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

This study has been completed.
Sponsor:
Collaborator:
Information provided by (Responsible Party):
Children's Oncology Group
ClinicalTrials.gov Identifier:
NCT00098683
First received: December 7, 2004
Last updated: February 11, 2014
Last verified: February 2014
  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 2

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:


Further study details as provided by Children's Oncology Group:

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 ]

Enrollment: 10
Study Start Date: January 2005
Study Completion Date: October 2009
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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

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

  Show 85 Study Locations
Sponsors and Collaborators
Children's Oncology Group
Investigators
Study Chair: Prasad Mathew, MD University of New Mexico Cancer Center
Investigator: Robert J. Arceci, MD, PhD Sidney Kimmel Comprehensive Cancer Center
  More Information

Additional Information:
Publications:
Responsible Party: Children's Oncology Group
ClinicalTrials.gov Identifier: NCT00098683     History of Changes
Other Study ID Numbers: AAML0121, CDR0000398140, COG-AAML0121
Study First Received: December 7, 2004
Last Updated: February 11, 2014
Health Authority: United States: Federal Government

Keywords provided by Children's Oncology Group:
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
Syndrome
Bone Marrow Diseases
Hematologic Diseases
Precancerous Conditions
Disease
Pathologic Processes
Amifostine
Radiation-Protective Agents
Protective Agents
Physiological Effects of Drugs
Pharmacologic Actions

ClinicalTrials.gov processed this record on September 22, 2014