Now Available: Final Rule for FDAAA 801 and NIH Policy on Clinical Trial Reporting

Phenylbutyrate, Dexamethasone, and Sargramostim in Treating Patients With Refractory or Relapsed Acute Myeloid Leukemia

This study has been completed.
Information provided by:
National Cancer Institute (NCI) Identifier:
First received: September 11, 2000
Last updated: April 27, 2015
Last verified: November 2002

RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Colony-stimulating factors such as sargramostim may increase the number of immune cells found in bone marrow or peripheral blood and may help a person's immune system recover from the side effects of chemotherapy.

PURPOSE: Phase II trial to study the effectiveness of combining phenylbutyrate, dexamethasone, and sargramostim in treating patients who have refractory or relapsed acute myeloid leukemia.

Condition Intervention Phase
Biological: sargramostim
Drug: dexamethasone
Drug: oral sodium phenylbutyrate
Phase 2

Study Type: Interventional
Study Design: Primary Purpose: Treatment
Official Title: A Pilot Study of Phenylbutyrate, Dexamethasone and GM-CSF in Refractory or Relapsed t(8;21) Acute Myeloid Leukemia

Resource links provided by NLM:

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

Study Start Date: October 2000
Study Completion Date: August 2001
Detailed Description:


  • Determine the objective response (complete hematologic remission induction) of phenylbutyrate, dexamethasone, and sargramostim (GM-CSF) in patients with refractory or relapsed t(8;21) acute myeloid leukemia.
  • Determine the correlation between histone acetylation, differentiation, and apoptosis in bone marrow mononuclear cells with response rate in patients treated with this regimen.
  • Determine the overall survival of patients on this regimen.
  • Determine the correlation between histone acetylation, differentiation, and apoptosis in bone marrow mononuclear cells with pharmacokinetics of this regimen in these patients.
  • Determine the safety and toxicity of this regimen in these patients.

OUTLINE: This is a multicenter study.

Patients receive phenylbutyrate IV continuously and sargramostim (GM-CSF) subcutaneously on days 1-7 and 15-21. Patients also receive oral dexamethasone on days 1-4 and 15-18. Treatment continues every 28 days in the absence of disease progression or unacceptable toxicity until complete hematologic remission is induced. Patients with stable disease at the end of 1 course receive at least 2 additional courses.

Patients are followed twice a week for 3 months, monthly for 1 year, every three months for the next 4 years, and then annually thereafter.

PROJECTED ACCRUAL: A total of 9-24 patients will be accrued for this study in at least 2 years.


Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No


  • Diagnosis of t(8;21) acute myeloid leukemia (AML)

    • Failed standard induction chemotherapy or stem cell transplantation (SCT) OR
    • Relapsed after standard induction chemotherapy or SCT OR
    • Refused or not a candidate for SCT or matched allogeneic sibling bone marrow transplantation or donor lymphocyte infusion OR
    • Refused of not a candidate for autologous SCT or bone marrow transplantation
  • No CNS leukemia



  • 18 and over

Performance status:

  • ECOG 0-2

Life expectancy:

  • At least 7 days


  • Not specified


  • AST or ALT no greater than 3 times upper limit of normal (ULN)
  • Bilirubin no greater than 3 times ULN
  • No hepatic disease that would preclude study


  • Creatinine no greater than 2 mg/dL
  • Creatinine clearance at least 60 mL/min
  • No renal disease that would preclude study


  • No cardiac disease that would preclude study
  • No New York Heart Association class III or IV heart disease
  • No myocardial infarction within past 8 weeks


  • No active infection except cystitis
  • Not pregnant or nursing
  • No altered mental status or seizure disorder
  • No other serious disease that would preclude study


Biologic therapy:

  • See Disease Characteristics


  • See Disease Characteristics

Endocrine therapy:

  • Not specified


  • Not specified


  • Not specified


  • At least 3 weeks since prior investigational antineoplastic drugs
  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: NCT00006240

United States, Maryland
Warren Grant Magnuson Clinical Center - NCI Clinical Studies Support
Bethesda, Maryland, United States, 20892-1182
National Heart, Lung, and Blood Institute
Bethesda, Maryland, United States, 20892
United States, New York
Mount Sinai Medical Center, NY
New York, New York, United States, 10029
United States, Pennsylvania
University of Pittsburgh Cancer Institute
Pittsburgh, Pennsylvania, United States, 15213-3489
Sponsors and Collaborators
National Heart, Lung, and Blood Institute (NHLBI)
Study Chair: Johnson Liu, MD National Heart, Lung, and Blood Institute (NHLBI)
  More Information Identifier: NCT00006240     History of Changes
Other Study ID Numbers: CDR0000068165  NHLBI-00-H-0156  NCI-171 
Study First Received: September 11, 2000
Last Updated: April 27, 2015
Health Authority: United States: Federal Government

Keywords provided by National Cancer Institute (NCI):
recurrent adult acute myeloid leukemia

Additional relevant MeSH terms:
Leukemia, Myeloid
Leukemia, Myeloid, Acute
Neoplasms by Histologic Type
Dexamethasone acetate
Dexamethasone 21-phosphate
4-phenylbutyric acid
BB 1101
Anti-Inflammatory Agents
Autonomic Agents
Peripheral Nervous System Agents
Physiological Effects of Drugs
Gastrointestinal Agents
Hormones, Hormone Substitutes, and Hormone Antagonists
Antineoplastic Agents, Hormonal
Antineoplastic Agents
Protease Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action processed this record on October 27, 2016