Decitabine in Treating Patients With Myelofibrosis

NCT ID: NCT00095784

Last Updated: 2025-08-24

Basic Information

• Recruitment Status: ACTIVE_NOT_RECRUITING
• Clinical Phase: PHASE2
• Total Enrollment: 21 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2004-09-29
• Study Completion Date: 2026-02-22

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.

Detailed Description

PRIMARY OBJECTIVES:

I. To determine response rate (complete and partial responses and hematological improvement) to decitabine in patients with myelofibrosis.

II. To determine the safety of decitabine in patients with myelofibrosis.

SECONDARY OBJECTIVES:

I. To determine the effects of decitabine on specific epigenetic changes including methylation status of specific target genes and gene re-expression.

II. To determine the effect of decitabine on hemoglobin F levels and on the absolute numbers of circulating cluster of differentiation (CD) 34+ progenitor cells and to investigate the potential utility of these markers as a surrogate for biologic activity of decitabine in myeloid metaplasia with myelofibrosis (MMM).

OUTLINE:

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

Conditions

Primary Myelofibrosis; Secondary Myelofibrosis

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Treatment (decitabine)

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.

Group Type: EXPERIMENTAL

Decitabine

Intervention Type: DRUG

Given SC

Laboratory Biomarker Analysis

Intervention Type: OTHER

Correlative studies

Interventions

Decitabine

Given SC

Intervention Type: DRUG

Laboratory Biomarker Analysis

Correlative studies

Intervention Type: OTHER

Other Intervention Names

5-Aza-2'-deoxycytidine; Dacogen; Decitabine for Injection; Deoxyazacytidine; Dezocitidine

Eligibility Criteria

Inclusion Criteria

• 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
• The two necessary criteria plus any other four optional criteria when splenomegaly is absent
• Patients may have had prior chemotherapy or radiation therapy including splenic irradiation; prior therapy with erythropoietin, granulocyte-colony stimulating factor (GCSF), other growth factors or androgenic steroids is also permitted; there is no limit to the number of prior regimens received; at least 4 weeks must have elapsed since prior chemo or radiation therapy; at least 2 weeks must have elapsed since growth factor (erythropoietin, GCSF, granulocyte-macrophage colony-stimulating factor [GM-CSF]) or other therapy
• Eastern Cooperative Oncology Group (ECOG) performance status =< 2 (Karnofsky >= 60%)
• Total bilirubin =< 2mg/dL

• In patients with associated hemolytic anemia; total bilirubin > 2mg/dL is permissible as long as this is as a result of predominantly unconjugated hyperbilirubinemia; such patients may be enrolled only after discussion with the study chair
• Aspartate aminotransferase (AST) (serum glutamic oxaloacetic transaminase [SGOT])/alanine aminotransferase (ALT) (serum glutamate pyruvate transaminase [SGPT]) =< 3 x institutional upper limit of normal unless due to disease
• Serum creatinine =< 2mg/dL
• Patients must not be pregnant or nursing; women of child- bearing potential and men must agree to use an effective contraceptive method; should a woman become pregnant or suspect she is pregnant while participating in this study, she should inform her treating physician immediately
• Ability to understand and the willingness to sign a written informed consent document

Exclusion Criteria

• 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

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

National Cancer Institute (NCI)

NIH

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Olatoyosi M Odenike

Role: PRINCIPAL_INVESTIGATOR

University of Chicago Comprehensive Cancer Center

Locations

University of Chicago Comprehensive Cancer Center

Chicago, Illinois, United States

Decatur Memorial Hospital

Decatur, Illinois, United States

NorthShore University HealthSystem-Evanston Hospital

Evanston, Illinois, United States

Ingalls Memorial Hospital

Harvey, Illinois, United States

Loyola University Medical Center

Maywood, Illinois, United States

Illinois CancerCare-Peoria

Peoria, Illinois, United States

Central Illinois Hematology Oncology Center

Springfield, Illinois, United States

Southern Illinois University School of Medicine

Springfield, Illinois, United States

Fort Wayne Medical Oncology and Hematology Inc-Parkview

Fort Wayne, Indiana, United States

Northern Indiana Cancer Research Consortium

South Bend, Indiana, United States

University of Maryland/Greenebaum Cancer Center

Baltimore, Maryland, United States

Oncology Care Associates PLLC

Saint Joseph, Michigan, United States

Ohio State University Comprehensive Cancer Center

Columbus, Ohio, United States

Medical College of Wisconsin

Milwaukee, Wisconsin, United States

Countries

United States

References

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.

Reference Type: DERIVED

PMID: 39250708 (View on PubMed)

Other Identifiers

NCI-2011-01444

Identifier Type: REGISTRY

Identifier Source: secondary_id

CDR0000393839

Identifier Type: -

Identifier Source: secondary_id

NCI-6814

Identifier Type: -

Identifier Source: secondary_id

UCCRC-13327A

Identifier Type: -

Identifier Source: secondary_id

13327A

Identifier Type: OTHER

Identifier Source: secondary_id

6814

Identifier Type: OTHER

Identifier Source: secondary_id

N01CM62201

Identifier Type: NIH

Identifier Source: secondary_id

View Link

N01CM62207

Identifier Type: NIH

Identifier Source: secondary_id

View Link

P30CA014599

Identifier Type: NIH

Identifier Source: secondary_id

View Link

NCI-2011-01444

Identifier Type: -

Identifier Source: org_study_id