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Genetic Study of Children With Soft Tissue Sarcoma or Rhabdomyosarcoma

Completed
Conditions
Sarcoma
Leukemia
Myelodysplastic Syndromes
Interventions
Genetic: microsatellite instability analysis
Genetic: clonality analysis
Genetic: mutation analysis
Registration Number
NCT00003793
Lead Sponsor
Children's Oncology Group
Brief Summary

RATIONALE: Determination of genetic markers for soft tissue sarcoma or rhabdomyosarcoma may help doctors identify patients who are at risk for therapy-related leukemia.

PURPOSE: Clinical trial to study genetic testing of children with soft tissue sarcoma or rhabdomyosarcoma to identify children who are at risk of developing leukemia from the chemotherapy used to treat sarcoma.

Detailed Description

OBJECTIVES:

* Identify genetically susceptible patients to therapy-induced myelodysplastic syndrome or acute myelogenous leukemia (t-MDS/AML) prior to initiation of high-dose chemotherapy for sarcoma.

* Identify patients who are at increased risk of t-MDS/AML during or after therapy.

OUTLINE: Blood is collected from patients at diagnosis (preferably before chemotherapy or transfusion), at end of therapy, and at 6 months, 1 year, 2 years, and 3 years after therapy.

Blood specimens are examined by clonality analysis (HUMARA), variant cell frequency (glycophorin A assay), GST NAT2/CYP1A1 genotyping, microsatellite instability, and ras mutation detection (single strand conformation polymorphism and sequencing of mutant alleles).

Patients do not receive the results of the genetic testing and the results do not influence the type or duration of treatment.

PROJECTED ACCRUAL: A total of 321 patients will be accrued for this study within 4 years.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
294
Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

Study Type
OBSERVATIONAL
Study Design
Not specified
Arm && Interventions
GroupInterventionDescription
Identification of Genetical suceptibility prior to therapymicrosatellite instability analysisDetermine the glutathione-s-transferase theta (GSTT1) or glutathione-s-transferase mu (GSTM1) null genotype is more frequent in individuals with t-MDS/AML. Determine the GSTT1 or GSTM1 null genotype is associated with a reduced incidence of relapse of sarcoma. Determine NAT2 or CYP1A1 genotype influences risk of t-MDS/AML. Determine development of a "mutator phenotype" as demonstrated by developing microsatellite instability is an early marker of individuals likely to progress to t-MDS/AML.
Increased Risk Of T-MDS/AML before/after Therapyclonality analysisDetermine clonal hematopoiesis develops in children receiving high intensity alkylating agent chemotherapy for sarcomas. Determine development of clonal hematopoiesis is associated with increased frequency of t-MDS/AML. Determine measurement of somatic cell mutation frequency, measured by the glycophorin A (GPA) assay prior to and after chemotherapy will predict individuals at increased risk of t-MDS/AML. Identify individuals with ras gene mutations in normal peripheral blood cells after therapy, and whether the identification of such mutations is associated with increased risk of t-MDS/AML blood cells after therapy, and whether the identification of such mutations is associated with increased risk of t-MDS/AML.
Increased Risk Of T-MDS/AML before/after Therapymicrosatellite instability analysisDetermine clonal hematopoiesis develops in children receiving high intensity alkylating agent chemotherapy for sarcomas. Determine development of clonal hematopoiesis is associated with increased frequency of t-MDS/AML. Determine measurement of somatic cell mutation frequency, measured by the glycophorin A (GPA) assay prior to and after chemotherapy will predict individuals at increased risk of t-MDS/AML. Identify individuals with ras gene mutations in normal peripheral blood cells after therapy, and whether the identification of such mutations is associated with increased risk of t-MDS/AML blood cells after therapy, and whether the identification of such mutations is associated with increased risk of t-MDS/AML.
Identification of Genetical suceptibility prior to therapymutation analysisDetermine the glutathione-s-transferase theta (GSTT1) or glutathione-s-transferase mu (GSTM1) null genotype is more frequent in individuals with t-MDS/AML. Determine the GSTT1 or GSTM1 null genotype is associated with a reduced incidence of relapse of sarcoma. Determine NAT2 or CYP1A1 genotype influences risk of t-MDS/AML. Determine development of a "mutator phenotype" as demonstrated by developing microsatellite instability is an early marker of individuals likely to progress to t-MDS/AML.
Increased Risk Of T-MDS/AML before/after Therapymutation analysisDetermine clonal hematopoiesis develops in children receiving high intensity alkylating agent chemotherapy for sarcomas. Determine development of clonal hematopoiesis is associated with increased frequency of t-MDS/AML. Determine measurement of somatic cell mutation frequency, measured by the glycophorin A (GPA) assay prior to and after chemotherapy will predict individuals at increased risk of t-MDS/AML. Identify individuals with ras gene mutations in normal peripheral blood cells after therapy, and whether the identification of such mutations is associated with increased risk of t-MDS/AML blood cells after therapy, and whether the identification of such mutations is associated with increased risk of t-MDS/AML.
Primary Outcome Measures
NameTimeMethod
Event Free Survival
Secondary Outcome Measures
NameTimeMethod

Trial Locations

Locations (96)

Southern California Permanente Medical Group

🇺🇸

Downey, California, United States

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

🇺🇸

Long Beach, California, United States

Jonsson Comprehensive Cancer Center at UCLA

🇺🇸

Los Angeles, California, United States

Children's Hospital Central California

🇺🇸

Madera, California, United States

UCSF Comprehensive Cancer Center

🇺🇸

San Francisco, California, United States

Stanford Comprehensive Cancer Center at Stanford University Medical Center

🇺🇸

Stanford, California, United States

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

🇺🇸

Farmington, Connecticut, United States

Alfred I. duPont Hospital for Children

🇺🇸

Wilmington, Delaware, United States

Lombardi Comprehensive Cancer Center at Georgetown University Medical Center

🇺🇸

Washington, District of Columbia, United States

Children's National Medical Center

🇺🇸

Washington, District of Columbia, United States

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Southern California Permanente Medical Group
🇺🇸Downey, California, United States
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