treatment of anemic myelofibrosis patients MedDRA version: 20.0 Level: LLT Classification code 10074692 Term: Post essential thrombocythaemia myelofibrosis System Organ Class: 100000004864 MedDRA version: 20.0 Level: LLT Classification code 10074691 Term: Post polycythaemia vera myelofibrosis System Organ Class: 100000004864
Conditions
Sponsors
Novartis Pharma Services AG
Eligibility
Sex/Gender
All
Inclusion criteria
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 =65 years) yes F.1.3.1 Number of subjects for this age range 30
Exclusion criteria
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To determine the spleen length response rate at Week 24; Secondary Objective: - 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 ;Primary end point(s): Proportion of patients achieving a 50% reduction in spleen length at Week 24;Timepoint(s) of evaluation of this end point: Week 24 | — |
Secondary
| Measure | Time frame |
|---|---|
| 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 ; Timepoint(s) of evaluation of this end point: - Week 48 - Week 48 - Week 48 - Week 48 - Week 48 | — |
Countries
Austria, Belgium, Bulgaria, Canada, Chile, Germany, Greece, Hungary, Italy, Japan, Russian Federation, Spain, Turkey
Contacts
Public ContactDrug Regulatory Affairs
Novartis Pharma GmbH
Outcome results
None listed