E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Ischemic heart failure is associated with significant morbidity and mortality. The number of patients with severe heart failure that remains symptomatic despite optimal pharmacological and non-pharmacological treatment increases rapidly. At present there are no widely available conventional therapeutic options for these patients who have an adverse prognosis: 2-year mortality over 50% in patients with functional class III and IV. |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 12.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10019279 |
E.1.2 | Term | Heart failure |
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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 |
We intend to assess the efficacy of cardiac stem cell therapy in ischemic heart failure patients. Specifically, we want to answer the question whether injection of autologous mononuclear bone marrow cells (BMCs) into the myocardium of no-option patients with ischemic heart failure leads to an improvement in Quality of Life, exercise capacity, myocardial perfusion and myocardial function. |
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E.2.2 | Secondary objectives of the trial |
Injection of autologous mononuclear bone marrow cells (BMCs) into the myocardium of no-option patients with ischemic heart failure is safe with respect to arrhythmia's and periprocedural myocardial damage. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- Ischemic heart failure NYHA class 3 or 4 despite optimal pharmacological and non- pharmacological therapy - No candidate for (repeat) surgery (revascularization, valve repair or ventricular reconstruction) - No candidate for (repeat) percutaneous revascularization - Optimal resynchronization therapy, or no candidate for resynchronization therapy. - Male or female, > 18 years and < 75 years old. - Life expectancy more than 6 months - Able to perform an exercise tolerance test prior to therapy - Able and willing to undergo all the tests used in this protocol including the traveling involved. - Written informed consent
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E.4 | Principal exclusion criteria |
- Evidence of cancer (except low grade and fully resolved non-melanoma skin malignancy) - Concurrent participation in a study using an experimental drug or an experimental procedure within 2 months before randomization. - Other severe concurrent illnesses (e.g. active infection, aortic stenosis, renal insufficiency) - Bleeding diathesis, HIV infection or pregnancy. - Any other condition that, in the opinion of the investigator, could pose a significant threat to the subject if the investigational therapy will be initiated. - Inability to undergo cardiac catheterization or nuclear testing - Inability to follow the protocol and comply with follow-up requirements - Candidate for surgery (revascularization, valve repair or ventricular reconstruction), resynchronization therapy or percutaneous revascularization
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E.5 End points |
E.5.1 | Primary end point(s) |
Efficacy: Left ventricular global ejection fraction as assessed by Gated SPECT. LV regional wall motion by echocardiography FDG-SPECT for assessment of viability and hibernation myocardial innervation imaging (MIBG-SPECT) for assessment of myocardial innervation. Exercise capacity by bicycle exercise testing with VO2 measurement. Quality of Life will be assessed using the Minnesota Living with Heart Failure questionnaire.
Safety: As many patients will already have an implanted cardioverter/defibrillator (ICD), occurrence of arrhythmia’s are monitored via these devices if possible. In patients without ICD regular Holter monitoring will be performed. Moreover, blood samples will be taken to assess possible inflammation and myocardial damage. |
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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) | Yes |
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 | No |
E.8.1.6 | Cross over | Yes |
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
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 1 |
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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If the safety monitoring committee decides that serious events related to the therapy have occurred, which are a reason to cease the trial. |
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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 | 0 |
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 | 0 |