Bexarotene and GM-CSF in Treating Patients With Myelodysplastic Syndrome or Acute Myeloid Leukemia
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.
Genetic: cytogenetic analysis
Genetic: fluorescence in situ hybridization
Other: flow cytometry
Other: laboratory biomarker analysis
|Study Design:||Masking: Open Label
Primary Purpose: Treatment
|Official Title:||A Phase II Study of Bexarotene + Sargromastastin as Agents of Differentiation in MDS and AML|
- Clinical response (complete and partial) [ Designated as safety issue: No ]
- Clinical activity as measured by improved peripheral blood counts and changes in transfusion requirements [ Designated as safety issue: No ]
- Biological activity as measured by in vivo induction of terminal differentiation of myeloid progenitors and in vivo changes in detectable chromosomal abnormalities [ Designated as safety issue: No ]
|Study Start Date:||November 2006|
|Estimated Primary Completion Date:||July 2016 (Final data collection date for primary outcome measure)|
- Assess the clinical response in patients with myelodysplastic syndromes or acute myeloid leukemia treated with bexarotene and sargramostim (GM-CSF).
- 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.
Please refer to this study by its ClinicalTrials.gov identifier: NCT00425477
|United States, Maryland|
|Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins|
|Baltimore, Maryland, United States, 21231-2410|
|Study Chair:||B. Douglas Smith, MD||Sidney Kimmel Comprehensive Cancer Center|