Pharmacokinetics of Understudied Drugs Administered to Children Per Standard of Care (PTN_POPS)
Understudied drugs will be administered to children per standard of care as prescribed by their treating caregiver and only biological sample collection during the time of drug administration will be involved. A total of approximately 2000 children aged <21 years who are receiving these drugs for standard of care will be enrolled and will be followed for up a maximum of 90 days. The goal of this study is to characterize the pharmacokinetics of understudied drugs for which specific dosing recommendations and safety data are lacking. The prescribing of drugs to children will not be part of this protocol. Taking advantage of procedures done as part of routine medical care (i.e. blood draws) this study will serve as a tool to better understand drug exposure in children receiving these drugs per standard of care. The data collected through this initiative will also provide valuable pharmacokinetic and dosing information of drugs in different pediatric age groups as well as special pediatric populations (i.e. obese).
|Study Design:||Observational Model: Cohort
Time Perspective: Prospective
|Official Title:||Pharmacokinetics of Understudied Drugs Administered to Children Per Standard of Care|
- Composite of pharmacokinetic outcomes for understudied drugs in children [ Time Frame: Data will be collected throughout the hospital or outpatient stay up to 90 days ] [ Designated as safety issue: No ]
As appropriate for each study drug, the following additional PK parameters will be estimated:
- maximum concentration (Cmax)
- time to achieve maximum concentration (Tmax)
- absorption rate constant (ka)
- elimination rate constant (kel)
- half-life (t1/2)
- area under the curve (AUC)
Penetration into body fluids will be determined by comparing exposure (i.e. AUC, Cmax) ratios between the body fluid and plasma or comparison of concentrations in paired samples.
- Composite pharmacodynamic outcomes of understudied drugs in children [ Time Frame: Data will be collected throughout the hospital or outpatient stay up to 90 days ] [ Designated as safety issue: No ]When applicable, Monte Carlo simulations will be performed to evaluate therapeutic target attainment rates (pharmacodynamics) in the population of interest. The final PK model and parameters estimated in the population PK analysis will be used to perform these simulations.
- Biomarkers associated with understudied drugs in children [ Time Frame: Data will be collected throughout the hospital or outpatient stay up to 90 days ] [ Designated as safety issue: No ]The dosing, sampling, and demographic information recorded on the eCRF will be merged with the bioanalytical information to create a biomarker dataset for each study drug. Biomarkers will be identified using metabolomics/proteomics and pharmacogenomics methodologies. Samples for biomarker analysis will be stored for future use in a PTN designated biorepository. Associations between biomarkers and drug exposure will be explored by visual inspection (i.e. scatter plots) and statistical comparisons as needed.
Biospecimen Retention: Samples With DNA
|Study Start Date:||November 2011|
|Estimated Study Completion Date:||June 2014|
|Estimated Primary Completion Date:||June 2014 (Final data collection date for primary outcome measure)|
The purpose of this study is to characterize the PK ( Pharmacokinetics) of understudied drugs administered to children per standard of care as prescribed by their treating caregiver. This will be accomplished by the collection of biological samples during the time of drug administration per standard of care as prescribed by the caregiver. The prescribing of drugs to children will not be part of this protocol.
Aim #1: Evaluate the PK of understudied drugs currently being administered to children.
Hypothesis #1: The PK of understudied drugs in children will differ from adults and within children according to pediatric age groups or special population.
Aim #2: Explore the pharmacodynamics (PD) of understudied drugs currently being administered to children.
Hypothesis #2: The PD of targeted drugs in children will differ from adults.
Aim #3: Evaluate the influence of genetic factors, metabolic and protein profiles on therapeutic exposure.
Hypothesis #3: Genetic polymorphisms in drug metabolizing enzymes and metabolic and proteomic profiles will impact drug exposure in children.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01431326
|Contact: Chiara Melloni, MDfirstname.lastname@example.org|
|Contact: Barrie L Harper, MT (ASCP)||email@example.com|
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|Principal Investigator:||Michael Cohen-Wolkowiez, MD||Duke University|
|Study Chair:||Chiara Melloni, MD||Duke University|