusion Criteria

1. Previous or planned septal reduction therapy
2. Previously documented myocardial infarction or severe coronary artery disease
3. Uncontrolled hypertension, defined as a systolic blood pressure of >180 mmHg or diastolic blood pressure of >100 mmHg at visit 1
4. 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.
5. Previously documented persistent atrial fibrillation
6. Anaemia, defined as haemoglobin being below the local site normal reference range, at visit 1
7. Iron deficiency, defined as serum iron being below the local site normal reference range, at visit 1
8. Copper deficiency, defined as serum copper being below the normal reference range, at visit 1
9. Pacemaker or implantable cardioverter-defibrillator
10. Known severe valvular heart disease, as demonstrated on the most recent heart imaging performed prior to the baseline visit
11. Previously documented other cardiomyopathic cause of myocardial hypertrophy (e.g. amyloidosis, Fabry disease, mitochondrial disease)
12. History of hypersensitivity to any of the components of the investigational medicinal product (IMP)
13. Known contraindication to MRI scanning
14. 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.
15. 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

Study & Design

Study Type
Interventional
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
Change in left ventricular mass index (LVMi, g/m2) is measured using a cardiovascular magnetic resonance (CMR) scan at baseline and week 52
Secondary Outcome Measures
NameTimeMethod
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