We updated the design of this site on September 25th. Learn more.
Show more
ClinicalTrials.gov
ClinicalTrials.gov Menu

Low-Dose Decitabine in Treating Patients With Symptomatic Myelofibrosis

This study has been terminated.
(Stopped due to slow accrual)
Sponsor:
ClinicalTrials.gov Identifier:
NCT00630994
First Posted: March 7, 2008
Last Update Posted: December 30, 2015
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.
Collaborator:
National Cancer Institute (NCI)
Information provided by (Responsible Party):
Mayo Clinic
  Purpose

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.


Condition Intervention Phase
Chronic Myeloproliferative Disorders Secondary Myelofibrosis Drug: Dacogen Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Phase II Trial of Low Dose Decitabine (Dacogen) in Patients With Primary Myelofibrosis and Post ET/PV Myelofibrosis

Resource links provided by NLM:


Further study details as provided by Mayo Clinic:

Primary Outcome Measures:
  • 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)



Secondary Outcome Measures:
  • 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.


Enrollment: 4
Study Start Date: March 2008
Study Completion Date: April 2012
Primary Completion Date: August 2009 (Final data collection date for primary outcome measure)
Detailed Description:

OBJECTIVES:

  • 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.

  Eligibility

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Ages Eligible for Study:   18 Years to 120 Years   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

DISEASE CHARACTERISTICS:

  • 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

PATIENT CHARACTERISTICS:

  • 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

PRIOR CONCURRENT THERAPY:

  • No other concurrent chemotherapy (e.g., hydroxyurea, thalidomide, interferon alpha, anagrelide, or other myelosuppressive agent) or experimental therapy
  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00630994


Locations
United States, Minnesota
Mayo Clinic
Rochester, Minnesota, United States, 55905
Sponsors and Collaborators
Mayo Clinic
National Cancer Institute (NCI)
Investigators
Study Chair: Ruben A. Mesa, MD Mayo Clinic
  More Information

Responsible Party: Mayo Clinic
ClinicalTrials.gov Identifier: NCT00630994     History of Changes
Other Study ID Numbers: CDR0000588839
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 )
First Submitted: March 6, 2008
First Posted: March 7, 2008
Results First Submitted: January 9, 2012
Results First Posted: April 2, 2012
Last Update Posted: December 30, 2015
Last Verified: November 2012

Keywords provided by Mayo Clinic:
primary myelofibrosis
secondary myelofibrosis
essential thrombocythemia
polycythemia vera

Additional relevant MeSH terms:
Primary Myelofibrosis
Myeloproliferative Disorders
Bone Marrow Diseases
Hematologic Diseases
Decitabine
Antimetabolites, Antineoplastic
Antimetabolites
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Enzyme Inhibitors