Decitabine in Treating Patients With Myelofibrosis

NCT00095784 | Active Not Recruiting Phase 2 Results

• 9 other identifiers: NCI-2011-01444CDR0000393839NCI-6814UCCRC-13327A13327A6814N01CM62201N01CM62207P30CA014599
• Study Type: interventional
• Target: 21
• Locations: 1 country
• Sites: 14

Brief Summary

This phase II trial studies the side effects and how well decitabine works in treating patients with myelofibrosis, a cancer of the blood system associated with fibrosis (scar tissue) in the bone marrow that is advanced and for which there is no standard therapy. Decitabine may block the actions of some proteins that are responsible for turning certain genes off in various cancers including myelofibrosis.

Conditions

Primary Myelofibrosis; Secondary Myelofibrosis

Outcome Measures

Primary Outcomes (2)

• Response Rate (Complete Response, Partial Response, or Hematologic Improvement.

  Complete response is normalization of counts and transfusion-independence. Partial response is hemoglobin increase to normal levels, multilineage improvement including absolute neutrophil count (ANC) and/or platelets. Hematologic improvement is red cell transfusion-independence or \>50% increase in platelet levels.

  Up to 36 weeks (6 cycles)

• Incidence of Toxicities, Graded According to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) v3.0

  Percentage of patients experiencing any toxicity, any grade level. Additional details on adverse events are reported in Adverse Events section.

  Up to 30 days of last dose of decitabine

Secondary Outcomes (18)

• CD34+ Cells

  Cycle 1, Day 1

• CD34+ Cells

  Cycle 1, Day 5

• CD34+ Cells

  Cycle 1, Day 12

• CD34+ Cells

  Cycle 2, Day 1

• CD34+ Cells

  Cycle 2, Day 5

Study Arms (1)

Treatment (decitabine)

EXPERIMENTAL

Patients receive decitabine SC on days 1-5 and 8-12. Treatment repeats every 42 days in the absence of disease progression or unacceptable toxicity.

Drug: Decitabine; Other: Laboratory Biomarker Analysis

Interventions

Decitabine DRUG

Given SC

Also known as: 5-Aza-2'-deoxycytidine, Dacogen, Decitabine for Injection, Deoxyazacytidine, Dezocitidine

Treatment (decitabine)

Laboratory Biomarker Analysis OTHER

Correlative studies

Treatment (decitabine)

Eligibility Criteria

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

You may qualify if:

• Patients must have histologically or cytologically confirmed myeloid metaplasia with myelofibrosis (this includes all subtypes - chronic idiopathic myelofibrosis or angiogenic myeloid metaplasia, post thrombocythemic and post polycythemic myelofibrosis); patients must have anemia (hemoglobin \< 11 g/dL) or palpable splenomegaly (measured in cm from costal margin - to be eligible); patients with palpable splenomegaly must have spleen size documented ultrasonographically as well; they must also meet standard diagnostic criteria for MMM
• Patients with morphologic evidence of advanced phases of the disease including accelerated (10-19% blasts) phase or with evidence of evolution to acute leukemia (\>= 20% blasts) are also eligible for this study
• The Italian Diagnostic Criteria for MMM
• Necessary criteria
• Diffuse bone marrow fibrosis
• Absence of the Philadelphia chromosome or BCR-ABL rearrangement in peripheral blood cells
• Optional criteria
• Splenomegaly of any grade
• Anisopoikilocytosis with tear drop erythrocytes
• Presence of circulating immature myeloid cells
• Presence of circulating erythroblasts
• Presence of clusters of megakaryoblasts and anomalous megakaryocytes in bone marrow sections
• Myeloid metaplasia
• Diagnosis of MMM is acceptable if the following combinations are present
• The two necessary criteria plus any other two optional criteria when splenomegaly is present OR

You may not qualify if:

• Prior therapy with decitabine
• Patients who have had chemotherapy or radiotherapy within 4 weeks (6 weeks for nitrosoureas or mitomycin C) prior to entering the study or those who have not recovered from adverse events due to agents administered more than 4 weeks earlier
• Patients may not be receiving any other investigational agents
• Patients with known central nervous system (CNS) disease should be excluded from this clinical trial
• History of allergic reactions attributed to compounds of similar chemical or biologic composition to decitabine
• Uncontrolled intercurrent 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; breastfeeding should be discontinued if the mother is treated with decitabine
• Human immunodeficiency virus (HIV)-positive patients receiving combination anti-retroviral therapy are excluded from the study

Sponsors & Collaborators

• National Cancer Institute (NCI): lead

Study Sites (14)

University of Chicago Comprehensive Cancer Center

Chicago, Illinois, 60637, United States

Location

Decatur Memorial Hospital

Decatur, Illinois, 62526, United States

Location

NorthShore University HealthSystem-Evanston Hospital

Evanston, Illinois, 60201, United States

Location

Ingalls Memorial Hospital

Harvey, Illinois, 60426, United States

Location

Loyola University Medical Center

Maywood, Illinois, 60153, United States

Location

Illinois CancerCare-Peoria

Peoria, Illinois, 61615, United States

Location

Central Illinois Hematology Oncology Center

Springfield, Illinois, 62702, United States

Location

Southern Illinois University School of Medicine

Springfield, Illinois, 62702, United States

Location

Fort Wayne Medical Oncology and Hematology Inc-Parkview

Fort Wayne, Indiana, 46845, United States

Location

Northern Indiana Cancer Research Consortium

South Bend, Indiana, 46628, United States

Location

University of Maryland/Greenebaum Cancer Center

Baltimore, Maryland, 21201, United States

Location

Oncology Care Associates PLLC

Saint Joseph, Michigan, 49085, United States

Location

Ohio State University Comprehensive Cancer Center

Columbus, Ohio, 43210, United States

Location

Medical College of Wisconsin

Milwaukee, Wisconsin, 53226, United States

Location

Related Publications (1)

• Lin C, Patel AA, Huo D, Karrison T, van Besien K, Godwin J, Sher D, Weiner H, Green M, Wade JL 3rd, Klisovic R, Baer MR, Larson RA, Stock W, Odenike O. A multicenter phase 2 clinical trial of low-dose subcutaneous decitabine in myelofibrosis. Blood Adv. 2024 Nov 26;8(22):5735-5743. doi: 10.1182/bloodadvances.2024013215.

  PMID: 39250708 DERIVED

MeSH Terms

Conditions

Primary Myelofibrosis

Interventions

Decitabine; Injections

Condition Hierarchy (Ancestors)

Myeloproliferative Disorders; Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases

Intervention Hierarchy (Ancestors)

Azacitidine; Aza Compounds; Organic Chemicals; Cytidine; Pyrimidine Nucleosides; Pyrimidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Nucleosides; Nucleic Acids, Nucleotides, and Nucleosides; Ribonucleosides; Drug Administration Routes; Drug Therapy; Therapeutics

Results Point of Contact

• Title: Olatoyosi Odenike, MD
• Organization: University of Chicago

todenike@medicine.bsd.uchicago.edu

773-702-3354

Study Officials

• Olatoyosi M Odenike

  University of Chicago Comprehensive Cancer Center

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: LTE60
• Restrictive Agreement: Yes

Study Design

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

Study Record Dates

• First Submitted: November 9, 2004
• First Posted: November 9, 2004
• Study Start: September 29, 2004
• Primary Completion: July 1, 2008
• Study Completion (Estimated): February 22, 2027
• Last Updated: April 29, 2026
• Results First Posted: January 5, 2015

Record last verified: 2026-02

Locations

US (14)