Myelodysplastic Syndrome Clinical Trial
RAD001(Everolimus) in Treating Patients With Myelodysplastic Syndromes
Summary
RATIONALE: RAD001(Everolimus) may stop the growth of cancer cells by blocking some of the enzymes needed for their growth and by blocking blood flow to the cancer.
PURPOSE: This phase II trial is studying how well RAD001(everolimus) works in treating patients with myelodysplastic syndromes.
Full Description
OBJECTIVES:
Primary
Determine the clinical activity (improvement in erythroid response and/or improvement in other cytopenias, bone marrow morphology/cytogenetics) of RAD001(everolimus) in patients with low or intermediate-1 risk myelodysplastic syndromes.
Assess the toxicity of this drug in these patients.
Secondary
Examine laboratory correlates (S6K1 levels, angiogenesis pre- and post-treatment) and determine how these correlates correspond to dosing and clinical activity of RAD001(everolimus).
Evaluate the presence of HLA-DR15 and cytotoxic T-cell populations in patients pre- and post-treatment and correlate this with response to treatment.
Examine the incidence of the null GSTT-1 phenotype in myelodysplastic syndromes patients and correlate this with response to RAD001(everolimus).
OUTLINE: Patients receive oral RAD001(everolimus) once daily on days 1-21. Courses repeat every 28 days in the absence of disease progression or relapse.
Blood samples are collected periodically during study. Samples are analyzed for S6K1 activity, effector T cells by flow cytometry, GSTT-1 by PCR, and HLA-DR15 levels.
Eligibility Criteria
DISEASE CHARACTERISTICS:
Low or intermediate-1 risk myelodysplastic syndromes by International Prognostic Scoring System (IPSS) criteria
IPSS score < 1.5
Requiring transfusion of 2 units of red blood cells at least once a month (four weeks prior to accrual on study)
High levels of endogenous epoetin alfa (i.e., > 200 mU/mL)
Unlikely to respond to epoetin alfa, or has a documented clinical non-response to epoetin alfa (at a dose of ≥ 40,000 U weekly) or darbepoetin alfa (at a dose > 200 mcg every other week) (i.e., < 2 g/dL increase in hemoglobin and no decrease in transfusion requirements after at least 4 weeks of treatment)
No chronic myelomonocytic leukemia
PATIENT CHARACTERISTICS:
ECOG Performance Status of 0-2
Liver enzymes (AST and ALT) and total bilirubin ≤ 2 times upper limit of normal
Serum creatinine ≤ 2 times upper limits of normal
No clinically significant anemia due to iron, B12, or folate deficiencies; autoimmune or hereditary hemolysis; or gastrointestinal bleeding
Not pregnant or nursing
Negative pregnancy test
Fertile patients must use effective contraception
No other serious or poorly controlled medical condition that could be exacerbated by or complicate compliance with study therapy
PRIOR CONCURRENT THERAPY:
At least 4 weeks since prior treatment (including growth factors)
No chronic use (> 2 weeks) of physiologic doses of a corticosteroid agent (dose equivalent to > 10 mg/day of prednisone) within 28 days of the first day of study drug
No concurrent use of another investigational agent
No concurrent therapy with any cytotoxic drugs, steroids, or growth factors
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There is 1 Location for this study
Cleveland Ohio, 44195, United States
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