| Phase II Study of Amifostine in Pediatric Patients With Newly Diagnosed De Novo Myelodysplastic Syndromes
Alternate Title
Basic Trial Information
Objectives
Entry Criteria
Expected Enrollment
Outcomes
Outline
Trial Contact Information
Registry Information
Alternate Title
Amifostine in Treating Young Patients With Newly Diagnosed De Novo Myelodysplastic Syndromes
Basic Trial Information
| Phase | Type | Status | Age | Protocol IDs |
|---|
| Phase II | Treatment | Completed | 1 to 21 at originial diagnosis | COG-AAML0121
AAML0121, NCT00098683 |
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.
Entry Criteria Disease Characteristics:
- Histologically confirmed diagnosis of myelodysplastic syndromes (MDS)
- One of the following subtypes:
- Refractory anemia (RA)
- RA with ringed sideroblasts
- RA with excess blasts
- Refractory cytopenia with multilineage dysplasia (RCMD)
- RCMD and ringed sideroblasts
- MDS, unclassified
- MDS associated with isolated del 5(q)
- De novo disease
- No juvenile myelomonocytic leukemia
- No Down syndrome, Fanconi's anemia, or other inherited forms of hypoplastic bone marrow failure
Prior/Concurrent Therapy:
Biologic therapy - More than 8 weeks since prior growth factors
- No concurrent growth factors
- No concurrent hematopoietic stem cell transplantation
- No concurrent immunomodulating agents
Chemotherapy - No prior amifostine
- No other concurrent anticancer chemotherapy
Endocrine therapy - No concurrent daily steroid therapy
Radiotherapy Surgery Other Patient Characteristics:
Age - 1 to 21 at original diagnosis
Performance status - Karnofsky 50-100% (patients > 16 years of age)
- Lansky 50-100% (patients 1 to 16 years of age)
Life expectancy Hematopoietic - See Disease Characteristics
Hepatic - Bilirubin ≤ 1.5 times upper limit of normal (ULN)
- ALT < 2.5 times ULN
Renal - Radioisotope glomerular filtration rate ≥ 60 mL/min
OR - Creatinine clearance > 60 mL/min (based on Schwartz formula)
- Calcium normal
Other - Not pregnant or nursing
- Negative pregnancy test
- Fertile patients must use effective contraception
- Serum electrolytes normal
- Phosphorus normal
- Magnesium normal
- Glucose normal
- No other concurrent malignancy
Expected Enrollment 20A total of 10-20 patients will be accrued for this study within 5-10 months. Outcomes Primary Outcome(s)Hematological effects (complete and partial response)
Safety and efficacy
Secondary Outcome(s)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
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.
Trial Contact Information
Trial Lead Organizations Children's Oncology Group  | | | | Prasad Mathew, MD, Protocol chair | | | | | Robert Arceci, MD, PhD, Protocol co-chair | | | |
| Registry Information | | | Official Title | | A Phase II Study Of Amifostine In Children With Myelodysplastic Syndrome | | | Trial Start Date | | 2005-01-31 | | | Registered in ClinicalTrials.gov | | NCT00098683 | | | Date Submitted to PDQ | | 2004-10-13 | | | Information Last Verified | | 2006-10-20 | | | NCI Grant/Contract Number | | CA98543 |
Note: The purpose of most clinical trials listed in this database is to test new cancer treatments, or new methods of diagnosing, screening, or preventing cancer. Because all potentially harmful side effects are not known before a trial is conducted, dose and schedule modifications may be required for participants if they develop side effects from the treatment or test. The therapy or test described in this clinical trial is intended for use by clinical oncologists in carefully structured settings, and may not prove to be more effective than standard treatment. A responsible investigator associated with this clinical trial should be consulted before using this protocol.  Back to Top |
