Amifostine in Treating Young Patients With Newly Diagnosed De Novo Myelodysplastic Syndromes
RATIONALE: Drugs used in chemotherapy, such as amifostine, work in different ways to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing.
PURPOSE: This phase II trial is studying how well amifostine works in treating young patients with newly diagnosed de novo myelodysplastic syndromes.
|Myelodysplastic Syndromes Myelodysplastic/Myeloproliferative Neoplasms||Drug: amifostine trihydrate||Phase 2|
|Study Design:||Masking: None (Open Label)
Primary Purpose: Treatment
|Official Title:||A Phase II Study Of Amifostine In Children With Myelodysplastic Syndrome|
- Hematological effects (complete and partial response)
- Safety and efficacy
- Duration of progression-free remission
- Effect of karyotypic abnormalities on survival
- Effect of the number of cytopenias on survival
- Correlation of the duration of time from diagnosis of myelodysplastic syndromes until conversion to acute myeloid leukemia
|Study Start Date:||January 2005|
|Study Completion Date:||October 2009|
|Primary Completion Date:||October 2007 (Final data collection date for primary outcome measure)|
- Determine the hematologic effects of amifostine, in terms of, complete and partial response, in pediatric patients with newly diagnosed de novo myelodysplastic syndromes (MDS).
- Determine the safety and efficacy of this drug in these patients.
- Determine the efficacy of this drug in preventing conversion of MDS to acute myeloid leukemia (AML) in terms of the proportion of patients who remain free of AML at the completion of study treatment.
- Determine the duration of progression-free remission from MDS conversion to AML in patients treated with this drug.
- Determine the effect of karyotypic abnormalities on survival and the duration from diagnosis of MDS until conversion to AML in patients treated with this drug.
- Determine the effect of bone marrow blast count on survival and the duration from diagnosis of MDS until conversion to AML in patients treated with this drug.
- Determine the effect of the number of cytopenias on survival in patients treated with this drug.
- Correlate the duration of time from diagnosis of MDS until conversion to AML with survival in patients treated with this drug.
OUTLINE: This is a multicenter study.
Patients receive amifostine IV over 1-3 minutes on days 1, 3, 5, 8, 10, 12, 15, 17, and 19. Treatment repeats every 5 weeks for 2 courses in the absence of disease progression or unacceptable toxicity. Patients with stable or responding disease who are planning to undergo matched donor bone marrow or cord blood transplantation continue therapy until transplantation. Patients with stable or responding disease who are not undergoing transplantation may receive up to 4 additional courses of amifostine in the absence of disease progression or unacceptable toxicity.
Following completion of therapy with amifostine, patients are followed monthly for 1 year, every 2 months for 1 year, every 3 months for 1 year, every 6 months for 1 year, and then annually thereafter.
PROJECTED ACCRUAL: A total of 10-20 patients will be accrued for this study within 5-10 months.
Please refer to this study by its ClinicalTrials.gov identifier: NCT00098683
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|Study Chair:||Prasad Mathew, MD||University of New Mexico Cancer Center|
|OverallOfficial:||Robert J. Arceci, MD, PhD||Sidney Kimmel Comprehensive Cancer Center|