Polycythemia vera (PV)
Conditions
Interventions
Single arm:Take ruxolitinib orally
Sponsors
Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College
Eligibility
Sex/Gender
All
Age
18 Years to No maximum
Inclusion criteria
Inclusion criteria: 1.Male or female patients aged 18 years or older at the time of screening who provide written informed consent. 2.Diagnosed with PV for at least 24 weeks prior to Screening according to the 2016 World Health Organization criteria 3.Have a treatment history for PV with resistance or intolerance to HU or IFN-a. (1)Resistance or intolerance to HU was defined by exhibiting at least one of the following five criteria: HU Resistance, defined at least 12 weeks into a course of HU therapy at a dose of at least 2 grams/day OR at the subjects maximally tolerated dose if that dose is less than 2 grams/day: Need for phlebotomy to keep HCT 400 x10^9/L AND WBC >10 x10^9/L, OR ?Failure to reduce massive splenomegaly (defined as spleen extending greater than 10 cm below the costal margin) by more than 50%, as measured by palpation, OR failure to completely relieve symptoms related to splenomegaly, OR HU Intolerance: Absolute neutrophil count (ANC) 400 x 10^9/L AND WBC > 10 x 10^9/L, OR Failure to achieve a > 50% reduction in palpable splenomegaly measuring > 10 cm from the left costal margin, OR IFN-a Intolerance: ANC = 1.5 x 10^9/L and PLT >= 100 x 10^9/L at Screening. 5.Peripheral blood blast count of 0% at Screening. 6.Subjects with an Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1 or 2 at Screening. 7.Women of childbearing potential (WOCBP) who are willing to use highly effective contraceptive measures during the study period.
Exclusion criteria
Exclusion criteria: 1.Pregnant or nursing (lactating) women. 2.Subjects with inadequate liver or renal function at Screening as demonstrated by: (1)Encephalopathy Grade 2 or more as per Child-Pugh System. (2)Known hepatocellular disease (e.g., hepatitis B or C, cirrhosis or other hepatocellular disease) (3)Direct bilirubin >= 2 x upper limit of laboratory normal (ULN). (4)Alanine aminotransferase (ALT) > 2.5 x ULN. (5)MDRD-eGFR < 30 mL/min/1.73m2 or on dialysis. 3.Subjects with impairment of gastrointestinal (GI) function or GI disease that may significantly alter the absorption of oral ruxolitinib (e.g., ulcerative diseases, uncontrolled nausea, vomiting, diarrhea, malabsorption syndrome, and small bowel resection). 4.Subjects with unresolved active severe infections (e.g. clinically significant bacterial, fungal, parasitic or viral infections). 5.Subjects with diagnosed primary immunodeficiency syndromes such as X-Linked Agammaglobulinemia and common Variable Immune Deficiency. 6.Subjects with an active malignancy over the previous 5 years except treated cervical intraepithelial neoplasia, basal cell carcinoma of the skin, or squamous cell carcinoma of the skin, with no evidence for recurrence in the past 3 years. 7.Subjects with clinically significant cardiac disease (NYHA Class III or IV). 8.Subjects receiving PEG-IFN-alpha-2a within 5 weeks of Screening or having a prior history of 32P therapy. 9.Subjects treated concurrently with a potent systemic inhibitor of CYP3A4 at the time of Screening (ketoconazole, clarithromycin, itraconazole, nefazodone or telithromycin). 10.Subjects who have previously received treatment with a JAK inhibitor. 11.Subjects being treated concurrently with any investigational agent or prior participation in an investigational study within 30 days prior to the first dose of study drug or within 5-half-lives of the investigational product, whichever is longer. 12.Subjects with active alcohol or drug addiction that would interfere with their ability to comply with the study requirements. 13.Subjects with an uncontrolled intercurrent illness or any concurrent condition that, in the investigators opinion, would jeopardize the safety of the subject or compliance with the protocol.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Complete hematological response; | — |
Secondary
| Measure | Time frame |
|---|---|
| Spleen size assessment;Myeloproliferative Neoplasm-SAF (MPN-SAF TSS) Assessment; | — |
Countries
China
Contacts
Public ContactXiao Zhijian
Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College
Outcome results
None listed