A Pilot Study of Phenylbutyrate, Dexamethasone and GM-CSF in Refractory or Relapsed t(8;21) Acute Myeloid Leukemia
18 Years
N/A
Both
Leukemia
Trial Information
A Pilot Study of Phenylbutyrate, Dexamethasone and GM-CSF in Refractory or Relapsed t(8;21) Acute Myeloid Leukemia
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.
Inclusion 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
Type of Study:
Interventional
Study Design:
Primary Purpose: Treatment
Principal Investigator
Johnson Liu, MD
Investigator Role:
Study Chair
Investigator Affiliation:
National Heart, Lung, and Blood Institute (NHLBI)
Authority:
United States: Federal Government
Study ID:
CDR0000068165
NCT ID:
NCT00006240
Start Date:
October 2000
Completion Date:
August 2001
Related Keywords:
- Leukemia
- recurrent adult acute myeloid leukemia
- Leukemia
- Leukemia, Myeloid, Acute
- Leukemia, Myeloid
Name | Location |
|---|---|
| Mount Sinai Medical Center, NY | New York, New York 10029 |
| University of Pittsburgh Cancer Institute | Pittsburgh, Pennsylvania 15213 |
| Warren Grant Magnuson Clinical Center - NCI Clinical Studies Support | Bethesda, Maryland 20892-1182 |
| National Heart, Lung, and Blood Institute | Bethesda, Maryland 20892 |
