NCT00425477

Bexarotene and GM-CSF in Treating Patients With Myelodysplastic Syndrome or Acute Myeloid Leukemia

A Phase II Study of Bexarotene + Sargromastastin as Agents of Differentiation in MDS and AML

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 26 (actual)
• Sponsor: Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins · Academic / Other
• Sex: All
• Age: 18 Years – 120 Years
• Healthy volunteers: Not accepted

Summary

RATIONALE: Bexarotene may help cancer or abnormal cells become more like normal cells, and to grow and spread more slowly. Colony-stimulating factors, such as GM-CSF, may increase the number of immune cells found in bone marrow or peripheral blood. Giving bexarotene together with GM-CSF may be an effective treatment for myelodysplastic syndrome (MDS) or acute myeloid leukemia. PURPOSE: This phase II trial is studying how well giving bexarotene together with GM-CSF works in treating patients with MDS or acute myeloid leukemia.

Detailed description

OBJECTIVES: Primary * Assess the clinical response in patients with myelodysplastic syndromes or acute myeloid leukemia treated with bexarotene and sargramostim (GM-CSF). Secondary * Determine the clinical activity of this regimen, in terms of transfusion requirements, in these patients. * Determine the biological activity of this regimen, in terms of biological markers and cytogenetic abnormalities, in these patients. * Assess the toxicity profile of this regimen in these patients. OUTLINE: Patients receive oral bexarotene and sargramostim (GM-CSF) subcutaneously on days 1-28. Treatment repeats every 28 days for up to 6 courses in the absence of disease progression or unacceptable toxicity. Blood and bone marrow samples are collected at baseline and after 1 or 2 courses of study therapy. Samples are examined by flow cytometry for laboratory studies, including biological markers, and by fluorescent in situ hybridization (FISH) for cytogenetic changes. After completion of study treatment, patients are followed periodically for 6 months. PROJECTED ACCRUAL: A total of 18 patients will be accrued for this study.

Conditions

• Leukemia
• Myelodysplastic Syndromes
• Myelodysplastic/Myeloproliferative Diseases

Interventions

Type	Name	Description
BIOLOGICAL	sargramostim
DRUG	bexarotene
GENETIC	cytogenetic analysis
GENETIC	fluorescence in situ hybridization
OTHER	flow cytometry
OTHER	laboratory biomarker analysis
PROCEDURE	biopsy

Timeline

• Start date: 2006-11-01
• Primary completion: 2016-09-30
• Completion: 2016-09-30
• First posted: 2007-01-23
• Last updated: 2018-10-05
• Results posted: 2018-09-05

Locations

1 site across 1 country: United States