Phenylbutyrate, Dexamethasone, and Sargramostim in Treating Patients With Refractory or Relapsed Acute Myeloid Leukemia
|ClinicalTrials.gov Identifier: NCT00006240|
Recruitment Status : Completed
First Posted : July 28, 2003
Last Update Posted : April 28, 2015
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 or disease||Intervention/treatment||Phase|
|Leukemia||Biological: sargramostim Drug: dexamethasone Drug: oral sodium phenylbutyrate||Phase 2|
- 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 (Clinical Trial)|
|Official Title:||A Pilot Study of Phenylbutyrate, Dexamethasone and GM-CSF in Refractory or Relapsed t(8;21) Acute Myeloid Leukemia|
|Study Start Date :||October 2000|
|Actual Study Completion Date :||August 2001|
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): 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|
|Study Chair:||Johnson Liu, MD||National Heart, Lung, and Blood Institute (NHLBI)|