NCT00006240

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

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

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: —
• Sponsor: National Heart, Lung, and Blood Institute (NHLBI) · NIH
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Leukemia

Interventions

Type	Name	Description
BIOLOGICAL	sargramostim
DRUG	dexamethasone
DRUG	oral sodium phenylbutyrate

Timeline

• Start date: 2000-10-01
• Completion: 2001-08-01
• First posted: 2003-07-28
• Last updated: 2015-04-28

Locations

4 sites across 1 country: United States