Phase II Study of Thalidomide in the Treatment of Myelodysplastic Syndromes in Adults: A Clinical and Biologic Study
I. Determine whether thalidomide improves cytopenias in patients with myelodysplastic
II. Determine the toxicity of this regimen in these patients. III. Determine whether this
regimen down regulates the peripheral blood levels of tumor necrosis factor alpha,
interferon gamma, and interleukin-12 and whether these changes correlate with clinical
response in these patients.
IV. Determine whether this regimen alters the peripheral blood T-cell subset distribution
and whether these changes correlate with clinical response in these patients.
V. Determine the effect of this regimen on bone marrow microvessel density and whether
these effects correlate with clinical response in these patients.
OUTLINE: This is a multicenter study. Patients are stratified according to prognosis
(favorable vs unfavorable). (Favorable stratum closed to accrual 12/28/01)
Patients receive oral thalidomide once daily. Treatment continues for 5 years in the absence
of disease progression or unacceptable toxicity.
Patients are followed every 6 months for 1 year and then annually for 4 years.
PROJECTED ACCRUAL: A total of 20-58 patients (10-29 per stratum) will be accrued for this
study within 20 months. (Favorable stratum closed to accrual 12/28/01)
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Confirmed response defined as complete hematologic response (CHR) or partial response (PR) or hematological improvement (HI) on 2 consecutive evaluations in terms of proportion of successes measured using criteria reported by Cheson et al
Ninety-five percent confidence intervals for the true success proportion will be calculated according to the approach of Duffy and Santner.
Up to 3 months
Alvaro Moreno Aspitia
North Central Cancer Treatment Group
United States: Food and Drug Administration
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