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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

This study has been completed.
National Cancer Institute (NCI)
Information provided by:
National Cancer Institute (NCI) Identifier:
First received: July 5, 2000
Last updated: June 20, 2013
Last verified: October 2002

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

Study Type: Interventional
Study Design: Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Pilot Study of Sodium Phenylbutyrate Plus Azacytidine

Resource links provided by NLM:

Further study details as provided by National Cancer Institute (NCI):

Study Start Date: May 2000
Study Completion Date: August 2003
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.


Ages Eligible for Study:   18 Years and older   (Adult, Senior)
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
  Contacts and Locations
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, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT00006019

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)
Study Chair: Peter Maslak, MD Memorial Sloan Kettering Cancer Center
  More Information Identifier: NCT00006019     History of Changes
Other Study ID Numbers: CDR0000068030
Study First Received: July 5, 2000
Last Updated: June 20, 2013

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:
Lung Neoplasms
Prostatic Neoplasms
Carcinoma, Non-Small-Cell Lung
Multiple Myeloma
Neoplasms, Plasma Cell
Leukemia, Myeloid, Acute
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
Genital Diseases, Male
Prostatic Diseases
Carcinoma, Bronchogenic processed this record on September 25, 2017