Genetic Study of Children With Soft Tissue Sarcoma or Rhabdomyosarcoma
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|ClinicalTrials.gov Identifier: NCT00003793|
Recruitment Status : Completed
First Posted : January 27, 2003
Last Update Posted : August 5, 2014
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.
|Condition or disease||Intervention/treatment|
|Leukemia Myelodysplastic Syndromes Sarcoma||Genetic: clonality analysis Genetic: microsatellite instability analysis Genetic: mutation analysis|
- 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.
|Study Type :||Observational|
|Actual Enrollment :||294 participants|
|Official Title:||Clinical and Biological Predictors of Therapy-Related Leukemia|
|Study Start Date :||December 1998|
|Actual Primary Completion Date :||December 2005|
|Actual Study Completion Date :||March 2007|
Identification of Genetical suceptibility prior to therapy
Determine 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.
|Genetic: microsatellite instability analysis Genetic: mutation analysis|
Increased Risk Of T-MDS/AML before/after Therapy
Determine 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.
|Genetic: clonality analysis Genetic: microsatellite instability analysis Genetic: mutation analysis|
- Event Free Survival
Biospecimen Retention: Samples With DNA
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00003793
Show 96 Study Locations
|Study Chair:||Stella M. Davies, MBBS, PhD||Children's Hospital Medical Center, Cincinnati|