E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Idiopathic Dilated Cardiomyopathy |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10056419 |
E.1.2 | Term | Dilated Cardiomyopathy |
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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 |
The design of the study seeks to determine whether a significant improvement in cardiac function and symptoms can be achieved by the relatively non-invasive procedure of G-CSF (Granocyte™) administration or whether an invasive approach requiring direct intracoronary injection provides the optimal improvement.
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E.2.2 | Secondary objectives of the trial |
The best way to deliver this novel treatment to patients. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
INCLUSION 60 symptomatic patients with a confirmed diagnosis of dilated cardiomyopathy (NYHA II-III) attending a ‘Heart Failure clinic’ who are on optimal heart failure treatment, with no further treatment options, under supervision from their physician or heart failure nurse specialist.
Patients who are NYHA II that have been hospitalised with a dilated cardiomyopathy related condition. Prior to recruitment to the study patients at risk of ventricular arrhythmia will have undergone electrophysiological assessment and appropriate clinical management (including implantable defibrillator insertion) where indicated (as per NICE guidelines). Coronary angiography will be performed where necessary to confirm the diagnosis and ensure no other conventional treatment options are indicated.
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E.4 | Principal exclusion criteria |
Exclusions Patients who satisfy one or more of the following criteria are not suitable for inclusion in the study: • NYHA I • Documented ejection fraction >45% (any imaging modality) • The presence of cardiogenic shock • The presence of acute left and/or right sided pump failure as judged by the presence of pulmonary oedema and/or new peripheral oedema • Known severe pre-existent left ventricular dysfunction (ejection fraction <10%) prior to randomisation • Congenital cardiac disease • Cardiomyopathy secondary to a reversible cause that has not been treated e.g. thyroid disease, alcohol abuse, hypophosphataemia, hypocalcaemia, cocaine abuse, selenium toxicity & chronic uncontrolled tachycardia • Cardiomyopathy in association with a neuromuscular disorder e.g. Duchenne’s progressive muscular dystrophy • Previous cardiac surgery • Contra-indication for bone marrow aspiration • Known active infection • Known infection with human immunodeficiency virus (HIV), hepatitis B virus (HBV), hepatitis C virus (HCV), HTLV or syphilis. • Chronic inflammatory disease requiring ongoing medication • Serious known concomitant disease with a life expectancy of less than one year • Follow-up impossible (no fixed abode, etc) • Patients with an irregular heart rhythm (AF allowed if paced in a regular rhythm) • Patients with renal impairment (Creatinine >200mmol/L) • Neoplastic disease without documented remission within the past 5 years • Subjects of childbearing potential • Weight>140kg
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E.5 End points |
E.5.1 | Primary end point(s) |
Change in Left Ventricular Ejection Fraction as measured with cardiac MRI (or cardiac CT) at 3 months |
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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 | Information not present in EudraCT |
E.7.1.2 | Bioequivalence study | Information not present in EudraCT |
E.7.1.3 | Other | Information not present in EudraCT |
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.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 3 |
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 | Information not present in EudraCT |
E.8.7 | Trial has a data monitoring committee | Yes |
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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End Criteria for terminating study: Valid scientific or administrative reasons and reasons related to protection of patients. Investigators and associated Ethics Committees will be notified in writing in the event of termination. Criteria for Terminating a Study Centre: Reasons include but are not limited to repeated protocol deviations or serious protocol violations, Failure to secure written patient informed consent prior to the procedure & Failure to report Serious Adverse Event’s (SAE).
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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 | 3 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 3 |
E.8.9.2 | In all countries concerned by the trial months | 0 |