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

This study is currently recruiting participants. (see Contacts and Locations)
Verified March 2017 by Duke University
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
The EMMES Corporation
OpAns, LLC
Information provided by (Responsible Party):
Daniel Benjamin, Duke University Medical Center Identifier:
First received: August 17, 2011
Last updated: March 21, 2017
Last verified: March 2017
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 Intervention
Adrenal Insufficiency
Airway Swelling
Autistic Disorder
Bacterial Meningitis
Bacterial Septicemia
Bipolar Disorder
Bone and Joint Infections
Brain Swelling
Central Nervous System Infections
Cytomegalovirus Retinitis
Early-onset Schizophrenia Spectrum Disorders
General Anesthesia
Gynecologic Infections
Herpes Simplex Virus
Infantile Hemangioma
Inflammatory Conditions
Intra-abdominal Infections
Lower Respiratory Tract Infections
Skeletal Muscle Spasms
Skin and Skin-structure Infections
Stable Angina
Treatment-resistant Schizophrenia
Urinary Tract Infection
Drug: The POPS study is collecting PK data on children prescribed the following drugs of interest per standard of care:

Study Type: Observational
Study Design: Observational Model: Cohort
Time Perspective: Prospective
Official Title: Pharmacokinetics of Understudied Drugs Administered to Children Per Standard of Care

Resource links provided by NLM:

Further study details as provided by Duke University:

Primary Outcome Measures:
  • 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:
  • 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.

  • 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

Estimated Enrollment: 10000
Study Start Date: November 2011
Estimated Study Completion Date: February 2018
Estimated Primary Completion Date: February 2018 (Final data collection date for primary outcome measure)
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
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.


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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT01431326

Contact: Chiara Melloni, MD 919-668-8646
Contact: Barrie L Harper, MT (ASCP) 919-668-8291

  Hide Study Locations
United States, Arkansas
Arkansas Children's Hospital Recruiting
Little Rock, Arkansas, United States, 72202
Contact: Lee Howard    501-364-6990   
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   
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   
Contact: Jendar Deschenes   
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   
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   
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   
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   
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   
Contact: Benjamin Traisman   
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   
Principal Investigator: Sokol Gregory, MD,         
United States, Kansas
Wesely Medical Center Recruiting
Wichita, Kansas, United States, 67214
Contact: Paula Delmore    316-962-8580   
Principal Investigator: Paula Delmore, MSN         
United States, Kentucky
Norton Children's Hospital Recruiting
Louisville, Kentucky, United States, 40202
Contact: Jackie Perry    502-629-5123   
Contact: Michelle Wiseheart    502-629-7595   
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   
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   
Contact: Christie Milleson    919-681-2401   
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   
Contact: Theresa Mottes    513-803-3296   
Principal Investigator: Stuart Goldstein, MD         
Rainbow Babies and Children's Hospital Recruiting
Cleveland, Ohio, United States, 44106
Contact: Eileen Goldblatt    216-844-5112   
Contact: Eloise Lemon    216-844-3339   
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   
Contact: Connie Swanson    503-494-2797   
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   
Contact: Patricia Infinger    832-792-7857   
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   
Contact: Hina Yazdani   
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   
Contact: Kathryn Nickel    206-884-1265   
Principal Investigator: Joseph Flynn, MD         
United States, Wisconsin
Medical College of Wisconsin Recruiting
Milwaukee, Wisconsin, United States, 53226
Contact: Katherine Mueller    414-955-2846   
Contact: Angela Beltrame    414-955-2847   
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   
Contact: Geert T'Jong, MD    1-204-789-3206   
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   
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   
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   
Principal Investigator: Julie Autmizguine, MD         
Assaf Harofeh Medical Center Recruiting
Zerifin, Tel Aviv, Israel, 70300
Contact: Matitiahu Berkovitch, MD    011-972-8-977-9152   
Contact: Elkana Kohn   
Principal Investigator: Matitiahu Berkovitch, MD         
Schneider Children's Medical Center of Israel Recruiting
Petah Tikva, Israel, 49202
Contact: Nava Mattiyahu    011-972-3-9253661   
Contact: Yehezkel (Hezi) Waisman, MD    011-972-3-925-3661   
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   
Contact: Kathy Liaw    1-65-6394-8939   
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   
Contact: Iain Macintosh, MD    011-44-0-23-8120-8328   
Principal Investigator: Iain Macintosh, MD         
Alder Hey Children's Hospital Recruiting
Liverpool, Merseyside, United Kingdom, L12 2AP
Contact: Laura Walsh    011-4401512525570 ext 3552   
Contact: Dan Hawcutt, MD    011-44-151-228-4811 ext 2441   
Principal Investigator: Dan Hawcutt, MD         
University of Bristol Not yet recruiting
Bristol, United Kingdom, BS8 1TH
Contact: Clare Harrison   
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

Additional Information:

Responsible Party: Daniel Benjamin, Professor of Pediatrics, Duke University Medical Center 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 )
Study First Received: August 17, 2011
Last Updated: March 21, 2017
Individual Participant Data  
Plan to Share IPD: No
Plan Description: Completed study datasets (limited PHI) may be requested from

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

Keywords provided by Duke University:
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
Meningitis, Bacterial
Hemangioma, Capillary
Port-Wine Stain
Toxemia processed this record on April 28, 2017