Ruxolitinib for Polycythemia vera

Inclusion criteria

• {"criterion_text":"- The participant must meet criteria 1–3 AND at least one of 4–8"}
• {"criterion_text":"- 1. Age ≥ 18 years AND"}
• {"criterion_text":"- 2. A confirmed diagnosis of PV according to the WHO 2016 criteria AND"}
• {"criterion_text":"- 3. A negative test for active or latent tuberculosis (from within the last month)"}
• {"criterion_text":"- 4. 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"}
• {"criterion_text":"- 5. 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"}
• {"criterion_text":"- OR 6. Pruritus OR"}
• {"criterion_text":"- 7. Symptomatic splenomegaly OR"}
• {"criterion_text":"- 8. Presence of previous thrombosis"}

Exclusion criteria

• {"criterion_text":"- 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*."}
• {"criterion_text":"- 2. Other active malignancy within the past 5 years (not including non-melanoma skin cancer and prostate cancer without need for treatment)."}
• {"criterion_text":"- 3. ECOG function scores> / = 3"}
• {"criterion_text":"- 4. Serum creatinine more than 2 x ULN"}
• {"criterion_text":"- 5. Total serum bilirubin greater than 1.5 x ULN"}
• {"criterion_text":"- 6. Plasma ALAT more than 3 x ULN"}
• {"criterion_text":"- 7. Former psychiatric disorder (depression diagnosed by a psychiatrist)"}
• {"criterion_text":"- 8. Uncontrolled metabolic (endocrinological) disease."}
• {"criterion_text":"- 9. Severe heart disease (heart failure NYHA class 3-4)."}
• {"criterion_text":"- 10. Severe myelosuppression. (WBC<1.5 Mill/L, PLA<100)"}
• {"criterion_text":"- 11. Chronic hepatitis with decompensated cirrhosis."}
• {"criterion_text":"- 12. Chronic hepatitis in patients who have been or recently (within 6 months) has been treated with immunosuppressive drugs except for corticosteroid treatment."}
• {"criterion_text":"- 13. Epilepsy and / or other serious CNS disorders."}
• {"criterion_text":"- *Spiral, birth control pills, implants, transdermal patch, vaginal ring or transdermal injection. Sterile / infertile subjects are exempt from the use of contraception. To be considered sterile or infertile, one must generally be surgically sterilized (vasectomy, bilateral tubectomy, hysterectomy or ovariectomy) or be postmenopausal, defined as absent menstruation for at least 12 months before study enrollment."}

Primary endpoints

• {"endpoint_text":"- Safety Profile: relevant adverse events (AE), serious AEs (SAE), withdrawal due to AEs/SAE","definition_or_measurement_approach":"Assessment of relevant adverse events (AE), serious AEs (SAE), and withdrawals due to AEs/SAE as reported during the study"}

Secondary endpoints

• {"endpoint_text":"- 1. Safety Profile: adverse events (AE), serious AEs (SAE), withdrawal, AEs/SAEs 2. Efficacy Profile: a) Proportion of patients achieving complete hematological remission (HCT < 45, PLA ≤400 and WBC < 10) after 3, 6, 8, 9, 12, 18 and 24 months. b) Remission rates at 12 and 24 months. c) JAK2V617F allele burden measured at baseline and after 3,6,12 and 24 months. d) Proportion of patients suffering thrombosis and/or hemorrhage at 12 at 24 months. with more e)-j)","definition_or_measurement_approach":"Includes AE/SAE reporting and efficacy measures: complete hematological remission defined as HCT <45, PLA ≤400 and WBC <10 at specified timepoints (3,6,8,9,12,18,24 months); remission rates at 12 and 24 months; JAK2V617F allele burden measured at baseline and at 3, 6, 12 and 24 months; proportion with thrombosis/hemorrhage at 12 and 24 months"}

Denmark

Earliest CTIS Part Ii Submission Date

23-10-2024

Latest Decision Or Authorization Date

04-11-2024

Processing Time Days

12

Number Of Sites

1

Number Of Participants

25

Primary sponsor

Full Name

Region Sjaelland

Organisation Type

Hospital/Clinic/Other health care facility

Country Of Registered Address

Denmark

Third parties

• {"country":"Denmark","full_name":"Frederiksberg Hospital","duties_or_roles":"sponsorDuties code 1","organisation_type":"Hospital/Clinic/Other health care facility"}