E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 9.1 |
E.1.2 | Level | SOC |
E.1.2 | Classification code | 10029205 |
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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 |
 Evaluation of clinical efficacy of RD combination in patients with POEMS syndrome, in terms of improvement of either the disability caused by the neuropathy (ONLS) or organ specific symptoms (appendix D) after 6 cycles. |
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E.2.2 | Secondary objectives of the trial |
 Evaluation of the safety of RD combination  Evaluation of the activity of RD combination in VEGF/pro-inflammatory cytokine serum levels reduction after 6 cycles  Evaluation of clinical efficacy of RD combination in terms of improvement of muscle strength assessed by the expanded MRC sum score after 6 cycles  Evaluation of clinical efficacy of RD combination in terms of improvement of sensation assessed by the INCAT sensory sum score after 6 cycles  Evaluation of clinical efficacy of RD combination in terms of improvement of motor conduction velocity, distal latency and distal CMAP amplitude in one upper limb and one lower limb nerve. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Age > 18 years 2. Patients with a diagnosis of POEMS syndrome based on published diagnostic criteria (Appendix B POEMS syndrome Diagnostic Criteria) 3. Patients with at least a moderate impairment due to neuropathy corresponding to an ONLS of 2 or more 4. Patients with a Karnofsky performance status = 60% 5. Patients with a life-expectancy > 6 months. 6. Patients willing and able to comply with the protocol requirements, 7. Patients who have given voluntary written informed consent before any study-related procedure not part of normal medical care, with the understanding that consent may be withdrawn by the patient at any time without prejudice to their future medical care. 8. Female patient should be either post-menopausal or surgically sterilised or willing to use two acceptable method of birth control. The two methods of reliable contraception must include one highly effective method (i.e. intrauterine device (IUD), hormonal except oral formulations, and one additional effective (barrier) method (i.e. latex condom, diaphragm, cervical cap). Combined oral contraceptive pills are not recommended because they carry an increased risk of venous thromboembolism. If a patient is currently using combined oral contraception, switching to another adequate method of contraception should be considered. 9. Male patient should agree to use an acceptable method for contraception (i.e., condom or abstinence) for the duration of the study. 10. Patients with the following laboratory values within 14 days before day 1 of the Cycle 1: Absolute neutrophil count > 1.5 x 109/L without the use of growth factors; Platelets count > 75 x 109/L without transfusion support within 7 days before the test Calculated or measured creatinine clearance: = 20 mL/minute Total bilirubin < 1.5 x the ULN AST (SGOT) and ALT (SGPT) < 2.5 x ULN |
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E.4 | Principal exclusion criteria |
Any serious medical condition, laboratory abnormality, or psychiatric illness that would prevent the subject from signing the informed consent form. 2. Pregnant or breast feeding females. 3. Any condition, including the presence of laboratory abnormalities, which places the subject at unacceptable risk. 4. Use of any other concomitant standard/experimental drug or therapy 5. Any prior use of Revlimid 6. Any of the following laboratory abnormalities: Platelets count < 75 � 109/L Absolute neutrophils count <1.5 � 109/L Calculated or measured creatinine clearance <20 mL/minute. 7. Known positive HIV or active infectious hepatitis, type B or C |
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E.5 End points |
E.5.1 | Primary end point(s) |
 Evaluation of clinical efficacy of RD combination in patients with POEMS syndrome, in terms of improvement of either the disability caused by the neuropathy (ONLS) or organ specific symptoms (appendix D) after 6 cycles. |
|
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 | No |
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 | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
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) | Information not present in EudraCT |
E.8.2.2 | Placebo | Information not present in EudraCT |
E.8.2.3 | Other | Information not present in EudraCT |
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 | 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
| |
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 |