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Phase II Study of Decitabine in Acute Myeloid Leukemia

Phase 2
18 Years
Open (Enrolling)
Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Del(5q), Adult Acute Myeloid Leukemia With Inv(16)(p13;q22), Adult Acute Myeloid Leukemia With t(15;17)(q22;q12), Adult Acute Myeloid Leukemia With t(16;16)(p13;q22), Adult Acute Myeloid Leukemia With t(8;21)(q22;q22), Secondary Acute Myeloid Leukemia, Untreated Adult Acute Myeloid Leukemia

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Trial Information

Phase II Study of Decitabine in Acute Myeloid Leukemia


I. Determine the rate of complete remission (CR) in patients with previously untreated acute
myeloid leukemia treated with decitabine.


I. Determine the rate of overall survival at 1 year in patients treated with this drug.

II. Determine the overall response rate (CR, incomplete CR, and partial remission) in
patients treated with this drug.

III. Correlate the biological activity of decitabine with clinical endpoints and maximum
concentration of plasma decitabine.

IV. Correlate intracellular concentration of decitabine with global DNA methylation, other
biological endpoints, and clinical response.


Patients receive decitabine IV over 1 hour on days 1-10. Treatment repeats every 28 days in
the absence of disease progression or unacceptable toxicity.

Patients undergo bone marrow aspiration and blood sample collection periodically for
pharmacological and correlative studies. Samples are analyzed for gene expression,
methylation of gene promoters, fetal hemoglobin (HgF), DNMT1 protein expression, maximum
concentration of plasma decitabine, and global DNA methylation. Samples are analyzed by
RT-PCR, Bio-COBRA, matrix-assisted laser desorption ionization time-of-flight mass
spectrometry, SDS-PAGE (polyacrylamide gel electrophoresis), immunoblotting, and LC-MS/MS.

After completion of study treatment, patients are followed for at least 30 days.

Inclusion Criteria:

- Histologically or cytologically confirmed acute myeloid leukemia (AML) meeting 1 of
the following criteria:

- At least 60 years of age and not a candidate for or refused standard induction

- Poor risk cytogenetics

- AML following antecedent hematologic disorder

- Therapy-related AML

- Secondary AML

- No granulocytic sarcoma as sole site of disease

- No active CNS disease or CNS relapse

- ECOG performance status 0-2

- Life expectancy > 6 months

- Total bilirubin < 2.0 mg/dL

- Creatinine < 2.0 mg/dL

- AST and ALT < 2.5 times upper limit of normal

- Not pregnant or nursing

- Negative pregnancy test

- Fertile patients must use effective contraception

- No NYHA class III or IV congestive heart failure

- No uncontrolled infection

- No history of allergic reactions attributed to compounds of similar chemical or
biologic composition to decitabine that are not easily managed

- No other uncontrolled illness including, but not limited to, any of the following:

- Symptomatic congestive heart failure

- Unstable angina pectoris

- Serious cardiac arrhythmia

- Psychiatric illness or social situations that would preclude compliance with
study requirements

- No active second malignancy involving the blood or marrow or likely to progress and
require therapy in the next 6 months

- No prior therapy for AML except emergency leukapheresis or hydroxyurea for

- No prior azacitidine or decitabine

- No prior cytarabine or other conventional chemotherapy agents for antecedent
hematologic disorders

- Prior myeloid growth factors, recombinant erythropoietin, thalidomide, or
lenalidomide allowed

- No concurrent palliative radiotherapy

- No other concurrent investigational agents

- No other concurrent direct anti-leukemia therapy

- No concurrent combination antiretroviral therapy for HIV-positive patients

Type of Study:


Study Design:

Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Rate of complete remission

Outcome Description:

Assessment of clinical response will be made according to International Working Group criteria. The major criteria for judging response will include physical examination and examination of blood and bone marrow. For the primary endpoint, all enrolled patients will be analyzed together (regardless of age).

Outcome Time Frame:

Every 4 weeks, assessed up to 30 days after completion of treatment

Safety Issue:


Principal Investigator

William Blum

Investigator Role:

Principal Investigator

Investigator Affiliation:

Ohio State University


United States: Food and Drug Administration

Study ID:




Start Date:

May 2007

Completion Date:

Related Keywords:

  • Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities
  • Adult Acute Myeloid Leukemia With Del(5q)
  • Adult Acute Myeloid Leukemia With Inv(16)(p13;q22)
  • Adult Acute Myeloid Leukemia With t(15;17)(q22;q12)
  • Adult Acute Myeloid Leukemia With t(16;16)(p13;q22)
  • Adult Acute Myeloid Leukemia With t(8;21)(q22;q22)
  • Secondary Acute Myeloid Leukemia
  • Untreated Adult Acute Myeloid Leukemia
  • Congenital Abnormalities
  • Leukemia
  • Leukemia, Myeloid, Acute
  • Leukemia, Myeloid



Ohio State University Medical Center Columbus, Ohio  43210