Amifostine in Treating Young Patients With Newly Diagnosed De Novo Myelodysplastic Syndromes - Full Text View

Sponsor:	Children's Oncology Group
Collaborator:	National Cancer Institute (NCI)
Information provided by:	National Cancer Institute (NCI)
ClinicalTrials.gov Identifier:	NCT00098683

Purpose

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.

Condition	Intervention	Phase
Myelodysplastic Syndromes Myelodysplastic/Myeloproliferative Neoplasms	Drug: amifostine trihydrate	Phase II

Study Type:	Interventional
Study Design:	Masking: Open Label Primary Purpose: Treatment
Official Title:	A Phase II Study Of Amifostine In Children With Myelodysplastic Syndrome

Resource links provided by NLM:

MedlinePlus related topics: Anemia Cancer Myelodysplastic Syndromes

Drug Information available for: Amifostine

U.S. FDA Resources

Further study details as provided by National Cancer Institute (NCI):

Primary Outcome Measures:

Hematological effects (complete and partial response) [ Designated as safety issue: No ]
Safety and efficacy [ Designated as safety issue: Yes ]

Secondary Outcome Measures:

Efficacy [ Designated as safety issue: No ]
Duration of progression-free remission [ Designated as safety issue: No ]
Effect of karyotypic abnormalities on survival [ Designated as safety issue: No ]
Effect of the number of cytopenias on survival [ Designated as safety issue: No ]
Correlation of the duration of time from diagnosis of myelodysplastic syndromes until conversion to acute myeloid leukemia [ Designated as safety issue: No ]

Estimated Enrollment:	20
Study Start Date:	January 2005
Primary Completion Date:	October 2007 (Final data collection date for primary outcome measure)

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.

Eligibility

Ages Eligible for Study:	1 Year to 21 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

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 treatment-induced MDS
No juvenile myelomonocytic leukemia
No Down syndrome, Fanconi's anemia, or other inherited forms of hypoplastic bone marrow failure

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

At least 8 weeks

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

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

Not specified

Surgery

Not specified

Other

No prior therapy for MDS

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00098683

Show 85 Study Locations

Sponsors and Collaborators

Children's Oncology Group

National Cancer Institute (NCI)

Investigators

Study Chair:	Prasad Mathew, MD	University of New Mexico
Investigator:	Robert J. Arceci, MD, PhD	Sidney Kimmel Comprehensive Cancer Center

More Information

Additional Information:

Clinical trial summary from the National Cancer Institute's PDQ® database This link exits the ClinicalTrials.gov site

Publications:

Mathew P, Gerbing R, Alonzo TA, Wallas T, Gong JZ, Jasty R, Jorstad DT, Raimondi SC, Chavez CM, Eisenberg NL, Hirsch B, Gamis A, Smith FO, Arceci RJ. A phase II study of amifostine in children with myelodysplastic syndrome: A report from the Children's Oncology Group study (AAML0121). Pediatr Blood Cancer. 2011 Jun 16; [Epub ahead of print]

ClinicalTrials.gov Identifier:	NCT00098683 History of Changes
Other Study ID Numbers:	CDR0000398140, COG-AAML0121
Study First Received:	December 7, 2004
Last Updated:	June 25, 2011
Health Authority:	United States: Federal Government

Keywords provided by National Cancer Institute (NCI):

refractory anemia with excess blasts
refractory anemia
refractory anemia with ringed sideroblasts

de novo myelodysplastic syndromes
myelodysplastic/myeloproliferative neoplasm, unclassifiable
refractory cytopenia with multilineage dysplasia

Additional relevant MeSH terms:

Neoplasms
Myelodysplastic Syndromes
Preleukemia
Myeloproliferative Disorders
Myelodysplastic-Myeloproliferative Diseases
Bone Marrow Diseases
Hematologic Diseases

Precancerous Conditions
Amifostine
Radiation-Protective Agents
Protective Agents
Physiological Effects of Drugs
Pharmacologic Actions

ClinicalTrials.gov processed this record on February 09, 2012