Ruxolitinib for the Treatment of Chronic Myelomonocytic Leukemia (CMML): A Phase 2 Expansion
Condition: Chronic Myelomonocytic Leukemia; Leukemia
MeSH terms: Leukemia; Leukemia, Myelomonocytic, Acute; Leukemia, Myelomonocytic, Chronic; Leukemia, Myelomonocytic, Juvenile
Intervention: Ruxolitinib
Intervention description: Ruxolitinib 5 mg tablets, 4 per dose
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Intervention model: Sequential Assignment
Masking: None (Open Label)
Enrollment: 29.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No
Keywords: CMML

Summary:

      This study is to find out if treating Chronic Myelomonocytic Leukemia (CMML) with a study
      drug (ruxolitinib) can improve outcomes of patients with CMML.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Confirmed diagnosis of Chronic Myelomonocytic Leukemia (CMML)using the World Health
             Organization (WHO) classification.

          -  18 years of age or older at the time of obtaining informed consent.

          -  Must be able to adhere to the study visit schedule and other protocol requirements.

          -  Participants must be able to provide adequate BM aspirate and biopsy specimens for
             histopathological analysis and standard cytogenetic analysis during the screening
             procedure.

          -  An Eastern Cooperative Oncology Group (ECOG) performance status score of 0, 1, or 2 is
             required.

          -  Women of childbearing potential must agree to use two reliable forms of contraception
             simultaneously or to practice complete abstinence from heterosexual intercourse 1) for
             at least 28 days before starting study drug; 2) while participating in the study; and
             3) for at least 28 days after discontinuation from the study.

          -  Must understand and voluntarily sign an informed consent form.

          -  Must have a life expectancy of greater than 3 months at time of screening.

          -  Must have symptomatic splenomegaly and/or an Myeloproliferative Neoplasms Symptom
             Assessment Form Total Symptom Score >17.

        Exclusion Criteria:

          -  Any of the following lab abnormalities: Platelet count of less than 35,000/uL,
             Absolute Neutrophil Count (ANC) less than 250/uL, Serum Creatinine ≥ 2.0, Serum total
             bilirubin >1.5x ULN

          -  Use of cytotoxic chemotherapeutic agents, or experimental agents (agents that are not
             commercially available) for the treatment of CMML within 28 days of the first day of
             study drug treatment.

          -  Prior history of metastatic malignancy in past 2 years

          -  Any serious medical condition or psychiatric illness that will prevent the subject
             from signing the informed consent form or will place the subject at unacceptable risk
             if he/she participates in the study.

          -  Concurrent use of Granulocyte-Macrophage Colony Stimulating Factor (GM-CSF).
             Granulocyte Colony Stimulating Factor (G-CSF) could be used for the short-term
             management of neutropenic infection. Stable doses of erythropoietin stimulating agents
             that were started >8 weeks from first ruxolitinib dose or corticosteroids that were
             being administered prior to screening are allowed.

          -  Uncontrolled current illness including, but not limited to ongoing or active
             infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac
             arrhythmia, or psychiatric illness/social situations that would limit compliance with
             study requirements.

          -  Pregnant women are excluded from this study because ruxolitinib has not been studied
             in pregnant participants. Because there is an unknown but potential risk for adverse
             events in nursing infants secondary to treatment of the mother with ruxolitinib,
             breastfeeding should be discontinued if the mother is treated with ruxolitinib.