E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
treatment of anemic myelofibrosis patients |
|
E.1.1.1 | Medical condition in easily understood language |
myelofibrosis patients with anemia |
|
E.1.1.2 | Therapeutic area | Diseases [C] - Cancer [C04] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10074692 |
E.1.2 | Term | Post essential thrombocythaemia myelofibrosis |
E.1.2 | System Organ Class | 100000004864 |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10074691 |
E.1.2 | Term | Post polycythaemia vera myelofibrosis |
E.1.2 | System Organ Class | 100000004864 |
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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 determine the spleen length response rate at Week 24 |
|
E.2.2 | Secondary objectives of the trial |
- To determine the spleen length response rate at Week 48 - To evaluate the effect of ruxolitinib on spleen length - To evaluate the effect of ruxolitinib on symptoms - To evaluate the effect of ruxolitinib on Patient Global Impression of Change (PGIC) - To evaluate the effect of ruxolitinib on transfusion requirements |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- Male or female patients aged ≥ 18 years of age. - Patients must be diagnosed with PMF, according to the 2016 revised International Standard Criteria (Arber et al, 2016), PPV MF or PET-MF (Barosi 2008), irrespective of JAK2 mutation status. - Patients with palpable splenomegaly that is equal to or greater than 5 cm below the left costal margin. - Patients with a hemoglobin less than 10 g/dL - Patients with a history of transfusions must have a documented transfusion record in the previous 12 weeks to baseline. - Patients with a peripheral blood blast percentage count of < 10%.
Additional inclusion criteria as per full protocol may apply. |
|
E.4 | Principal exclusion criteria |
- Patients with prior treatment with any JAK1 or JAK2 inhibitor. - Patients with inadequate bone marrow reserve at baseline visit asdemonstrated by at least one of the following: ANC that is ≤ 1,000/μL. Platelet count that is <50,000/μL without the assistance of growth factors, thrombopoietic factors or platelet transfusions. Hemoglobin count that is ≤ 6.5 g/dL despite transfusions. - Patients with severely impaired renal function - Patients with inadequate liver function - Patients being treated concurrently with a strong (potent) systemic inhibitor or inducer of CYP3A4 at the time of Screening - Acute viral hepatitis or active chronic hepatitis B or C infection. - History of progressive multifocal leuko-encephalopathy (PML)
Additional exclusion criteria as per full protocol may apply. |
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E.5 End points |
E.5.1 | Primary end point(s) |
Proportion of patients achieving a 50% reduction in spleen length at Week 24 |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
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E.5.2 | Secondary end point(s) |
- Proportion of patients achieving a 50% reduction in spleen length at week 48 - Percent change from baseline in spleen length over time - Summary of the Modified MFSAF v2.0 and MF-7 over time - PGIC at each visit where measured - Summary of transfusions over time |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
- Week 48 - Week 48 - Week 48 - Week 48 - Week 48
|
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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 | No |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | Yes |
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 | Yes |
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.8.2.4 | Number of treatment arms in the trial | 1 |
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 | 18 |
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 | No |
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 |
Austria |
Belgium |
Bulgaria |
Canada |
Chile |
Germany |
Greece |
Hungary |
Italy |
Japan |
Russian Federation |
Spain |
Turkey |
|
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
|
The end of the study (last patient last visit) will occur after completion of the last follow up visit of the last patient on treatment with ruxolitinib. |
|
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 1 |
E.8.9.1 | In the Member State concerned months | 11 |
E.8.9.1 | In the Member State concerned days | 1 |
E.8.9.2 | In all countries concerned by the trial years | 1 |
E.8.9.2 | In all countries concerned by the trial months | 11 |