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

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ClinicalTrials.gov Identifier: NCT01431326
Recruitment Status : Recruiting
First Posted : September 9, 2011
Last Update Posted : March 22, 2017
Information provided by (Responsible Party):

Study Description
Brief Summary:
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 3000 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).

Condition or disease Intervention/treatment
Adenovirus Adrenal Insufficiency Airway Swelling Anesthesia Anxiety Anxiolysis Autism Autistic Disorder Bacterial Meningitis Bacterial Septicemia Benzodiazepine Bipolar Disorder Bone and Joint Infections Brain Swelling Central Nervous System Infections Convulsions Cytomegalovirus Retinitis Early-onset Schizophrenia Spectrum Disorders Edema Epilepsy General Anesthesia Gynecologic Infections Headaches Herpes Simplex Virus Hypertension Infantile Hemangioma Infection Inflammation Inflammatory Conditions Influenza Intra-abdominal Infections Lower Respiratory Tract Infections Migraines Pain Pneumonia Prophylaxis Schizophrenia Sedation Seizures Skeletal Muscle Spasms Skin and Skin-structure Infections Stable Angina Thromboprophylaxis Thrombosis Treatment-resistant Schizophrenia Urinary Tract Infection Withdrawal Drug: The POPS study is collecting PK data on children prescribed the following drugs of interest per standard of care:

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 Design

Study Type : Observational
Estimated Enrollment : 10000 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Pharmacokinetics of Understudied Drugs Administered to Children Per Standard of Care
Study Start Date : November 2011
Estimated Primary Completion Date : February 2018
Estimated Study Completion Date : February 2018

Groups and Cohorts

Intervention Details:
    Drug: The POPS study is collecting PK data on children prescribed the following drugs of interest per standard of care:
    Other Names:
    • aripiprazole
    • ceftazidime
    • cidofovir
    • ciprofloxacin
    • dexamethasone
    • diazepam
    • levetiracetam
    • meropenem
    • methylprednisolone
    • midazolam
    • nicardipine
    • olanzapine
    • oseltamivir
    • oxycarbazepine
    • phosphenytoin
    • quetiapine
    • risperidone
    • timolol
    • topiramate
    • valproic acid
    • tobramycin
    • alfentanil
    • clozapine
    • fosphenytoin
    • haloperidol
    • heparin (low molecular weight)
    • hydromorphone
    • lurasidone
    • molindone
    • morphine
    • pentobarbital
    • propofol
    • warfarin (oral)
    • ziprasidone

Outcome Measures

Primary Outcome Measures :
  1. 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.

Secondary Outcome Measures :
  1. 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.

  2. 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 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
whole blood

Eligibility Criteria

Information from the National Library of Medicine

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Ages Eligible for Study:   up to 21 Years   (Child, Adult)
Sexes Eligible for Study:   All
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

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.
Contacts and Locations

Information from the National Library of Medicine

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): NCT01431326

Contact: Chiara Melloni, MD 919-668-8646 chiara.melloni@dm.duke.edu
Contact: Barrie L Harper, MT (ASCP) 919-668-8291 barrie.harper@duke.edu

  Hide Study Locations
United States, Arkansas
Arkansas Children's Hospital Recruiting
Little Rock, Arkansas, United States, 72202
Contact: Lee Howard    501-364-6990    howardleee@uams.edu   
Principal Investigator: Laura James, MD         
United States, California
University of California at San Diego Medical Center Active, not recruiting
La Jolla, California, United States, 92093
Axis Clinical Trials Terminated
Los Angeles, California, United States, 90036
University of California, Los Angeles Medical Center Recruiting
Los Angeles, California, United States, 90095
Contact: Elizabeth Lang    310-206-2439    elang@mednet.ucla.edu   
Principal Investigator: Jaime Deville, MD         
United States, Colorado
The Children's Hospital Colorado Recruiting
Aurora, Colorado, United States, 80045
Contact: Matthew Steinbess    720-777-3019    matthew.steinbess@childrenscolorado.org   
Contact: Jendar Deschenes       jendar.deschenes@childrenscolorado.org   
Principal Investigator: Peter Mourani, MD         
United States, Connecticut
Yale New Haven Children's Hospital Recruiting
New Haven, Connecticut, United States, 06504
Contact: Christine Mulvey    203-688-2320    christine.mulvey@yale.edu   
Principal Investigator: Matthew Bizzarro, MD         
United States, Delaware
Alfred I. DuPont Hospital for Children Recruiting
Wilmington, Delaware, United States, 19803
Contact: Ramany John    302-651-7521    ramany.john@nemours.org   
Principal Investigator: Marisa Meyer, MD         
United States, District of Columbia
Children's National Medical Center Recruiting
Washington, District of Columbia, United States, 20010
Contact: Elaine Williams    202-476-2245    efwillia@cnmc.org   
Principal Investigator: John van den Anker, MD, PhD, FAAP, FCP         
United States, Florida
University of Florida Jacksonville Shands Medical Center Recruiting
Jacksonville, Florida, United States, 32209
Contact: Saniyyah Mahmoudi    904-244-5031    saniyyah.mahmoudi@jax.ufl.edu   
Principal Investigator: Mobeen Rathore, MD         
United States, Illinois
Lurie Children's Hospital of Chicago Recruiting
Chicago, Illinois, United States, 60614
Contact: Laura Fearn    312-227-6285    lfearn@luriechildrens.org   
Contact: Benjamin Traisman       btraisman@luriechildrens.org   
Principal Investigator: Ram Yogev, MD         
United States, Indiana
Riley Hospital for Children at Indiana University Recruiting
Indianapolis, Indiana, United States, 46202
Contact: Susan Gunn    317-278-5239    sgunn@iu.edu   
Principal Investigator: Sokol Gregory, MD,         
United States, Kansas
Wesely Medical Center Recruiting
Wichita, Kansas, United States, 67214
Contact: Paula Delmore    316-962-8580    paula.delmore@wesleymc.com   
Principal Investigator: Paula Delmore, MSN         
United States, Kentucky
Norton Children's Hospital Recruiting
Louisville, Kentucky, United States, 40202
Contact: Jackie Perry    502-629-5123    jacqueline.perry@louisville.edu   
Contact: Michelle Wiseheart    502-629-7595    mlpatt02@exchange.louisville.edu   
Principal Investigator: Janice Sullivan, MD, FAAP, FCCM, CPI, CIP         
United States, Maryland
University of Maryland Active, not recruiting
Baltimore, Maryland, United States, 21201
United States, Michigan
Children's Hospital of Michigan Terminated
Detroit, Michigan, United States, 48201
United States, Missouri
Children's Mercy Hospitals and Clinics Terminated
Kansas City, Missouri, United States, 64108
United States, North Carolina
UNC Hospital Neonatal-Perinatal Medicine Recruiting
Chapel Hill, North Carolina, United States, 27599
Contact: Janice Bernhardt    919-843-2373    bernhardt@unc.edu   
Principal Investigator: Matthew Laughon, MD, MPH         
Duke University Medical Center (PICU / NICU) Recruiting
Durham, North Carolina, United States, 27710
Contact: Samantha Wrenn    919-681-2554    samantha.wrenn@duke.edu   
Contact: Christie Milleson    919-681-2401    christie.milleson@duke.edu   
Principal Investigator: Kevin Watt, MD         
Principal Investigator: Hornik Chi, PharmD         
United States, Ohio
Cincinnati Childrens Hospital Medical Center Recruiting
Cincinnati, Ohio, United States, 45229-3039
Contact: Tara Terrell    513-803-3294    Tara.Terrell@cchmc.org   
Contact: Theresa Mottes    513-803-3296    theresa.mottes@cchmc.org   
Principal Investigator: Stuart Goldstein, MD         
Rainbow Babies and Children's Hospital Recruiting
Cleveland, Ohio, United States, 44106
Contact: Eileen Goldblatt    216-844-5112    eileen.goldblatt@uhhospitals.org   
Contact: Eloise Lemon    216-844-3339    eloise.lemon@uhhospitals.org   
Principal Investigator: David Speicher, MD         
Akron Children's Hospital Terminated
Cleveland, Ohio, United States, 44313
United States, Oregon
Oregon Health and Science University Recruiting
Portland, Oregon, United States, 97201-2701
Contact: Kira Clark    503-418-8112    clarki@ohsu.edu   
Contact: Connie Swanson    503-494-2797    swansoco@ohsu.edu   
Principal Investigator: Amira Al-Uzri, MD         
United States, South Carolina
Medical University of South Carolina Recruiting
Charleston, South Carolina, United States, 29425
Contact: Layla Al-Sarraf    843-792-5309    alsarral@musc.edu   
Contact: Patricia Infinger    832-792-7857    infinge@musc.edu   
Principal Investigator: Andrew Atz, MD         
United States, Utah
University of Utah Hospitals and Clinics Recruiting
Salt Lake City, Utah, United States, 84108
Contact: Sharada Dixit, RN    801-587-7525    sharada.dixit@hsc.utah.edu   
Contact: Hina Yazdani       hina.yazdani@hsc.utah.edu   
Principal Investigator: Catherine Sherwin, MD         
United States, Virginia
University of Virginia Children's Hospital Terminated
Charlottesville, Virginia, United States, 22908-0386
United States, Washington
Seattle Children's Hospital Recruiting
Seattle, Washington, United States, 98105
Contact: Megan Kelton-Rehkopf    206-884-1422    megan.keltonr@seattlechildrens.org   
Contact: Kathryn Nickel    206-884-1265    kathryn.nickel@seattlechildrens.org   
Principal Investigator: Joseph Flynn, MD         
United States, Wisconsin
Medical College of Wisconsin Recruiting
Milwaukee, Wisconsin, United States, 53226
Contact: Katherine Mueller    414-955-2846    kamueller@mcw.edu   
Contact: Angela Beltrame    414-955-2847    anbeltrame@mcw.edu   
Principal Investigator: Beth Drolet, MD         
Canada, Manitoba
Manitoba Institute of Child Health Recruiting
Winnipeg, Manitoba, Canada, R3E 3P4
Contact: Jeannine Schellenberg    1-204-975-7716    jschellenberg@mich.ca   
Contact: Geert T'Jong, MD    1-204-789-3206    gtjong@mich.ca   
Principal Investigator: Geert T'Jong, MD         
Canada, Ontario
Children's Hospital of Eastern Ontario Recruiting
Ottawa, Ontario, Canada, K1H 8L1
Contact: Angie Tuttle    613-737-7600 ext 6058    atuttle@cheo.on.ca   
Principal Investigator: Hugh McMillan, MD         
The Hospital for Sick Children Recruiting
Toronto, Ontario, Canada, M5G 1X8
Contact: Maggie Rumantir, MD    1-416-813-7654 ext 309153    maggie.rumantir@sickkids.ca   
Principal Investigator: Yaron Finkelstein, MD         
Canada, Quebec
Hospital Sainte-Justine Recruiting
Montreal, Quebec, Canada, T3T 1C5
Contact: Mariana Dumitrascu    1-514-345-4931 ext 6648    mariana.dumitrascu@recherche-ste-justine.qc.ca   
Principal Investigator: Julie Autmizguine, MD         
Assaf Harofeh Medical Center Recruiting
Zerifin, Tel Aviv, Israel, 70300
Contact: Matitiahu Berkovitch, MD    011-972-8-977-9152    mberkovitch@asaf.health.gov.il   
Contact: Elkana Kohn       elkanak@asaf.health.gov.il   
Principal Investigator: Matitiahu Berkovitch, MD         
Schneider Children's Medical Center of Israel Recruiting
Petah Tikva, Israel, 49202
Contact: Nava Mattiyahu    011-972-3-9253661    navama4@clalit.org.il   
Contact: Yehezkel (Hezi) Waisman, MD    011-972-3-925-3661    waisy@clalit.org.il   
Principal Investigator: Yehezkel (Hezi) Waisman, MD         
KK Women's and Children's Hospital Pte Ltd Recruiting
Singapore, Singapore, 229899
Contact: Jan Hau Lee, MD    1-65-9699-5200    lee.jan.hau@kkh.com.sg   
Contact: Kathy Liaw    1-65-6394-8939    kathy.liaw.cs@kkh.com.sg   
Principal Investigator: Jan Hau Lee, MD         
United Kingdom
Southampton General Hospital Recruiting
Southampton, Hampshire, United Kingdom, SO16 6YD
Contact: Danny Pratt    011-44-0-23-8120-3853    danny.pratt@uhs.nhs.uk   
Contact: Iain Macintosh, MD    011-44-0-23-8120-8328    iain.macintosh@uhs.nhs.uk   
Principal Investigator: Iain Macintosh, MD         
Alder Hey Children's Hospital Recruiting
Liverpool, Merseyside, United Kingdom, L12 2AP
Contact: Laura Walsh    011-4401512525570 ext 3552    laura.walsh@alderhey.nhs.uk   
Contact: Dan Hawcutt, MD    011-44-151-228-4811 ext 2441    d.hawcutt@liverpool.ac.uk   
Principal Investigator: Dan Hawcutt, MD         
University of Bristol Not yet recruiting
Bristol, United Kingdom, BS8 1TH
Contact: Clare Harrison       clare.harrison@uhbristol.nhs.uk   
Principal Investigator: Marion Roderick, MD         
Sponsors and Collaborators
Daniel Benjamin
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
The EMMES Corporation
OpAns, LLC
Principal Investigator: Michael Cohen-Wolkowiez, MD Duke University
Study Chair: Chiara Melloni, MD Duke University
More Information


Responsible Party: Daniel Benjamin, Professor of Pediatrics, Duke University Medical Center
ClinicalTrials.gov Identifier: NCT01431326     History of Changes
Other Study ID Numbers: Pro00029638
IND 113645 ( Other Identifier: FDA )
IND 114369 ( Other Identifier: FDA )
IND 114531 ( Other Identifier: FDA )
IND 114892 ( Other Identifier: FDA )
IND 115226 ( Other Identifier: FDA )
IND 118329 ( 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 )
First Posted: September 9, 2011    Key Record Dates
Last Update Posted: March 22, 2017
Last Verified: March 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Plan Description: Completed study datasets (limited PHI) may be requested from https://pediatrictrials.org/data-sharing-opportunities

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Daniel Benjamin, Duke University Medical Center:
adrenal insufficiency
afterload inducer
autistic disorder
bipolar disorder
herpes simplex virus
infantile hemangioma

Additional relevant MeSH terms:
Communicable Diseases
Migraine Disorders
Bipolar Disorder
Urinary Tract Infections
Respiratory Tract Infections
Adenoviridae Infections
Angina, Stable
Herpes Simplex
Autistic Disorder
Intraabdominal Infections
Adrenal Insufficiency
Cytomegalovirus Retinitis
Hemangioma, Capillary
Port-Wine Stain
Meningitis, Bacterial