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Phenylbutyrate Plus Azacitidine in Treating Patients With Acute Myeloid Leukemia, Myelodysplasia, Non-Hodgkin's Lymphoma, Multiple Myeloma, Non-small Cell Lung Cancer, or Prostate Cancer

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: NCT00006019
Recruitment Status : Completed
First Posted : August 29, 2003
Last Update Posted : June 24, 2013
National Cancer Institute (NCI)
Information provided by:
National Cancer Institute (NCI)

Brief Summary:

RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Combining more than one chemotherapy drug may kill more cancer cells.

PURPOSE: Phase II trial to study the effectiveness of phenylbutyrate plus azacitidine in treating patients who have acute myeloid leukemia, myelodysplasia, non-Hodgkin's lymphoma, multiple myeloma, non-small cell lung cancer, or prostate cancer.

Condition or disease Intervention/treatment Phase
Leukemia Lung Cancer Lymphoma Multiple Myeloma and Plasma Cell Neoplasm Myelodysplastic Syndromes Prostate Cancer Drug: azacitidine Drug: sodium phenylbutyrate Phase 2

Detailed Description:


  • Determine the ability of azacytidine in vivo to demethylate selected genes known to be transcriptionally repressed in patients with acute myeloid leukemia, myelodysplasia, non-Hodgkin's lymphoma, multiple myeloma, non-small cell lung cancer, or prostate cancer.
  • Determine the ability of phenylbutyrate plus azacytidine to induce transcription of target genes that are known to be repressed as a consequence of DNA methylation in these patients.
  • Determine the effect of this treatment regimen upon gene methylation and histone acetylation in target cells in these patients.
  • Determine the technical feasibility of serially monitoring transcriptional activity and methylation status of selected genes in vivo in these patients.
  • Determine the safety and potential antitumor efficacy of this treatment regimen in these patients.

OUTLINE: Patients receive azacytidine subcutaneously on days 1-7 and phenylbutyrate IV over 1-2 hours on days 8-12. Patients with acute myeloid leukemia who respond to therapy may receive a second course approximately 10 days after the end of the first. Subsequent courses in these patients, and all additional courses in all other patients, are repeated every 21 to 28 days in the absence of disease progression or unacceptable toxicity.

PROJECTED ACCRUAL: A total of 20 patients will be accrued for this study.

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Study Type : Interventional  (Clinical Trial)
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Pilot Study of Sodium Phenylbutyrate Plus Azacytidine
Study Start Date : May 2000
Actual Study Completion Date : August 2003

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No


  • Diagnosis of one of the following neoplastic diseases:

    • Acute myeloid leukemia
    • Myelodysplasia
    • Low or intermediate grade non-Hodgkin's lymphoma
    • Multiple myeloma
    • Non-small cell lung cancer
    • Prostate cancer
  • Failed prior conventional therapy and no other known curative therapy exists
  • Patients with non-Hodgkin's lymphoma, non-small cell lung cancer, and prostate cancer must have tumor cells in bone marrow or malignant effusions that are accessible for bone marrow aspiration or paracentesis/thoracentesis NOTE: A new classification scheme for adult non-Hodgkin's lymphoma has been adopted by PDQ. The terminology of "indolent" or "aggressive" lymphoma will replace the former terminology of "low", "intermediate", or "high" grade lymphoma. However, this protocol uses the former terminology.



  • 18 and over

Performance status:

  • Karnofsky 60-100%

Life expectancy:

  • Not specified


  • Patients without leukemia or myeloma:

    • WBC at least 2,500/mm^3
    • Platelet count at least 75,000/mm^3


  • Bilirubin no greater than 2.5 mg/dL


  • Creatinine no greater than 2.5 mg/dL


  • Not pregnant or nursing
  • Negative pregnancy test
  • Fertile patients must use effective contraception during and for 4 weeks after study


Biologic therapy:

  • Not specified


  • Patients without leukemia:

    • At least 3 weeks since prior cytotoxic chemotherapy

Endocrine therapy:

  • Not specified


  • Patients without leukemia:

    • At least 3 weeks since prior radiotherapy


  • Not specified

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 identifier (NCT number): NCT00006019

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United States, New York
Memorial Sloan-Kettering Cancer Center
New York, New York, United States, 10021
Sponsors and Collaborators
Memorial Sloan Kettering Cancer Center
National Cancer Institute (NCI)
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Study Chair: Peter Maslak, MD Memorial Sloan Kettering Cancer Center
Layout table for additonal information Identifier: NCT00006019    
Other Study ID Numbers: CDR0000068030
First Posted: August 29, 2003    Key Record Dates
Last Update Posted: June 24, 2013
Last Verified: October 2002
Keywords provided by National Cancer Institute (NCI):
recurrent non-small cell lung cancer
refractory multiple myeloma
recurrent adult acute myeloid leukemia
recurrent prostate cancer
adult acute myeloblastic leukemia without maturation (M1)
adult acute myeloblastic leukemia with maturation (M2)
adult acute promyelocytic leukemia (M3)
adult acute myelomonocytic leukemia (M4)
adult acute monoblastic leukemia (M5a)
recurrent grade 1 follicular lymphoma
recurrent grade 2 follicular lymphoma
recurrent grade 3 follicular lymphoma
recurrent adult diffuse small cleaved cell lymphoma
recurrent adult diffuse mixed cell lymphoma
recurrent adult diffuse large cell lymphoma
adult acute monocytic leukemia (M5b)
previously treated myelodysplastic syndromes
secondary myelodysplastic syndromes
recurrent marginal zone lymphoma
recurrent small lymphocytic lymphoma
extranodal marginal zone B-cell lymphoma of mucosa-associated lymphoid tissue
nodal marginal zone B-cell lymphoma
splenic marginal zone lymphoma
Additional relevant MeSH terms:
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Lung Neoplasms
Prostatic Neoplasms
Multiple Myeloma
Neoplasms, Plasma Cell
Myelodysplastic Syndromes
Neoplasms by Histologic Type
Lymphoproliferative Disorders
Lymphatic Diseases
Immunoproliferative Disorders
Immune System Diseases
Pathologic Processes
Respiratory Tract Neoplasms
Thoracic Neoplasms
Neoplasms by Site
Lung Diseases
Respiratory Tract Diseases
Genital Neoplasms, Male
Urogenital Neoplasms
Prostatic Diseases
Hemostatic Disorders
Vascular Diseases
Cardiovascular Diseases