Pharmacogenetic Determinants Of Treatment Response In Children
Children Being Treated for Catastrophic Illness
Procedure: Blood draw
|Study Design:||Endpoint Classification: Pharmacokinetics Study
Intervention Model: Single Group Assignment
Masking: Open Label
|Official Title:||Pharmacogenetic Determinants Of Treatment Response In Children|
- To investigate whether genetic polymorphisms in genes encoding proteins involved in the metabolism or effects of drugs or environmental agents influence the disposition or effects of these xenobiotic substrates. [ Time Frame: 28 years ] [ Designated as safety issue: No ]
|Study Start Date:||July 1998|
|Estimated Study Completion Date:||March 2034|
|Estimated Primary Completion Date:||December 2033 (Final data collection date for primary outcome measure)|
All participants enrolled on this study will have blood drawn for genetic testing.
Procedure: Blood draw
DNA or RNA will typically be obtained simultaneously with blood that is drawn for clinical reasons.
Pharmacogenetics is that discipline devoted to elucidating the genetic determinants of drug response. Particularly in the area of drug metabolism, many genes exhibit genetic polymorphism; that is, a stable percentage of the population (which generally differs by ethnic group) is deficient in the functional expression of the enzyme involved, and the deficiency is typically inherited as an autosomal recessive trait. With currently known polymorphisms in drug metabolism, the percentage of homozygous deficient individuals ranges from 0.3% to as many as 90% of the population, depending on the enzyme and the ethnic group.
Our prior studies have revealed multigenic pharmacogenetic models that are significantly predictive of various drug response phenotypes (e.g., drug resistance, drug clearance, drug toxicity, disease response) in children with ALL. The large number of candidate loci and the relatively small number of patients illustrate the fact that larger sample sizes are required to definitively establish these polygenic models. The fact that there were significant race/genotype interactions, such that predictions differed in whites vs blacks, highlights the need for adequate numbers of patients within racial and ethnic groups to allow differential analysis of genotypic predictors after adjusting for confounding demographic factors in pharmacogenetic studies via stratified design and analyses.
Please refer to this study by its ClinicalTrials.gov identifier: NCT00730678
|Contact: William E Evans, Pharm.Dfirstname.lastname@example.org|
|United States, Tennessee|
|St. Jude Children's Research Hospital||Recruiting|
|Memphis, Tennessee, United States, 38105|
|Contact: William E Evans, Pharm.D 866-278-5833 email@example.com|
|Principal Investigator: William E Evans, Pharm.D|
|Principal Investigator:||William E Evans, Pharm.D||St. Jude Children's Research Hospital|