E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
ITP Primary Immune Thrombocytopenic Purpura in newly diagnosed untreated adult patients |
|
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 |
|
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 |
To evaluate the role of therapy intensification in newly diagnosed untreated primary ITP adult patients with high-dose DXM (HD-DXM), in terms of improvement of response at six months after initial response, in comparison with standard doses of prednisone (PDN) |
|
E.2.2 | Secondary objectives of the trial |
Rate of initial response Quality of response per arm Rate of final responses Rate of bleeding events Rate of resumed response with HD-DXM in non responder patients or patients who have lost response (for patients of ARM A only) Time to platelet number increase until a hemostatically effective level is reached and/or disappearance of bleeding symptoms. Rate of persistent response Rate of rescue interventions Rate of eligible patients for splenectomy Rate of patients undergone to splenectomy Rate of patients who develop connective tissue diseases or underlying hematological diseases (myelodisplastic syndromes, chronic lymphoproliferative disesases, others) Patients self reported Quality of Life |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Signed written informed consent according to IGH/EU/GCP and national local laws Newly diagnosed untreated ITP adult patients Age > 18 < 80 years Platelet count <20x109/L Platelet count > 20 x109/L and <50x109/L plus bleeding with score > 8 (according to grading scale at paragraph 7.1) Baseline Quality of Life evaluation questionnaire filled in |
|
E.4 | Principal exclusion criteria |
Active malignancy at time of study entry Steroids administration (PDN <1mg/Kg/day) for more than 2 days before randomization Concomitant treatment with anti-platelet and or anti-coagulant drugs Concomitant severe psychiatric disorders Not confirmed diagnosis of ITP for o *Positivity of autoimmunity markers: antinucleus (1:80), anti-tireoglobulin, anti-tireoperoxidase, anti-cardiolipin (≥ 40 GPL UmL), anti-b2glycoprotein (≥ 40 IgG U/mL) antibodies, Lupus Anticoagulant (KCT ratio, dRVVT ratios 1.5 times the upper normal limit ), direct antiglobulin test (DAT ). o Presence of autoimmune hemolytic anemia o Presence of connective tissue disease Women who are pregnant or breastfeeding Hypertension, cardiovascular diseases and diabetes requiring treatment Liver and kidney function impairment (creatinine, ALT, AST >2 times upper normal limit) HCVAb, HIVAb, HBsAg, HBcAb seropositive status Chronic liver disease Documented viral illness by the positivity of IgM, or vaccination both occurred one month before diagnosis Intake of drugs not previously taken within one week before diagnosis Bleeding score 15 due to ICH or to GI bleeding (according to grading scale at paragraph 7.1, Tab. 3) Active gastric ulcer |
|
E.5 End points |
E.5.1 | Primary end point(s) |
Final response (CR+PR+MR, see paragraph 8.1) rate at day +180 from evaluation of initial response |
|
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 | Yes |
E.6.13.1 | Other scope of the trial description |
|
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 | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
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) | Yes |
E.8.2.2 | Placebo | No |
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 | 50 |
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 | 5 |
E.8.9.1 | In the Member State concerned months | 8 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 5 |
E.8.9.2 | In all countries concerned by the trial months | 8 |