E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Polycythaemia Vera & Essential Thrombocythaemia |
|
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 | 10015494 |
E.1.2 | Term | Essential thrombocythemia |
|
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 | 10036061 |
E.1.2 | Term | Polycythemia vera |
|
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 safety and efficacy of erlotinib in the treatment of patients with JAK2-positive polycythaemia vera (PV) and essential thrombocythaemia (ET). To evaluate if erlotinib as monotherapy for patients with JAK2-positive PV and ET is followed by a decline in clonal myeloproliferation as assessed by conventional disease activity parameters (a decrease in the need of phlebotomy (PV), leucocyte and platelet count) To study if treatment with erlotinib influcences the JAK2 mutation status as assessed by quantitative PCR |
|
E.2.2 | Secondary objectives of the trial |
To perform integrated gene expression (global and focused) profiling and proteome studies (serum-based and phosphoproteomics) before and during treatment with erlotinib in order to describe in detail the various (heterogenous) response patterns at the molecular level. To study changes in bone marrow morphology before and after treatment with erlotinib. To study in vivo granulocyte, platelet and endothelial activation before and during treatment with erlotinib. |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- Male or female patient > 18 years of age - A confirmed diagnosis of JAK2-positive PV and ET (according to the Nordic Guidelines) - Biochemical evidence of active disease as defined by a) A need for phlebotomy within the last 3 months b) A leucocyte count > 10 Mia/l in the absence of infection or inflammation (normal CRP) (PV/ET) c) A platelet count > 400 Mia/l in the absence of infection or inflammation (normal CRP) (PV/ET) |
|
E.4 | Principal exclusion criteria |
- A platelet count > 1000 Mia/l (a need for cytoreduction in platelet count) - Patients of childbearing potential without a negative pregnancy test prior to initiation of study drug - ECOG Performance Status Score >/= 3 - Serum creatinine more than 2 x's the ULN - Total serum bilirubine more than 1.5 x's the ULN - Plasma ASAT/ALAT more than 3 x's the ULN - Interpheron alpha within 1 week of day 1 - Hydroxyurea within 1 week of day 1 - Anagrelide within 1 week of day 1 - Any other investigational drug within 28 days of day 1 - Grade 3/4 cardiac disease - Any serious concomitant disease |
|
E.5 End points |
E.5.1 | Primary end point(s) |
1) Normalisation of the haematocrit or at least >50% reduction in the need of phlebotomy 2) Platelet count < 400 Mia/l or > 50% reduction of platelet count 3) Leucocyte count < 10 Mia/l. |
|
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 | 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) | No |
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
| 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 | Information not present in EudraCT |
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
|
By the end of follow up after 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 | 2 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | 0 |