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 | LLT |
E.1.2 | Classification code | 10028576 |
E.1.2 | Term | Myeloproliferative disorder |
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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 |
To assess the benefit/risk profile of cytoreductive therapy with phlebotomy and/or hydroxyurea (HU) aimed at maintaining hematocrit (HCT) < 45% Vs. maintaining HCT in the range of 45-50% in patients with PV treated at the best of recommended therapies (e.g., low dose of aspirin when indicated and adequated control of standard cardiovascular risk factors). No specific drug is recommended by the protocol for the two study arms: the choice to use phlebotomy and/or any cytoreductive drug is left to the Investigator. |
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E.2.2 | Secondary objectives of the trial |
Assessment of the full benefit/risk profile of experimental treatments in terms of efficacy and safety endpoints |
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E.2.3 | Trial contains a sub-study | Yes |
E.2.3.1 | Full title, date and version of each sub-study and their related objectives |
ALTRI SOTTOSTUDI: PROSPECTIVE EVALUATION OF THE PREDICTIVE ROLE OF JAK2V617F ALLELE BURDEN ON CLINICAL OUTCOMES IN PATIENTS WITH POLYCYTHEMIA VERA
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E.3 | Principal inclusion criteria |
Age ³ 18 years Diagnosis of PV according to WHO criteria Ability and willingness to comply with all study requirements Written and signed informed consent |
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E.4 | Principal exclusion criteria |
Pregnant or lactating women or women of childbearing potential who are not protected from pregnancy by an accepted method of contraception Known hypersensitivity or contraindication to study treatments Significant liver (AST or ALT > 2.5 times ULN) or renal disease (creatinine > 2 mg/ml) Presence of any life-threatening condition or of any disease (e.g. cancer) that is likely to significantly shorten life expectancy History of active substance or alcohol abuse within the last year Any condition that in the opinion of the investigator would jeopardize the evaluation of efficacy or safety or be associated with poor adherence to the protocol - Baseline and follow-up (F-UP) visits schedule and assessments |
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E.5 End points |
E.5.1 | Primary end point(s) |
To demonstrate that maintaining HCT < 45% (either with phlebotomy or HU) (standard cytoreduction) is more effective than maintaining HCT in the range of 45-50% (either with phlebotomy or HU) (experimental cytoreduction) in the reduction of CV deaths plus thrombotic events (stroke, acute coronary syndrome [ACS], transient ischemic attack [TIA], pulmonary embolism [PE], splanchnic thrombosis, deep vein thrombosis [DVT], and any other clinically relevant thrombotic event) in patients with PV treated at the best of recommended therapies (e.g., low dose of aspirin when indicated and adequated control of standard cardiovascular risk factors). |
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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 | Yes |
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 | 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 | Yes |
E.8.2.3.1 | Comparator description |
salassi; - same IMP used at different dosage |
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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 | 35 |
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
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Interruzione prematura dello studio per problemi di safety sulla base dell'indicazione del Data Safety and Monitoring Board. |
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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 | 7 |
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 | 7 |
E.8.9.2 | In all countries concerned by the trial months | 0 |