The purpose of this study is to evaluate the efficacy and safety of ruxolitinib versus anagrelide in subjects with essential thrombocythemia who are resistant to or intolerant of hydroxyurea.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Enrollment
12
Ruxolitinib administered orally twice daily (BID) at the protocol-defined starting dose.
Anagrelide administered orally at a starting dose of 1 mg BID.
Anagrelide-placebo administered orally BID
Proportion of Subjects Who Achieve Platelet and White Blood Cell (WBC) Control
Defined as proportion of subjects who achieve a simultaneous reduction of platelet counts to \< 600 × 10\^9/L with a reduction of WBC counts to \< 10 × 10\^9/L for at least 80% of biweekly measurements for a consecutive 12-week period between Weeks 32 and 52.
Time frame: 52 weeks
Number of Participants With Treatment Emergent Adverse Events (TEAEs)
Adverse events reported for the first time or worsening of a pre-existing event after first dose of study drug/treatment.
Time frame: Baseline through the end of randomized period -up to 14 months per participant
Proportion of Subjects Who Achieve Complete Remission or Partial Remission
Defined as proportion of subjects who achieve CR or PR at Week 32 based on European LeukemiaNet (ELN) 2013 response criteria. Per ELN criteria: Complete Remission: durable resolution of disease related signs and symptoms, durable blood count normalization, absence of hemorrhagic or thrombotic events, absence of signs of progressive disease and bone marrow histological remission including disappearance of megakaryocyte hyperplasia and absence of reticulin fibrosis \>Grade 1. Partial remission: durable resolution of disease related signs and symptoms, durable blood count normalization, absence of hemorrhagic or thrombotic events, absence of signs of progressive disease, persistance of megakaryocyte hyperplasia. No response: any response that does not satisfy partial remission. Progressive Disease: transformation in PET-MF, MDS or acute leukemia.
Time frame: 32 weeks
Time to Treatment Discontinuation
Defined as the time when treatment is discontinued
Time frame: 98 weeks
Duration of Response
Defined as measurement of response from the onset of response to the loss of response for responders.
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Ruxolitinib-placebo administered orally BID.
Mayo Clinic
Phoenix, Arizona, United States
Pacific Shores Medical Group
Long Beach, California, United States
University of Southern California
Los Angeles, California, United States
Ventura County Hematology-Oncology Specialists
Oxnard, California, United States
Compassionate Cancer Care Medical Group
Riverside, California, United States
Innovative Clinical Research Institute
Whittier, California, United States
Bond Clinic, PA
Winter Haven, Florida, United States
Tift Regional
Tifton, Georgia, United States
Edward Cancer Center
Naperville, Illinois, United States
North Shore Cancer Research Association-Skokie
Skokie, Illinois, United States
...and 16 more locations
Time frame: 142 weeks
Proportion of Subjects Who Achieve Reduction of Platelet Counts to < 600 × 10^9/L
Defined as Proportion of subjects who achieve reduction of platelet counts to \< 600 × 10\^9/L for at least 80% of biweekly measurements for a consecutive 12-week period between Weeks 32 and 52.
Time frame: Between 32 and 52 weeks
Proportion of Subjects Who Achieve a Reduction of WBC Counts to < 10 × 109/L
Defined as Proportion of subjects who achieve a reduction of WBC counts to \< 10 × 109/L for at least 80% of biweekly measurements for a consecutive 12-week period between Weeks 32 and 52.
Time frame: 52 weeks