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

April 27, 2015 updated by: National Heart, Lung, and Blood Institute (NHLBI)

A Pilot Study of Phenylbutyrate, Dexamethasone and GM-CSF in Refractory or Relapsed t(8;21) Acute Myeloid Leukemia

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Maryland
      • Bethesda, Maryland, United States, 20892-1182
        • Warren Grant Magnuson Clinical Center - NCI Clinical Studies Support
      • Bethesda, Maryland, United States, 20892
        • National Heart, Lung, and Blood Institute
    • New York
      • New York, New York, United States, 10029
        • Mount Sinai Medical Center, NY
    • Pennsylvania
      • Pittsburgh, Pennsylvania, United States, 15213-3489
        • University of Pittsburgh Cancer Institute

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

National Heart, Lung, and Blood Institute (NHLBI)

Investigators

  • Study Chair: Johnson Liu, MD, National Heart, Lung, and Blood Institute (NHLBI)

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

October 1, 2000

Study Completion (Actual)

August 1, 2001

Study Registration Dates

First Submitted

September 11, 2000

First Submitted That Met QC Criteria

July 25, 2003

First Posted (Estimate)

July 28, 2003

Study Record Updates

Last Update Posted (Estimate)

April 28, 2015

Last Update Submitted That Met QC Criteria

April 27, 2015

Last Verified

November 1, 2002

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CDR0000068165
  • NHLBI-00-H-0156
  • NCI-171

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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