E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Autologous cell therapy (ACT) is new technique for patients with critical limb ischemia and diabetic foot (DF). In our study, the effect of ACT and also the repetitive treatment will be compared with standard treatment in DF patients ineligible for angioplasty. We suppose an enhanced effect of ACT on on macro- and microcirculation and improved limb outflow. Expected results of the project include improved tissue oxygenation, enhanced DF healing and a reduced risk of major amputation. |
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E.1.1.1 | Medical condition in easily understood language |
Autologous cells therapy of critical limb ischemia and diabetic foot will be compared with standard treamtent by means of - tissue oxygenation, major amputation and wound healing. |
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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 | 19.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10034576 |
E.1.2 | Term | Peripheral ischaemia |
E.1.2 | System Organ Class | 10047065 - Vascular 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 |
The aim of the project is to compare the effect on tissue oxygenation and clinical outcome of autologous cell therapy with standard treatment in patients with diabetic foot disease (DFD) not eligible for PTA and to verify the effect of repetitive cell therapy.
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E.2.2 | Secondary objectives of the trial |
Other objectives of the project are quality of life assessment and side effects of study treatment. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
To include int the study the following criteria must be fullfilled:
1. diabetic foot disease (DFD; ulcer distal from ankle) or status after minor amputation, in accordance with international classification TEXAS 2-3 (C-D), Wagner 2-4
2. presence of chronic critical limb ischemia – ulcers or gangrene attributable to objectively proven arterial occlusive disease (transcutaneous oxygen pressure TcPO2 under 35 mm Hg), non-eligibility for standard revascularization (PTA or by-pass)
3. age 18-90 years
4. diabetes mellitus type 1 or 2
5. signed informed consent
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E.4 | Principal exclusion criteria |
Patients who fullfilled one of these criteria will be excluded:
1. severe active deep infection of DFD
2. deep vein thrombosis less than 6 months
3. severe limb oedema that rule out intramuscular injection of cell suspension
4. severe non-treated diabetic retinopathy requiring acutely a laser therapy
5. severe haematological disease
6. diagnosed neoplastic process of any organ
7. expected life prognosis shorter than 6 months
8. contracindication of general anestesia
9. females of childbearing potential must be willing to use a highly effective method of contraception (hormonal or barrier method of birth control; abstinence). Contraceptive methods that can achieve a failure rate of less than 1% per year when used consistently and correctly are considered as highly effective birth control methods.
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E.5 End points |
E.5.1 | Primary end point(s) |
Comparison of cell therapy with standard treatment in several parameters:
a) Assessment of tissue oxygenation – improvement of parameters of ischemia (measured by transcutaneous oxygen pressure andoptionally by other angiological methods)
b) Clinical effect – wound healing, change in pain, rate of major amputation, amputation-free survival
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
Transcutaneous oxygen pressure and other parameters will be assessed after 1, 3 and 6 months after the ACT. |
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E.5.2 | Secondary end point(s) |
1. Safety: adverse events - expected and unexpected
2. Assesment of study treatment by patients (psychological questionaries)
3. To assess the relation between the quality of cell suspension and tissue oxygenation
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
Safety: during and up to 6 months after the procedure
Questionaries: after 1, 3 and 6 months after the procedure
Quality: directly after the procedure |
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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) | 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 | Yes |
E.8.1.4 | Double blind | No |
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 | No |
E.8.2.3 | Other | Yes |
E.8.2.3.1 | Comparator description |
standard conservative treatment |
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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.8.5 | 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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LVLS |
Poslední návštěva posledního pacienta |
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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 | 5 |
E.8.9.1 | In the Member State concerned months | |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 5 |