Polycythemia Vera MedDRA version: 20.1 Level: LLT Classification code 10036061 Term: Polycythemia vera System Organ Class: 100000004864
Conditions
Interventions
Trade Name: Jakavi
Pharmaceutical Form: Tablet
INN or Proposed INN: Ruxolitinib
Other descriptive name: RUXOLITINIB
Concentration unit: mg milligram(s)
Concentration type: range
Concentration number:
Sponsors
Zealand University hospital
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Age = 18 years AND 2. A confirmed diagnosis of PV according to the WHO 2016 criteria AND 3. Biochemical evidence of active disease as defined by a) Elevated hematocrit and/or red cell count and/or b) Leucocyte count > 10 x 109/L and /or c) Platelet count > 400 x 109/L. OR 4. Hypermetabolic symptoms such as weight loss (> 10% within 6 months), night sweats and subfebrilia (temperature> 38 º C for more than 2 weeks without signs of infection OR 5. Pruritus OR 6. Symptomatic splenomegaly OR 7. Presence of previous thrombosis 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 15 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 10
Exclusion criteria
Exclusion criteria: 1. Patients of childbearing potential without a negative pregnancy test prior to initiation of study drug. Participants must use contraceptives during the entire study period*. 2. Other active malignancy within the past 5 years (not including non-melanoma skin cancer and prostate cancer without need for treatment). 3. ECOG function scores> / = 3 4. Serum creatinine more than 2 x ULN 5. Total serum bilirubin greater than 1.5 x ULN 6. Plasma ALAT more than 3 x ULN 7. Former psychiatric disorder (depression diagnosed by a psychiatrist) 8. Uncontrolled metabolic disease. 9. Severe heart disease (heart failure NYHA class 3-4). 10. Severe myelosuppression. (WBC<1.5 Mill/L, PLA<100) 11. Chronic hepatitis with decompensated cirrhosis. 12. Chronic hepatitis in patients who have been or recently (within 6 months) has been treated with immunosuppressive drugs except for corticosteroid treatment. 13. Epilepsy and / or other serious CNS disorders.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: Safety;Secondary Objective: Efficacy;Primary end point(s): 1.Safety Profile: adverse events (AE), serious AEs (SAE), withdrawal due to AEs/SAE;Timepoint(s) of evaluation of this end point: 24 months | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1. Safety profile of COMBI therapy in patients with polycythemia Vera after 12 months as assessed by AE’s, SAE’s and withdrawal AE’s/SAE’s. 2. Efficacy Profile as assessed by normalization of red blood cell values, white blood cell counts (WBC) and platelet counts (PLA); response rates (see below); changes in the JAK2-V617F allele burden; proportion of patients suffering thrombosis and/or hemorrhage; proportion of patients achieving no need of phlebotomies; proportion of patients achieving normal spleen size; proportion of patients with progression of disease*; progression free survival. ;Timepoint(s) of evaluation of this end point: 12 and 24 months | — |
Countries
Denmark
Contacts
Public ContactProject manager
Zealand University Hospital
Outcome results
None listed