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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT01431326
Recruitment Status : Recruiting
First Posted : September 9, 2011
Last Update Posted : April 4, 2019
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
The Emmes Company, LLC
Information provided by (Responsible Party):
Daniel Benjamin, Duke University

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 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).

Condition or disease Intervention/treatment
Adenovirus Anesthesia Anxiety Anxiolysis Autism Autistic Disorder Bacterial Meningitis Bacterial Septicemia Benzodiazepine Bipolar Disorder Bone and Joint Infections Central Nervous System Infections Convulsions Cytomegalovirus Retinitis Early-onset Schizophrenia Spectrum Disorders Epilepsy General Anesthesia Gynecologic Infections Herpes Simplex Virus Infantile Hemangioma Infection Inflammation Inflammatory Conditions Intra-abdominal Infections Lower Respiratory Tract Infections Migraines Pain Pneumonia Schizophrenia Sedation Seizures Skeletal Muscle Spasms Skin and Skin-structure Infections Treatment-resistant Schizophrenia Urinary Tract Infections Withdrawal Sepsis Gram-negative Infection Bradycardia Cardiac Arrest Cardiac Arrhythmia Staphylococcal Infections Nosocomial Pneumonia Neuromuscular Blockade Methicillin Resistant Staphylococcus Aureus Endocarditis Neutropenia Headache Fibrinolytic Bleeding Pulmonary Arterial Hypertension CMV Retinitis Hypertension Chronic Kidney Diseases Hyperaldosteronism Hypokalemia Heart Failure Hemophilia Heavy Menstrual Bleeding Insomnia 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.

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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 2020
Estimated Study Completion Date : February 2020

Intervention Details:
  • Drug: The POPS study is collecting PK data on children prescribed the following drugs of interest per standard of care:
    Other Names:
    • cidofovir
    • ciprofloxacin
    • methylprednisolone
    • tobramycin
    • alfentanil
    • clozapine
    • haloperidol
    • lurasidone
    • molindone
    • pentobarbital
    • warfarin (oral)
    • ziprasidone
    • amikacin
    • cefepime
    • nafcillin
    • piperacillin-tazobactam
    • rocuronium
    • vecuronium
    • vancomycin
    • aminocaproic acid
    • bosentan
    • fosfomycin
    • labetalol
    • nifedipine
    • oxycodone
    • sevelamer carbonate
    • spironolactone
    • tranexamic acid
    • zolpidem

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 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.

Biospecimen Retention:   Samples With DNA
whole blood

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.

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 identifier (NCT number): NCT01431326

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Contact: Barrie Harper, MT (ASCP), PMP 919-668-8291
Contact: POP01 StudyMailbox

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United States, Alaska
Alaska Native Medical Center Recruiting
Anchorage, Alaska, United States, 99508
Contact: Amy Swango Wilson    907-729-2987   
Principal Investigator: Rosalyn Singleton         
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: Alleluiah Rutebemberwa    720-777-5380   
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 Terminated
Washington, District of Columbia, United States, 20010
United States, Florida
University of Florida Jacksonville Shands Medical Center Recruiting
Jacksonville, Florida, United States, 32209
Contact: Alexandrea Borges    904-244-2266   
Principal Investigator: Mobeen Rathore, MD         
United States, Hawaii
Kapiolani Womens and Childrens Medical Center Recruiting
Honolulu, Hawaii, United States, 96826
Contact: Venkataraman Balaraman    808-369-1247   
Principal Investigator: Annette Amiotte         
United States, Illinois
Lurie Children's Hospital of Chicago Recruiting
Chicago, Illinois, United States, 60614
Contact: Laura Fearn    312-227-6285   
Contact: Rohit Kalra    312-227-6281   
Principal Investigator: William Muller, MD         
United States, Indiana
Riley Hospital for Children at Indiana University Recruiting
Indianapolis, Indiana, United States, 46202
Contact: Brianne Kost    317-274-4708   
Principal Investigator: Gregory Sokol, MD,         
United States, Kansas
University of Kansas Medical Center Not yet recruiting
Fairway, Kansas, United States, 66205
Contact: Megan Bledsoe    816-960-3007   
Principal Investigator: Pahud Barbara, MD         
Children's Mercy Hospital and Clinics Recruiting
Kansas City, Kansas, United States, 66160
Contact: Megan Bledsoe    816-960-3007   
Principal Investigator: Pahud Barbara, MD         
Wesley 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: Jen Comings    502-629-4698   
Contact: Jackie Perry    502-629-5123   
Principal Investigator: Janice Sullivan, MD, FAAP, FCCM, CPI, CIP         
United States, Louisiana
Tulane University Health Science Center Recruiting
New Orleans, Louisiana, United States, 70112
Contact: Rachel Lee    504-988-1438   
Principal Investigator: Stacy Drury         
Ochsner Baptist Clinical Trials Unit Not yet recruiting
New Orleans, Louisiana, United States, 70115-6969
Contact: Sarah Geiger    504-894-2861   
Principal Investigator: Amanda England, MD         
United States, Maryland
University of Maryland Terminated
Baltimore, Maryland, United States, 21201
United States, Massachusetts
Boston Children's Hospital Not yet recruiting
Boston, Massachusetts, United States, 02115
Contact: Rachel Bernier    857-218-5348   
Principal Investigator: Vivian Nasr, MD         
United States, Michigan
Children's Hospital of Michigan Terminated
Detroit, Michigan, United States, 48201
United States, Mississippi
University of Mississippi Medical Center Recruiting
Jackson, Mississippi, United States, 39216
Contact: Lacy Malloch    601-815-4517   
Principal Investigator: Jospeh Majure         
United States, Missouri
Children's Mercy Hospitals and Clinics Terminated
Kansas City, Missouri, United States, 64108
United States, Montana
University of Montana Recruiting
Missoula, Montana, United States, 59804
Contact: Sara Cox    406-370-7379   
Principal Investigator: Paul Smith         
United States, Nebraska
University of Nebraska Medical Center Recruiting
Omaha, Nebraska, United States, 68198
Contact: Rachel Aikman    402-552-9878   
Principal Investigator: McCulloh Russell, MD         
United States, New Hampshire
Dartmouth-Hitchcock Medical Center Recruiting
Lebanon, New Hampshire, United States, 03756
Contact: J Jarvis    603-653-9064   
Principal Investigator: Paul Palumbo         
United States, New Mexico
University of New Mexico, Health Sciences Center Recruiting
Albuquerque, New Mexico, United States, 87131
Contact: Olivia Nunez    505-272-9898   
Principal Investigator: Hengameh Raissy         
United States, New York
Kravis Children's Hospital at Mt. Sinai Not yet recruiting
New York, New York, United States, 10029
Contact: iris Mandell, MD    212-241-6489   
Principal Investigator: George Ofori-Amanfo, MD         
United States, North Carolina
UNC Hospital Neonatal-Perinatal Medicine Recruiting
Chapel Hill, North Carolina, United States, 27599
Contact: Jennifer Talbert    984-974-7865   
Contact: Matthew Massaro    919-843-2373   
Principal Investigator: Matthew Laughon, MD, MPH         
Duke University Medical Center (PICU / NICU) Recruiting
Durham, North Carolina, United States, 27710
Contact: Melissa Harward    919-668-3910   
Contact: Grace Jefferson    919-668-3873   
Principal Investigator: Hornik Chi, PharmD         
United States, Ohio
Cincinnati Childrens Hospital Medical Center Recruiting
Cincinnati, Ohio, United States, 45229-3039
Contact: Cassie Kirbie    513-803-5332   
Contact: Bradley Gerhardt   
Principal Investigator: Stuart Goldstein, MD         
Rainbow Babies and Children's Hospital Terminated
Cleveland, Ohio, United States, 44106
Akron Children's Hospital Terminated
Cleveland, Ohio, United States, 44313
United States, Oklahoma
Board of Regents of the University of Oklahoma Recruiting
Oklahoma City, Oklahoma, United States, 73104
Contact: Christi Madden    405-271-4407   
Principal Investigator: Christine Allen, MD         
United States, Oregon
Oregon Health and Science University Recruiting
Portland, Oregon, United States, 97201-2701
Contact: Kira Clark    503-418-8112   
Contact: Kimberly Grzesek    503-494-2797   
Principal Investigator: Amira Al-Uzri, MD         
United States, Rhode Island
Rhode Island Hospital Recruiting
Providence, Rhode Island, United States, 02903
Contact: Barbara Bancroft    401-444-2884   
Principal Investigator: Margaret Scheffler         
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         
University of South Carolina Recruiting
Columbia, South Carolina, United States, 29203
Contact: Barbara Thompson    803-434-4986   
Principal Investigator: Lisa Knight         
United States, Tennessee
Monroe Carell Jr. Children's Hospital at Vanderbilt Not yet recruiting
Nashville, Tennessee, United States, 37232-9550
Contact: Theresa Rogers   
Contact: Steven Steele    1-615-322-0299   
Principal Investigator: Jorn-Hendrik Weitkamp, MD         
United States, Utah
University of Utah Hospitals and Clinics Terminated
Salt Lake City, Utah, United States, 84108
United States, Vermont
University of Vermont Medical Center Recruiting
Burlington, Vermont, United States, 05405
Contact: Laurie Chassereau    802-656-9401   
Principal Investigator: Kelly Cowan         
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, West Virginia
West Virginia University Hospital Not yet recruiting
Morgantown, West Virginia, United States, 26506
Contact: Michelle Shaffer    304-293-2081   
Contact: Kaitlin Earle    1-304-293-9313   
Principal Investigator: Lee Pyles         
United States, Wisconsin
Medical College of Wisconsin Recruiting
Milwaukee, Wisconsin, United States, 53226
Contact: Sadaf Shad   
Contact: Mary Lynn Kasch    1-414-266-1785   
Principal Investigator: Nathan Thompson, MD         
Australia, New South Wales
Kids Research Institute Not yet recruiting
Randwick, New South Wales, Australia, 2031
Contact: Indira Mismit    011-02-9845-0364   
Contact: Regienald Gayaman    011-02-9485-1413   
Principal Investigator: Chris Cowell, MD         
Australia, Victoria
Royal Children's Hospital Not yet recruiting
Parkville, Victoria, Australia, 3052
Contact: Shannon Kokoszka    1-613-934-5557   
Principal Investigator: Noel Cranswick         
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: Sara Leradi    613-737-7600 ext 6058   
Principal Investigator: Daniela Pohl, 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: Ron Berant   
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 Terminated
Southampton, Hampshire, United Kingdom, SO16 6YD
Alder Hey Children's Hospital Terminated
Liverpool, Merseyside, United Kingdom, L12 2AP
University of Bristol Withdrawn
Bristol, United Kingdom, BS8 1TH
Sponsors and Collaborators
Daniel Benjamin
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
The Emmes Company, LLC
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Principal Investigator: Michael Cohen-Wolkowiez, MD Duke University
Study Chair: Chiara Melloni, MD Duke University

Additional Information:
Publications of Results:

Other Publications:

Publications automatically indexed to this study by Identifier (NCT Number):
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Responsible Party: Daniel Benjamin, Professor of Pediatrics, Duke University 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 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 )
First Posted: September 9, 2011    Key Record Dates
Last Update Posted: April 4, 2019
Last Verified: April 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Plan Description: Completed study datasets (limited PHI) may be requested from Study data may be posted to the NICHD Data and Specimen Hub (DASH)

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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:
autistic disorder
bipolar disorder
herpes simplex virus
herpes simplex virus (HSV)
infantile hemangioma
lower respiratory tract infection (LRTI)
muscle spasms

Additional relevant MeSH terms:
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Child Development Disorders, Pervasive
Communicable Diseases
Heart Failure
Kidney Diseases
Renal Insufficiency, Chronic
Migraine Disorders
Sleep Initiation and Maintenance Disorders
Bipolar Disorder
Hemophilia A
Heart Arrest
Familial Primary Pulmonary Hypertension
Urinary Tract Infections
Respiratory Tract Infections
Adenoviridae Infections
Herpes Simplex
Autistic Disorder
Staphylococcal Infections