E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Hypertrophic Cardiomyopathy |
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E.1.1.1 | Medical condition in easily understood language |
Hypertrophic Cardiomyopathy |
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E.1.1.2 | Therapeutic area | Diseases [C] - Cardiovascular Diseases [C14] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10020871 |
E.1.2 | Term | Hypertrophic cardiomyopathy |
E.1.2 | System Organ Class | 10010331 - Congenital, familial and genetic disorders |
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E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
i. Does Trientine lead to an improvement in high-energy phosphate metabolism (myocardial energetics, i.e. energy processing) in patients with HCM? |
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E.2.2 | Secondary objectives of the trial |
ii. Does Trientine lead to reduction in left ventricular (LV) i.e. heart muscle mass in patients with HCM? iii. Does Trientine lead to an improvement in myocardial fibrosis (heart muscle scarring) in HCM? iv. Does Trientine lead to improved exercise capacity in patients with HCM? |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
• Male or female > 18 years of age • Females will be non-pregnant and non-lactating with no intention of pregnancy during study treatment • Confirmed diagnosis of HCM in line with 2011 ACCF / AHA consensus document • Β-myosin heavy chain genotype • Asymmetric septal hypertrophy-type HCM phenotype • LV ejection fraction ≥ 50%
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E.4 | Principal exclusion criteria |
• History of any other cardiovascular disorder, including aortic stenosis, aortic coarctation, hypertension, renal artery stenosis, atrial fibrillation • NYHA Class III / IV heart failure • Diabetes Mellitus • Contraindication to magnetic resonance imaging (MRI) scanning (including claustrophobia) • Known hypersensitivity to Trientine or excipients • Known hypersensitivity to Gadolinium-based contrast agent • eGFR < 50ml/min/1.73m2 • BMI > 40kg/m2 • History of significant malabsorption • Copper deficiency at baseline • Iron deficiency at baseline • Haemoglobin < 10g/dL • Unresolved haematological disorder • Severe hepatic impairment • Untreated thyroid disease • Autoimmune disorders/connective tissue disease • Drug or alcohol abuse • Pregnancy/breast-feeding. Women of childbearing potential (not >2 years post- menopausal and/or not surgically sterilised) must have a negative blood serum pregnancy test, performed at visit 1 prior to administration of study medication • Any clinically significant or unstable medical or psychiatric condition that would interfere with the patient’s ability to participate in the study • Any other condition, which in the opinion of the research team, may put participants at risk during the study, or which may affect the outcome of the study • New medication within the preceding month of the study (excluding short term prescriptions) • Participation in another study involving an investigational product in the previous 12 weeks |
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E.5 End points |
E.5.1 | Primary end point(s) |
To evaluate the change in myocardial energetics, as measured using cardiac MRI, in patients with HCM after treatment with Trientine. |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
This end-point will be evaluated after 6 months of treatment |
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E.5.2 | Secondary end point(s) |
ii. Does Trientine lead to reduction in left ventricular (LV) mass in patients with HCM? iii. Does Trientine lead to an improvement in myocardial fibrosis in HCM? iv. Does Trientine lead to improved exercise capacity in patients with HCM? |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
These end-point will be evaluated after 6 months of treatment |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | Yes |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | Information not present in EudraCT |
E.8.2.2 | Placebo | Information not present in EudraCT |
E.8.2.3 | Other | Information not present in EudraCT |
E.8.3 |
The trial involves single site in the Member State concerned
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
E.8.5 | The trial involves multiple Member States | No |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.7 | Trial has a data monitoring committee | No |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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2 weeks after LVLS, as we will follow up via a telephone consultation 2 weeks after finishing taking trientine. |
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 1 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | 1 |
E.8.9.2 | In all countries concerned by the trial years | 1 |
E.8.9.2 | In all countries concerned by the trial months | 0 |
E.8.9.2 | In all countries concerned by the trial days | 1 |