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

This study has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT00006240
First Posted: July 28, 2003
Last Update Posted: April 28, 2015
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.
Information provided by:
National Cancer Institute (NCI)
  Purpose

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
Leukemia 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:

OBJECTIVES:

  • 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.

  Eligibility

Information from the National Library of Medicine

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

DISEASE CHARACTERISTICS:

  • 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

PATIENT CHARACTERISTICS:

Age:

  • 18 and over

Performance status:

  • ECOG 0-2

Life expectancy:

  • At least 7 days

Hematopoietic:

  • Not specified

Hepatic:

  • 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

Renal:

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

Cardiovascular:

  • 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

Other:

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

PRIOR CONCURRENT THERAPY:

Biologic therapy:

  • See Disease Characteristics

Chemotherapy:

  • See Disease Characteristics

Endocrine therapy:

  • Not specified

Radiotherapy:

  • Not specified

Surgery:

  • Not specified

Other:

  • At least 3 weeks since prior investigational antineoplastic drugs
  Contacts and Locations
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 ClinicalTrials.gov identifier (NCT number): NCT00006240


Locations
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)
Investigators
Study Chair: Johnson Liu, MD National Heart, Lung, and Blood Institute (NHLBI)
  More Information

ClinicalTrials.gov Identifier: NCT00006240     History of Changes
Other Study ID Numbers: CDR0000068165
NHLBI-00-H-0156
NCI-171
First Submitted: September 11, 2000
First Posted: July 28, 2003
Last Update Posted: April 28, 2015
Last Verified: November 2002

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

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