E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Hypertrophic cardiomyopathy (non-obstructive) |
|
E.1.1.1 | Medical condition in easily understood language |
Hypertrophic cardiomyopathy is an inherited heart condition affecting 1 in 500 people. The heart is abnormally thickened which causes breathlessness, chest pain and sudden death. |
|
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 | 16.1 |
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 |
|
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 |
Principle question: Does trimetazidine improve exercise capacity in patients with HCM?
We will test trimetazidine against placebo (dummy drug) in patients who have symptoms despite standard treatment.
|
|
E.2.2 | Secondary objectives of the trial |
Secondary question: Does trimetazidine improve quality of life, heart pump function and heart rhythm problems in patients with HCM? |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
• Non-obstructive hypertrophic cardiomyopathy (gradient < 50 mmHg) • NYHA Class ≥ 2 • Abnormal peak VO2 as % predicted for age and gender • Age 18 years or above • Resting heart rate < 90/minute at rest. Rhythm may be sinus rhythm (SR), atrial fibrillation (AF) or paced. • No significant renal or hepatic impairment • Able to comply with study requirements and able to give informed consent
|
|
E.4 | Principal exclusion criteria |
• Female participant who is pregnant, breast-feeding or planning pregnancy during the course of the study. Females of childbearing potential must have a negative pregnancy test within 7 days prior to being registered for trial treatment. • Significant renal (GFR <60ml/min) or hepatic impairment • Involvement in another research study in the past 12 weeks • Diabetes mellitus • Known hypersensitivity to trimetazidine • Participant has Parkinson’s disease or Parkinsonism. |
|
E.5 End points |
E.5.1 | Primary end point(s) |
Peak oxygen consumption (peak VO2) |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
|
E.5.2 | Secondary end point(s) |
• 6 minute walk distance • Number of ventricular ectopics as determined by 24 h Holter, • Minnesota Heart Failure score • Echo (ejection fraction %, category of diastolic dysfunction. left atrial area, global systolic strain) • Lab tests (NT pro BNP, Troponin T, Insulin/Glucose ratio) • Sub maximal exercise parameters (VE/VO2 slope, VO2/work slope, VE/VCO2).
|
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
|
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) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | Yes |
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) | No |
E.8.2.2 | Placebo | Yes |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 2 |
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.8.4.1 | Number of sites anticipated in Member State concerned | 1 |
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
|
The end of the trial will be the date of the last telephone follow-up visit by the last participant |
|
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 2 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 2 |
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 | 0 |