primary myelofibrosis (PMF), post polycythemia vera myelofibrosis (PPV MF) or Post essential thrombocythemia myelofibrosis (PET-MF)
Conditions
Interventions
Product Name: ruxolitinib
Product Code: INC424
Pharmaceutical Form: Tablet
INN or Proposed INN: ruxolitinib
Current Sponsor code: INC301
Sponsors
Novartis Pharmaceuticals UK Ltd
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Patients must give written informed consent according to local guideliens prior to any screening procedures 2. Patients must not be eligible for another ongoing INC424 clinical trial 3. Male or female patients aged = 18 years of age 4. Patients must be diagnosed with PMF, PPV MF or PET MF according to the WHO criteria 2008 5. Patients with PMF requiring therapy must be classified as high risk or intermediate risk level 2 or intermediate risk level 1 with enlarged spleen 6. Patients with Intermediate-1 and splenomegaly must have a palpable spleen measuring 5 cm or greater from the costal margin to the point of greatest splenic protrusion See protocol for further criteria Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 33 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Patients eligible for hematopoietic stem cell transplantation 2. Patients with a history of malignancy in the past 3 years, except for treated early stage squamous or basal cell carcinoma in situ 3. Patients undergoing treatment with haematopoietic growth factor receptor agonists at any time within 2 weeks prior to screening or 4 weeks prior to baseline 4. Patients currently participating in COMFORT-I and COMFORT -II trials 5. Patients receiving any medications listed in the "prohibited medications" listing 6. Impairment of GI function or GI disease that may significantly alter the absorption of oral INC424 7. Patients with cardiac disease which may jeopardize the safety of the patient or compliance with the protocol 8. Patients with currently uncontrolled or unstable angina, rapid or paroxysmal fibrillation or recent myocardial infarction or acute coronary syndrome 9. Patients with clinically significant infections (for further details see protocol) 10. Patients with known active hepatitis A, B, C or who are HIV -positive 11. Patients with coagulation parameters =1.5 12. Pregnant or nursing women See protocol for further criteria
Design outcomes
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): To assess the best overall response rate of INC424 in patients with PMF, PPV-MF or PET-MF, as evaluated by the investigator To collect Quality of Life (QoL) information To document Medical Resource Utilization (MRU) To collect safety data ;Timepoint(s) of evaluation of this end point: Monthly for the first 3 months, then every 3 months and at study discontinuation | — |
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the efficacy of daily oral doses of 15mg BID or 20mg BID of INC424 in patients with PMF, PPV-MF, or PET-MF, based on the proportion of patients experiencing treatment success at the 48 week timepoint.; Secondary Objective: To document the best overall response rate to INC424 in patients with PMF, PPV-MF, or PET-MF as evaluated by the investigator To collect quality of life (QoL) information To document medical resource utilization ;Primary end point(s): Treatment success measured by change in spleen size by palpation and/or change in myelofibrosis symptoms assessment form (MF-SAF).;Timepoint(s) of evaluation of this end point: Monthly for the first 3 months, then every 3 months and at study discontinuation | — |
Countries
United Kingdom
Contacts
Public ContactMedical Collaboration Centre
Novartis Pharmaceuticals UK Ltd
Outcome results
None listed