NCT00098683

Amifostine in Treating Young Patients With Newly Diagnosed De Novo Myelodysplastic Syndromes

A Phase II Study Of Amifostine In Children With Myelodysplastic Syndrome

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 10 (actual)
• Sponsor: Children's Oncology Group · Network
• Sex: All
• Age: 1 Year – 21 Years
• Healthy volunteers: Not accepted

Summary

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.

Detailed description

OBJECTIVES: Primary * 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. Secondary * 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.

Conditions

• Myelodysplastic Syndromes
• Myelodysplastic/Myeloproliferative Neoplasms

Interventions

Type	Name	Description
DRUG	amifostine trihydrate

Timeline

• Start date: 2005-01-01
• Primary completion: 2007-10-01
• Completion: 2009-10-01
• First posted: 2004-12-08
• Last updated: 2014-02-13

Locations

85 sites across 4 countries: United States, Australia, Canada, Puerto Rico