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Pilot Study of Sodium Phenylbutyrate Plus Azacytidine

Phase 2
18 Years
Not Enrolling
Leukemia, Lung Cancer, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Prostate Cancer

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Trial Information

Pilot Study of Sodium Phenylbutyrate Plus Azacytidine


- 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

- 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.

Inclusion Criteria


- 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



- 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

Type of Study:


Study Design:

Masking: Open Label, Primary Purpose: Treatment

Principal Investigator

Peter Maslak, MD

Investigator Role:

Study Chair

Investigator Affiliation:

Memorial Sloan-Kettering Cancer Center


United States: Federal Government

Study ID:




Start Date:

May 2000

Completion Date:

August 2003

Related Keywords:

  • Leukemia
  • Lung Cancer
  • Lymphoma
  • Multiple Myeloma and Plasma Cell Neoplasm
  • Myelodysplastic Syndromes
  • Prostate Cancer
  • 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
  • Neoplasms
  • Carcinoma, Non-Small-Cell Lung
  • Leukemia
  • Leukemia, Myeloid, Acute
  • Leukemia, Myeloid
  • Lung Neoplasms
  • Lymphoma
  • Lymphoma, Non-Hodgkin
  • Multiple Myeloma
  • Neoplasms, Plasma Cell
  • Plasmacytoma
  • Myelodysplastic Syndromes
  • Preleukemia
  • Prostatic Neoplasms



Memorial Sloan-Kettering Cancer Center New York, New York  10021