Low-Dose Decitabine in Treating Patients With Symptomatic Myelofibrosis

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00630994
Recruitment Status : Terminated (Stopped due to slow accrual)
First Posted : March 7, 2008
Results First Posted : April 2, 2012
Last Update Posted : December 30, 2015
National Cancer Institute (NCI)
Information provided by (Responsible Party):
Mayo Clinic

March 6, 2008
March 7, 2008
January 9, 2012
April 2, 2012
December 30, 2015
March 2008
August 2009   (Final data collection date for primary outcome measure)
Number of Participants Who Achieve a Confirmed Response (Complete Remission (CR), Partial Remission (PR), or Clinical Improvement (CI)), According to International Working Group (IWG) Consensus Criteria. [ Time Frame: Every 4 weeks during treatment (up to 16 weeks) ]

Confirmed response: objective status of CR, PR, or CI on 2 consecutive evaluations >=4 weeks apart.

CR:Complete resolution of disease-related symptoms and signs; peripheral blood count remission; normal leukocyte differential; bone marrow histologic remission.

PR: All criteria for CR except the bone marrow histologic remission. CI: one of the following in the absence of both disease progression and CR/PR: minimum (MI) 20-g/L increase (INC) in hemoglobin level; MI 50% reduction in palpable splenomegaly (>=10cm); MI 100% INC in platelet count(>=50000x10^9/L) or ANC (>=0.5x10^9/L)

Confirmed response rate (complete remission, partial remission, or clinical improvement)
Complete list of historical versions of study NCT00630994 on Archive Site
  • Overall Survival(OS) [ Time Frame: up to 3 years ]
    OS was defined as the time from registration to death of any cause.
  • Time to Disease Progression [ Time Frame: up to 3 years ]

    Time to disease progression is defined as the time from registration to progression of disease or death due to any cause.

    Progression was defined as any one or more of the following:

    1)progressive splenomegaly; 2) leukemic transformation confirmed by a bone marrow blast count of >= 20%; 3) an increase in peripheral blood blast percentage of >=20% that lasts for >= 8 weeks.

  • Number of Participants With Constitutional Symptoms [ Time Frame: Up to 48 weeks ]
    Constitutional symptoms including the presence of one or more of the following felt to be attributed to the disease: severe night sweats, fevers, weight loss and bone pain. Symptoms were assessed every cycle during treatment.
  • Number of Participants With Severe Adverse Events [ Time Frame: Up to 48 weeks ]
    Severe adverse events were defined as grade 3 or higher, regardless of attribution to study drugs. Adverse events were graded according to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) version 3. Adverse events were assessed every cycle during treatment.
  • Overall survival
  • Time to Disease Progression
  • Constitutional symptoms
  • Toxicity as assessed by NCI CTCAE v3.0
  • Pertinent biologic characteristics of primary myelofibrosis as assessed before and during treatment with decitabine
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Low-Dose Decitabine in Treating Patients With Symptomatic Myelofibrosis
Phase II Trial of Low Dose Decitabine (Dacogen) in Patients With Primary Myelofibrosis and Post ET/PV Myelofibrosis

RATIONALE: Drugs used in chemotherapy, such as decitabine, work in different ways to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing.

PURPOSE: This phase II trial is studying the side effects and how well low-dose decitabine works in treating patients with symptomatic myelofibrosis.


  • Determine the efficacy and safety of low-dose decitabine in patients with symptomatic primary myelofibrosis (PMF) or post essential thrombocythemic (ET) or polycythemic vera (PV) myelofibrosis.
  • Analyze the ability of this drug to decrease pathologic angiogenesis and other stromal reactive features intrinsic to PMF or post ET/PV myelofibrosis.

OUTLINE: Patients receive low-dose decitabine IV over 1 hour on days 1-5. Treatment repeats every 28 days for up to 6 courses in the absence of disease progression or unacceptable toxicity. Patients achieving partial remission, complete remission, or clinical improvement may receive up to 12 courses of decitabine in the absence of disease progression or unacceptable toxicity.

After completion of study therapy, patients are followed periodically for up to 3 years.

Phase 2
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
  • Chronic Myeloproliferative Disorders
  • Secondary Myelofibrosis
Drug: Dacogen
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*   Includes publications given by the data provider as well as publications identified by Identifier (NCT Number) in Medline.
April 2012
August 2009   (Final data collection date for primary outcome measure)


  • Histological confirmation of primary myelofibrosis or post essential thrombocythemic or polycythemic vera myelofibrosis

    • Reticulin fibrosis ≥ grade 1
  • Evaluable and symptomatic disease worthy of treatment, characterized by ≥ 1 of the following:

    • Anemia, defined as hemoglobin < 11 g/dL or erythrocyte transfusion dependence
    • Palpable and symptomatic splenomegaly (palpable and symptomatic hepatomegaly is acceptable if previously splenectomized)
    • Severe, disease-related constitutional symptoms, including ≥ 1 of the following:

      • Severe night sweats
      • Fevers
      • Weight loss
      • Bone pain
  • Absence of t(9;22) by fluorescent in situ hybridization (FISH) or standard cytogenetics OR prior demonstration of a lack of this translocation


  • Eastern Co-operative Oncology Group (ECOG) performance status 0-3
  • Absolute neutrophil count (ANC) ≥ 1,000/mm³
  • Platelet count ≥ 50,000/mm³
  • Creatinine ≤ 2.0 mg/dL
  • Direct or total bilirubin ≤ 2.0 mg/dL
  • Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤ 3 times upper limit of normal (ULN) (≤ 5 times ULN if elevation is attributed to hepatic extramedullary hematopoiesis)
  • Not pregnant or nursing
  • Negative pregnancy test
  • Fertile patients must use effective contraception
  • Not incarcerated in a municipality, county, state, or federal prison
  • No serious medical condition or psychiatric illness that would preclude signing the informed consent
  • No condition that, in the opinion of the treating physician, places the patient at unacceptable risk for study participation or confounds the ability to interpret study data
  • Able to adhere to the study visit schedule and other study requirements


  • No other concurrent chemotherapy (e.g., hydroxyurea, thalidomide, interferon alpha, anagrelide, or other myelosuppressive agent) or experimental therapy
Sexes Eligible for Study: All
18 Years to 120 Years   (Adult, Older Adult)
Contact information is only displayed when the study is recruiting subjects
United States
P30CA015083 ( U.S. NIH Grant/Contract )
MC0788 ( Other Identifier: Mayo Clinic Cancer Center )
NCI-2009-01330 ( Registry Identifier: NCI-CTRO )
07-005296 ( Other Identifier: Mayo Clinic IRB )
Not Provided
Not Provided
Mayo Clinic
Mayo Clinic
National Cancer Institute (NCI)
Study Chair: Ruben A. Mesa, MD Mayo Clinic
Mayo Clinic
November 2012

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP