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

NCT03722407 | Active Not Recruiting Phase 2 Results FDA Drug

• 1 other identifier: MCC-19727
• Study Type: interventional
• Target: 29
• Locations: 1 country
• Sites: 5

Brief 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.

Conditions

Leukemia; Chronic Myelomonocytic Leukemia

Keywords

CMML

Outcome Measures

Primary Outcomes (1)

• Overall Response

  Number 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.

  At week 16

Secondary Outcomes (3)

• Time to Acute Myeloid Leukemia (AML) Transformation

  Every 6 months after conclusion of treatment until end of study (40.3 months)

• Overall Survival

  Up to 2 years

• Duration of Response

  Up to 2 years

Study Arms (1)

Ruxolitinib

EXPERIMENTAL

All 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.

Drug: Ruxolitinib

Interventions

Ruxolitinib DRUG

Ruxolitinib 5 mg tablets, 4 per dose

Also known as: Jakafi

Ruxolitinib

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Confirmed diagnosis of Chronic Myelomonocytic Leukemia (CMML)using the World Health Organization (WHO) classification.
• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• H. Lee Moffitt Cancer Center and Research Institute: lead
• Incyte Corporation: collaborator

Study Sites (5)

H. Lee Moffitt Cancer Center and Research Institute

Tampa, Florida, 33612, United States

Location

Johns Hopkins Sidney Kimmel Comprehensive Cancer Center

Baltimore, Maryland, 21231, United States

Location

Dana Farber Cancer Institute

Boston, Massachusetts, 02215, United States

Location

Weill Medical College of Cornell University

New York, New York, 10021, United States

Location

Cleveland Clinic

Cleveland, Ohio, 44195, United States

Location

Related Links

• Moffitt Cancer Center Clinical Trials Website

MeSH Terms

Conditions

Leukemia, Myelomonocytic, Chronic; Leukemia

Interventions

ruxolitinib

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Neoplasms by Histologic Type; Neoplasms; Myelodysplastic-Myeloproliferative Diseases; Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Results Point of Contact

• Title: Eric Padron, MD
• Organization: Moffitt Cancer Center

eric.padron@moffitt.org

813-745-2864

Study Officials

• Eric Padron, MD

  H. Lee Moffitt Cancer Center and Research Institute

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 25, 2018
• First Posted: October 29, 2018
• Study Start: August 28, 2019
• Primary Completion: September 19, 2022
• Study Completion (Estimated): June 1, 2026
• Last Updated: April 2, 2026
• Results First Posted: October 17, 2023

Record last verified: 2026-03

Locations

US (5)