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

Phase 2
Completed
Conditions
Myelodysplastic Syndromes
Myelodysplastic/Myeloproliferative Neoplasms
Registration Number
NCT00098683
Lead Sponsor
Children's Oncology Group
Brief 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.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
10
Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

Study Type
INTERVENTIONAL
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
Hematological effects (complete and partial response)
Safety and efficacy
Secondary Outcome Measures
NameTimeMethod
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
Efficacy

Trial Locations

Locations (85)

Phoenix Children's Hospital

🇺🇸

Phoenix, Arizona, United States

Arkansas Cancer Research Center at University of Arkansas for Medical Sciences

🇺🇸

Little Rock, Arkansas, United States

Jonathan Jaques Children's Cancer Center at Miller Children's Hospital

🇺🇸

Long Beach, California, United States

Children's Hospital Central California

🇺🇸

Madera, California, United States

Kaiser Permanente Medical Center - Oakland

🇺🇸

Sacramento, California, United States

Carole and Ray Neag Comprehensive Cancer Center at the University of Connecticut Health Center

🇺🇸

Farmington, Connecticut, United States

Lee Cancer Care of Lee Memorial Health System

🇺🇸

Fort Myers, Florida, United States

University of Florida Shands Cancer Center

🇺🇸

Gainesville, Florida, United States

Memorial Cancer Institute at Memorial Regional Hospital

🇺🇸

Hollywood, Florida, United States

Miami Children's Hospital

🇺🇸

Miami, Florida, United States

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Phoenix Children's Hospital
🇺🇸Phoenix, Arizona, United States

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