E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Immune thrombocytopenic purpura (ITP) |
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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 | LLT |
E.1.2 | Classification code | 10021245 |
E.1.2 | Term | Idiopathic thrombocytopenic purpura |
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E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
Primary objective: To assess in both treatment arms the rate of splenectomy at 1.5 years.
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E.2.2 | Secondary objectives of the trial |
Secondary objectives: 1. Response rates. 2. Relapse rate and duration of response. 3. Mortality rate. 4. Complications` rate: bleeding, infection and thromboembolic events. 5. Cost-effectiveness analysis. 6. Consumption of corticosteriods and IVIG in both arms. 7. Immune-reconstruction at 1.5 years.
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Inclusion criteria (for randomisation): 1. ITP with platelet count <30 x 10e9 /l or 30-50 x 10e9 /l if a higher platelet count is considered necessary because of any of the following: a. Concomitant medical illness predisposing to bleeding (gastric ulcer, bleeding diathesis, previous history of bleeding). b. Concomitant medical condition requiring aspirin and/or clopidogrel intake or anticoagulation. c. Persistent bleeding manifestations despite platelets > 30 x 10e9 /l. d. Other patient related factors necessitating higher platelet count as occupation, hobby, psychological intolerability. e. Age >75 years. 2. Previous treatment with corticosteroids for a minimum duration of 2 weeks as recommended by the protocol (prednisone or prednisolone 1-2 mg/kg/day) with either no response (i.e. failed to achieve an initial increase in Platelet count >30 x 10e9 /l) or relapse (Platelet count falls to < 30 x 10e9 /l) during the dose tapering period or after discontinuation of corticosteroids. 3. Subject is > or equal = 18 years. 4. Subject has signed and dated written informed consent. 5. Subject is able to understand and comply with protocol requirements and instructions, and intends to complete the study as planned. 6. Females in child-bearing age should accept to use of contraceptive means for at least 6 months following the administration of the study drugs.
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E.4 | Principal exclusion criteria |
Exclusion criteria: 1. Previous splenectomy, chemotherapy, treatment with anti-D Ig, rituximab, or immune-suppressive treatments other than corticosteroids, Dapsone or Danazol. 2. Underlying malignancy or previous history of malignancy in the past 5 years (except skin carcinoma). 3. Pregnancy and lactation. 4. Not willing to participate in the study. 5. Expected survival of < 2 years. 6. Known intolerance to murine antibodies. 7. Females in child-bearing age not willing to use contraception for 6 months. 8. HIV/AIDS-, Hepatitis -B virus antigen positive- or Hepatitis -C virus antibodies positive- patients. 9. Patients with Systemic Lupus Erythematosus (SLE) (> or = 4 of the American College of Rheumatology Criteria) (Tan et al, 1982;Hochberg, 1997). 10. Patients currently involved in another clinical trial with evaluation of drug treatment
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E.5 End points |
E.5.1 | Primary end point(s) |
Primary endpoint: Splenectomy during the follow-up period after randomisation.
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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 | Yes |
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 | 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 | 2 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 9 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | Information not present in EudraCT |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
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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 of trial will be when thelast treated subjectd has been followed up to 1.5 years |
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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 | 2 |
E.8.9.1 | In the Member State concerned months | 6 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 4 |
E.8.9.2 | In all countries concerned by the trial months | 10 |