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

Primary Purpose

Leukemia

Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
sargramostim
dexamethasone
oral sodium phenylbutyrate
Sponsored by
National Heart, Lung, and Blood Institute (NHLBI)
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an interventional treatment trial for Leukemia focused on measuring recurrent adult acute myeloid leukemia

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Warren Grant Magnuson Clinical Center - NCI Clinical Studies Support
  • National Heart, Lung, and Blood Institute
  • Mount Sinai Medical Center, NY
  • University of Pittsburgh Cancer Institute

Outcomes

Primary Outcome Measures

Secondary Outcome Measures

Full Information

First Posted
September 11, 2000
Last Updated
April 27, 2015
Sponsor
National Heart, Lung, and Blood Institute (NHLBI)
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1. Study Identification

Unique Protocol Identification Number
NCT00006240
Brief Title
Phenylbutyrate, Dexamethasone, and Sargramostim in Treating Patients With Refractory or Relapsed Acute Myeloid Leukemia
Official Title
A Pilot Study of Phenylbutyrate, Dexamethasone and GM-CSF in Refractory or Relapsed t(8;21) Acute Myeloid Leukemia
Study Type
Interventional

2. Study Status

Record Verification Date
November 2002
Overall Recruitment Status
Completed
Study Start Date
October 2000 (undefined)
Primary Completion Date
undefined (undefined)
Study Completion Date
August 2001 (Actual)

3. Sponsor/Collaborators

Name of the Sponsor
National Heart, Lung, and Blood Institute (NHLBI)

4. Oversight

5. Study Description

Brief Summary
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.
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Leukemia
Keywords
recurrent adult acute myeloid leukemia

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2

8. Arms, Groups, and Interventions

Intervention Type
Biological
Intervention Name(s)
sargramostim
Intervention Type
Drug
Intervention Name(s)
dexamethasone
Intervention Type
Drug
Intervention Name(s)
oral sodium phenylbutyrate

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Johnson Liu, MD
Organizational Affiliation
National Heart, Lung, and Blood Institute (NHLBI)
Official's Role
Study Chair
Facility Information:
Facility Name
Warren Grant Magnuson Clinical Center - NCI Clinical Studies Support
City
Bethesda
State/Province
Maryland
ZIP/Postal Code
20892-1182
Country
United States
Facility Name
National Heart, Lung, and Blood Institute
City
Bethesda
State/Province
Maryland
ZIP/Postal Code
20892
Country
United States
Facility Name
Mount Sinai Medical Center, NY
City
New York
State/Province
New York
ZIP/Postal Code
10029
Country
United States
Facility Name
University of Pittsburgh Cancer Institute
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15213-3489
Country
United States

12. IPD Sharing Statement

Learn more about this trial

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

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