Bexarotene and GM-CSF in Treating Patients With Myelodysplastic Syndrome or Acute Myeloid Leukemia
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|ClinicalTrials.gov Identifier: NCT00425477|
Recruitment Status : Completed
First Posted : January 23, 2007
Results First Posted : September 5, 2018
Last Update Posted : October 5, 2018
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.
|Condition or disease||Intervention/treatment||Phase|
|Leukemia Myelodysplastic Syndromes Myelodysplastic/Myeloproliferative Diseases||Biological: sargramostim Drug: bexarotene Genetic: cytogenetic analysis Genetic: fluorescence in situ hybridization Other: flow cytometry Other: laboratory biomarker analysis Procedure: biopsy||Phase 2|
- 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.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||26 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Phase II Study of Bexarotene + Sargromastastin as Agents of Differentiation in MDS and AML|
|Actual Study Start Date :||November 2006|
|Actual Primary Completion Date :||September 30, 2016|
|Actual Study Completion Date :||September 30, 2016|
Experimental: Bexarotene + GM-CSF
BEX and GM-CSF were administered in 4 week cycles. BEX was given orally with food daily for 28 days at the FDA-approved dose for treatment of CTCL of 300 mg/m2 and GM-CSF was given at a daily dose of 125 µg/m2 subcutaneously for 28 days.
Genetic: cytogenetic analysis
Genetic: fluorescence in situ hybridization
Other: flow cytometry
Other: laboratory biomarker analysis
- Clinical Response (Complete and Partial) [ Time Frame: assessed after 2 cycles, up to 2 years ]Response to treatment was assessed after two cycles, according to International Working Group (IWG) criteria.
- Clinical Activity as Measured by Change in Peripheral Blood Counts and Changes in Transfusion Requirements [ Time Frame: Baseline and after two cycles ]ANC count at baseline and after two cycles were measured and compared. Due to the limited number of clinical responders, the changes in transfusion requirements were not measured.
- Biological Activity as Measured by in Vivo Induction of Terminal Differentiation of Myeloid Progenitors and in Vivo Changes in Detectable Chromosomal Abnormalities [ Time Frame: Baseline and 6, 12, 24, and 36 weeks ]
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): 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 at Johns Hopkins|