Pharmacokinetics of Understudied Drugs Administered to Children Per Standard of Care (PTN_POPS)

August 31, 2023 updated by: Daniel Benjamin
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 7000 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 Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

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.

Study Type

Observational

Enrollment (Actual)

3520

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Canada
    • Manitoba
      • Winnipeg, Manitoba, Canada, R3E 3P4
        • Manitoba Institute of Child Health
    • Ontario
      • Ottawa, Ontario, Canada, K1H 8L1
        • Children's Hospital of Eastern Ontario
      • Toronto, Ontario, Canada, M5G 1X8
        • The Hospital for Sick Children
    • Quebec
      • Montreal, Quebec, Canada, T3T 1C5
        • Hospital Sainte-Justine
  • Israel
      • Petah Tikva, Israel, 49202
        • Schneider Children's Medical Center of Israel
    • Tel Aviv
      • Zerifin, Tel Aviv, Israel, 70300
        • Assaf Harofeh Medical Center
  • Singapore
      • Singapore, Singapore, 229899
        • KK Women's and Children's Hospital Pte Ltd
  • United Kingdom
    • Hampshire
      • Southampton, Hampshire, United Kingdom, SO16 6YD
        • Southampton General Hospital
    • Merseyside
      • Liverpool, Merseyside, United Kingdom, L12 2AP
        • Alder Hey Children's Hospital
  • United States
    • Alaska
      • Anchorage, Alaska, United States, 99508
        • Alaska Native Medical Center
    • Arkansas
      • Little Rock, Arkansas, United States, 72202
        • Arkansas Children's Hospital
    • California
      • La Jolla, California, United States, 92093
        • University of California at San Diego Medical Center
      • Los Angeles, California, United States, 90036
        • Axis Clinical Trials
      • Los Angeles, California, United States, 90095
        • University Of California, Los Angeles Medical Center
    • Colorado
      • Aurora, Colorado, United States, 80045
        • The Children's Hospital Colorado
    • Connecticut
      • New Haven, Connecticut, United States, 06504
        • Yale New Haven Children's Hospital
    • Delaware
      • Wilmington, Delaware, United States, 19803
        • Alfred I. DuPont Hospital for Children
    • District of Columbia
      • Washington, District of Columbia, United States, 20010
        • Children's National Medical Center
    • Florida
      • Jacksonville, Florida, United States, 32209
        • University of Florida Jacksonville Shands Medical Center
    • Hawaii
      • Honolulu, Hawaii, United States, 96826
        • Kapiolani Womens and Childrens Medical Center
    • Illinois
      • Chicago, Illinois, United States, 60614
        • Lurie Children's Hospital of Chicago
    • Indiana
      • Indianapolis, Indiana, United States, 46202
        • Riley Hospital for Children at Indiana University
    • Kansas
      • Fairway, Kansas, United States, 66205
        • University of Kansas Medical Center
      • Kansas City, Kansas, United States, 66160
        • Children's Mercy Hospital and Clinics
      • Wichita, Kansas, United States, 67214
        • Wesley Medical Center
    • Kentucky
      • Louisville, Kentucky, United States, 40202
        • Norton Children's Hospital
    • Louisiana
      • New Orleans, Louisiana, United States, 70112
        • Tulane University Health Science Center
      • New Orleans, Louisiana, United States, 70115-6969
        • Ochsner Baptist Clinical Trials Unit
    • Maryland
      • Baltimore, Maryland, United States, 21201
        • University of Maryland
    • Michigan
      • Detroit, Michigan, United States, 48201
        • Children's Hospital of Michigan
    • Mississippi
      • Jackson, Mississippi, United States, 39216
        • University of Mississippi Medical Center
    • Missouri
      • Kansas City, Missouri, United States, 64108
        • Children's Mercy Hospitals and Clinics
    • Montana
      • Missoula, Montana, United States, 59804
        • University of Montana
    • Nebraska
      • Omaha, Nebraska, United States, 68198
        • University of Nebraska Medical Center
    • New Hampshire
      • Lebanon, New Hampshire, United States, 03756
        • Dartmouth-Hitchcock Medical Center
    • New Mexico
      • Albuquerque, New Mexico, United States, 87131
        • University of New Mexico, Health Sciences Center
    • North Carolina
      • Chapel Hill, North Carolina, United States, 27599
        • UNC Hospital Neonatal-Perinatal Medicine
      • Durham, North Carolina, United States, 27710
        • Duke University Medical Center (PICU / NICU)
    • Ohio
      • Cincinnati, Ohio, United States, 45229-3039
        • Cincinnati Childrens Hospital Medical Center
      • Cleveland, Ohio, United States, 44106
        • Rainbow Babies and Children's Hospital
      • Cleveland, Ohio, United States, 44313
        • Akron Children's Hospital
    • Oklahoma
      • Oklahoma City, Oklahoma, United States, 73104
        • Board of Regents of the University of Oklahoma
    • Oregon
      • Portland, Oregon, United States, 97201-2701
        • Oregon Health and Science University
    • Rhode Island
      • Providence, Rhode Island, United States, 02903
        • Rhode Island Hospital
    • South Carolina
      • Charleston, South Carolina, United States, 29425
        • Medical University of South Carolina
      • Columbia, South Carolina, United States, 29203
        • University of South Carolina
    • Utah
      • Salt Lake City, Utah, United States, 84108
        • University of Utah Hospitals and Clinics
    • Vermont
      • Burlington, Vermont, United States, 05405
        • University of Vermont Medical Center
    • Virginia
      • Charlottesville, Virginia, United States, 22908-0386
        • University of Virginia Children's Hospital
    • Washington
      • Seattle, Washington, United States, 98105
        • Seattle Children's Hospital
    • West Virginia
      • Morgantown, West Virginia, United States, 26506
        • West Virginia University Hospital
    • Wisconsin
      • Milwaukee, Wisconsin, United States, 53226
        • Medical College of Wisconsin

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

No older than 21 years (Child, Adult)

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

Children (<21 years of age) receiving drugs per standard of care as prescribed by treating caregiver

Description

Inclusion Criteria:

  • 1) Children (< 21 years of age) who are receiving understudied drugs of interest per standard of care as prescribed by their treating caregiver

Exclusion Criteria:

  • 1) Failure to obtain consent/assent (as indicated)
  • 2) Known pregnancy as determined via interview or testing if available.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Cohort
  • Time Perspectives: Prospective

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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

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.
Data will be collected throughout the hospital or outpatient stay up to 90 days

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Composite pharmacodynamic outcomes of understudied drugs in children
Time Frame: Data will be collected throughout the hospital or outpatient stay up to 90 days
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.
Data will be collected throughout the hospital or outpatient stay up to 90 days
Biomarkers associated with understudied drugs in children
Time Frame: Data will be collected throughout the hospital or outpatient stay up to 90 days
The dosing, sampling, and demographic information recorded on the electronic data collection forms 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.
Data will be collected throughout the hospital or outpatient stay up to 90 days

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Daniel Benjamin

Collaborators

Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
The Emmes Company, LLC

Investigators

  • Principal Investigator: Chi Hornik, PharmD, Duke University

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

November 1, 2011

Primary Completion (Actual)

November 1, 2019

Study Completion (Actual)

November 1, 2019

Study Registration Dates

First Submitted

August 17, 2011

First Submitted That Met QC Criteria

September 7, 2011

First Posted (Estimated)

September 9, 2011

Study Record Updates

Last Update Posted (Estimated)

September 6, 2023

Last Update Submitted That Met QC Criteria

August 31, 2023

Last Verified

August 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • Pro00029638
  • IND 113645 (Other Identifier: FDA)
  • IND 114369 (Other Identifier: FDA)
  • IND 114531 (Other Identifier: FDA)
  • IND 118358 (Other Identifier: FDA)
  • HHSN20100006 (Other Grant/Funding Number: NICHD)
  • HHSN27500020 (Other Grant/Funding Number: NICHD)
  • HHSN27500027 (Other Grant/Funding Number: NICHD)
  • HHSN27500043 (Other Grant/Funding Number: NICHD)
  • HHSN27500049 (Other Grant/Funding Number: NICHD)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

IPD Plan Description

Completed study datasets (limited PHI) may be requested from https://pediatrictrials.org/data-sharing-opportunities Study data may be posted to the NICHD Data and Specimen Hub (DASH)

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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