Hypertrophic Cardiomyopathy MedDRA version: 20.0 Level: PT Classification code 10020871 Term: Hypertrophic cardiomyopathy System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Trade Name: Trientine dihydrochloride
Product Name: Trientine dihydrochloride
Pharmaceutical Form: Capsule, hard
INN or Proposed INN: Trientine Dihydroc
Sponsors
Manchester University NHS Foundation Trust
Eligibility
Sex/Gender
All
Inclusion criteria
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 • ?-myosin heavy chain genotype • Asymmetric septal hypertrophy-type HCM phenotype • LV ejection fraction = 50% Are the trial subjects under 18? no Number of subjects for this age range: 0 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 18 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 2
Exclusion criteria
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 40kg/m2 • History of significant malabsorption • Copper deficiency at baseline • Iron deficiency at baseline • Haemoglobin 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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: i. Does Trientine lead to an improvement in high-energy phosphate metabolism (myocardial energetics, i.e. energy processing) in patients with HCM?; Secondary Objective: 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? ;Primary end point(s): To evaluate the change in myocardial energetics, as measured using cardiac MRI, in patients with HCM after treatment with Trientine.;Timepoint(s) of evaluation of this end point: This end-point will be evaluated after 6 months of treatment | — |
Secondary
| Measure | Time frame |
|---|---|
| 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? ;Timepoint(s) of evaluation of this end point: These end-point will be evaluated after 6 months of treatment | — |
Countries
United Kingdom
Contacts
Public ContactAnna Reid
Manchester University NHS Foundation Trust
Outcome results
None listed