Clinical Trials /

Ruxolitinib for the Treatment of Chronic Myelomonocytic Leukemia (CMML): A Phase 2 Expansion

NCT03722407

Description:

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

Related Conditions:
  • Chronic Myelomonocytic Leukemia
Recruiting Status:

Recruiting

Phase:

Phase 2

Trial Eligibility

Document

Title

  • Brief Title: Ruxolitinib for the Treatment of Chronic Myelomonocytic Leukemia (CMML): A Phase 2 Expansion
  • Official Title: A Sequential Two-Stage Dose Escalation Study to Evaluate the Safety and Efficacy of Ruxolitinib for the Treatment of Chronic Myelomonocytic Leukemia (CMML): A Phase 2 Expansion

Clinical Trial IDs

  • ORG STUDY ID: MCC-19727
  • NCT ID: NCT03722407

Conditions

  • Chronic Myelomonocytic Leukemia
  • Leukemia

Interventions

DrugSynonymsArms
RuxolitinibJakafiRuxolitinib

Purpose

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

Trial Arms

NameTypeDescriptionInterventions
RuxolitinibExperimentalAll patients will be given their first dose of oral Ruxolitinib, 20 mg at first scheduled visit. After that dose and on all other days patients will self-administer oral Ruxolitinib at a dose of 40 mg daily divided into two equal doses approximately 12 hours apart. Patients will be treated for a total of 16 weeks. After treatment, patients will be followed monthly.
  • Ruxolitinib

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.
      
Maximum Eligible Age:N/A
Minimum Eligible Age:18 Years
Eligible Gender:All
Healthy Volunteers:No

Primary Outcome Measures

Measure:Overall Response
Time Frame:At week 16
Safety Issue:
Description:Proportion of participants achieving clinical benefit defined as hematologic improvement, complete remission, partial remission, or stable disease by the International Working Group Myelodysplastic/Myeloproliferative Neoplasms (MDS/MPN) Criteria.

Secondary Outcome Measures

Measure:Time to Acute Myeloid Leukemia (AML) transformation
Time Frame:Every 6 months after conclusion of treatment until end of study (24 months)
Safety Issue:
Description:Time to AML transformation according to World Health Organization (WHO) Critieria
Measure:Overall Survival
Time Frame:Up to 2 years
Safety Issue:
Description:Overall survival will be from first dose of study drug until failure or death from any cause.
Measure:Duration of Response
Time Frame:Up to 2 years
Safety Issue:
Description:Duration of response measured using time to AML transformation according to WHO Critieria

Details

Phase:Phase 2
Primary Purpose:Interventional
Overall Status:Recruiting
Lead Sponsor:H. Lee Moffitt Cancer Center and Research Institute

Trial Keywords

  • CMML

Last Updated

December 30, 2019