AzTrials
AzTrials

Primary MyelofibrosisPhase 2NCT00095784

Decitabine in Treating Patients With Myelofibrosis

A Phase 2 study testing Decitabine and Laboratory Biomarker Analysis in people with Primary Myelofibrosis and Secondary Myelofibrosis, run by National Cancer Institute (NCI) at 14 sites in United States.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
Search all trials

Trial 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. Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
Decitabine and Laboratory Biomarker Analysis
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
21 participants
Where
14 sitesUnited States
Study dates
29 Sep 2004to 22 Feb 2027
Phase
Phase 2
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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
Show all 24Show fewer
  • 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

Who cannot join

  • 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

Trial locations and contacts

14 sites in 1 country.

14 sites in 1 country

United States14 sites

  • University of Maryland/Greenebaum Cancer Center

    Baltimore, Maryland, 21201

  • University of Chicago Comprehensive Cancer Center

    Chicago, Illinois, 60637

  • Ohio State University Comprehensive Cancer Center

    Columbus, Ohio, 43210

  • Decatur Memorial Hospital

    Decatur, Illinois, 62526

  • NorthShore University HealthSystem-Evanston Hospital

    Evanston, Illinois, 60201

  • Fort Wayne Medical Oncology and Hematology Inc-Parkview

    Fort Wayne, Indiana, 46845

  • Ingalls Memorial Hospital

    Harvey, Illinois, 60426

  • Loyola University Medical Center

    Maywood, Illinois, 60153

  • Medical College of Wisconsin

    Milwaukee, Wisconsin, 53226

  • Illinois CancerCare-Peoria

    Peoria, Illinois, 61615

  • Oncology Care Associates PLLC

    Saint Joseph, Michigan, 49085

  • Northern Indiana Cancer Research Consortium

    South Bend, Indiana, 46628

  • Central Illinois Hematology Oncology Center

    Springfield, Illinois, 62702

  • Southern Illinois University School of Medicine

    Springfield, Illinois, 62702

Other trials in United States

About this study

Official title: A Phase II Study of Decitabine in Myelofibrosis

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.

Read the detailed description

PRIMARY OBJECTIVES:

  1. 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:

  1. 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.

Being tested
Decitabine, Laboratory Biomarker Analysis
Medical subject headings
Primary Myelofibrosis

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • DecitabineDrug

    Also called 5-Aza-2'-deoxycytidine, Dacogen, Decitabine for Injection, Deoxyazacytidine

    Given SC

  • Laboratory Biomarker AnalysisOther

    Correlative studies

Study groups in NCT00095784
Study groupRoleWhat they receive
Treatment (decitabine)ExperimentalPatients receive decitabine SC on days 1-5 and 8-12. Treatment repeats every 42 days in the absence of disease progression or unacceptable toxicity.
Receives: Drug: Decitabine; Other: Laboratory Biomarker Analysis

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. 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.

    Measured: Up to 36 weeks (6 cycles)

  2. 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.

    Measured: Up to 30 days of last dose of decitabine

Secondary outcomes (18)

  1. CD34+ Cells

    CD34 positive(+) cells are determined by flow immunostaining and light scatter in peripheral blood. Samples are then analyzed by flow cytometry, and CD34+ cells quantitated after 75,000 CD45 events are studied. (At least 75,000 CD45 events must be studied to ensure accuracy of the assay). Absolute numbers are determined by multiplying the % CD34+ cells by the total white blood cell count obtained on a CBC that is processed simultaneously.

    Measured: Cycle 1, Day 1

  2. CD34+ Cells

    CD34 positive(+) cells are determined by flow immunostaining and light scatter in peripheral blood. Samples are then analyzed by flow cytometry, and CD34+ cells quantitated after 75,000 CD45 events are studied. (At least 75,000 CD45 events must be studied to ensure accuracy of the assay). Absolute numbers are determined by multiplying the % CD34+ cells by the total white blood cell count obtained on a CBC that is processed simultaneously.

    Measured: Cycle 1, Day 5

  3. CD34+ Cells

    CD34 positive(+) cells are determined by flow immunostaining and light scatter in peripheral blood. Samples are then analyzed by flow cytometry, and CD34+ cells quantitated after 75,000 CD45 events are studied. (At least 75,000 CD45 events must be studied to ensure accuracy of the assay). Absolute numbers are determined by multiplying the % CD34+ cells by the total white blood cell count obtained on a CBC that is processed simultaneously.

    Measured: Cycle 1, Day 12

  4. CD34+ Cells

    CD34 positive(+) cells are determined by flow immunostaining and light scatter in peripheral blood. Samples are then analyzed by flow cytometry, and CD34+ cells quantitated after 75,000 CD45 events are studied. (At least 75,000 CD45 events must be studied to ensure accuracy of the assay). Absolute numbers are determined by multiplying the % CD34+ cells by the total white blood cell count obtained on a CBC that is processed simultaneously.

    Measured: Cycle 2, Day 1

  5. CD34+ Cells

    CD34 positive(+) cells are determined by flow immunostaining and light scatter in peripheral blood. Samples are then analyzed by flow cytometry, and CD34+ cells quantitated after 75,000 CD45 events are studied. (At least 75,000 CD45 events must be studied to ensure accuracy of the assay). Absolute numbers are determined by multiplying the % CD34+ cells by the total white blood cell count obtained on a CBC that is processed simultaneously.

    Measured: Cycle 2, Day 5

  6. CD34+ Cells

    CD34 positive(+) cells are determined by flow immunostaining and light scatter in peripheral blood. Samples are then analyzed by flow cytometry, and CD34+ cells quantitated after 75,000 CD45 events are studied. (At least 75,000 CD45 events must be studied to ensure accuracy of the assay). Absolute numbers are determined by multiplying the % CD34+ cells by the total white blood cell count obtained on a CBC that is processed simultaneously.

    Measured: Cycle 2, Day 12

  7. CXCR4

    CXCR4 gene expression level measured by real-time RT_PCR. Ratio of CXCR4 vs a housekeeping gene (i.e., ABL)

    Measured: Cycle 1, Day 1

  8. CXCR4

    CXCR4 gene expression level measured by real-time RT_PCR. Ratio of CXCR4 vs a housekeeping gene (i.e., ABL)

    Measured: Cycle 1, Day 5

  9. CXCR4

    CXCR4 gene expression level measured by real-time RT_PCR. Ratio of CXCR4 vs a housekeeping gene (i.e., ABL)

    Measured: Cycle 1, Day 12

  10. CXCR4

    CXCR4 gene expression level measured by real-time RT_PCR. Ratio of CXCR4 vs a housekeeping gene (i.e., ABL)

    Measured: Cycle 2, Day 1

  11. CXCR4

    CXCR4 gene expression level measured by real-time RT_PCR. Ratio of CXCR4 vs a housekeeping gene (i.e., ABL)

    Measured: Cycle 2, Day 5

  12. CXCR4

    CXCR4 gene expression level measured by real-time RT_PCR. Ratio of CXCR4 vs a housekeeping gene (i.e., ABL)

    Measured: Cycle 2, Day 12

  13. Hemoglobin F

    Percentage of hemoglobin F as a proportion of total Hb (%HbF) measured by HPLC on peripheral blood samples.

    Midpoint of 0.5% imputed for values reported as below limit of detection (1.0%). Hemoglobin F has been previously shown to be upregulated by decitabine in other hematologic disorders- sickle cell disease specifically. The rationale for exploring it in this study was to evaluate its potential utility as a biomarker of drug effect/PD marker.

    Measured: Cycle 1, Day 1

  14. Hemoglobin F

    Percentage of hemoglobin F as a proportion of total Hb (%HbF) measured by HPLC on peripheral blood samples.

    Midpoint of 0.5% imputed for values reported as below limit of detection (1.0%). Hemoglobin F has been previously shown to be upregulated by decitabine in other hematologic disorders- sickle cell disease specifically. The rationale for exploring it in this study was to evaluate its potential utility as a biomarker of drug effect/PD marker.

    Measured: Cycle 1, Day 5

  15. Hemoglobin F

    Percentage of hemoglobin F as a proportion of total Hb (%HbF) measured by HPLC on peripheral blood samples.

    Midpoint of 0.5% imputed for values reported as below limit of detection (1.0%). Hemoglobin F has been previously shown to be upregulated by decitabine in other hematologic disorders- sickle cell disease specifically. The rationale for exploring it in this study was to evaluate its potential utility as a biomarker of drug effect/PD marker.

    Measured: Cycle 1, Day 12

  16. Hemoglobin F

    Percentage of hemoglobin F as a proportion of total Hb (%HbF) measured by HPLC on peripheral blood samples.

    Midpoint of 0.5% imputed for values reported as below limit of detection (1.0%). Hemoglobin F has been previously shown to be upregulated by decitabine in other hematologic disorders- sickle cell disease specifically. The rationale for exploring it in this study was to evaluate its potential utility as a biomarker of drug effect/PD marker.

    Measured: Cycle 2, Day 1

  17. Hemoglobin F

    Percentage of hemoglobin F as a proportion of total Hb (%HbF) measured by HPLC on peripheral blood samples.

    Midpoint of 0.5% imputed for values reported as below limit of detection (1.0%). Hemoglobin F has been previously shown to be upregulated by decitabine in other hematologic disorders- sickle cell disease specifically. The rationale for exploring it in this study was to evaluate its potential utility as a biomarker of drug effect/PD marker.

    Measured: Cycle 2, Day 5

  18. Hemoglobin F

    Percentage of hemoglobin F as a proportion of total Hb (%HbF) measured by HPLC on peripheral blood samples.

    Midpoint of 0.5% imputed for values reported as below limit of detection (1.0%). Hemoglobin F has been previously shown to be upregulated by decitabine in other hematologic disorders- sickle cell disease specifically. The rationale for exploring it in this study was to evaluate its potential utility as a biomarker of drug effect/PD marker.

    Measured: Cycle 2, Day 12

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted9 Nov 2004 (estimated)
  2. Study start29 Sep 2004
  3. Primary completion1 Jul 2008
  4. Study completion22 Feb 2027 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
21 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT00095784
Sponsor's protocol ID
NCI-2011-01444
Other IDs
NCI-2011-01444, CDR0000393839, NCI-6814, UCCRC-13327A, 13327A, 6814, N01CM62201, N01CM62207
Status last verified
Aug 2026
First submitted
9 Nov 2004
Last updated
26 Aug 2026

Publications

  • 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.

    PubMed 39250708

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Decitabine and Laboratory Biomarker Analysis in people with Primary Myelofibrosis and Secondary Myelofibrosis, run by National Cancer Institute (NCI) at 14 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 24 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: under way, not looking for participants, as of the sponsor's last update on 26 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 14 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 5 Jan 2015). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Response Rate (Complete Response, Partial Response, or Hematologic Improvement. (time frame: Up to 36 weeks (6 cycles)). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT00095784, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Aug 2026.

AzTrials uses cookies for analytics and advertising. You can accept or reject them; the site works either way. Privacy Policy.

Free account

Keep searching with a free account

You’ve used your 3 free searches for this session. Log in or create a free AzTrials account to keep searching clinical trials and save the ones you want to follow.

Create a free accountLog in