Comment Period Extended to 3/23/2015 for Notice of Proposed Rulemaking (NPRM) for FDAAA 801 and NIH Draft Reporting Policy for NIH-Funded Trials

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

This study is currently recruiting participants. (see Contacts and Locations)
Verified July 2014 by Duke University
The EMMES Corporation
Information provided by (Responsible Party):
Daniel Benjamin, Duke University Medical Center Identifier:
First received: August 17, 2011
Last updated: July 30, 2014
Last verified: July 2014

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).

Condition Intervention
Benzodiazepine Withdrawal
Bipolar Disorder
Autistic Disorder
Influenza Treatment or Prophylaxis
Acute Decompensated Heart Failure
Stable Angina
Life-threatening Fungal Infections
Nosocomial Pneumonia
Community Acquired Pneumonia
Acute Bacterial Exacerbation of Chronic Bronchitis
Complicated Skin and Skin Structure Infections
Uncomplicated Skin and Skin Structure Infections
Chronic Bacterial Prostatitis
Complicated Urinary Tract Infections
Acute Pyelonephritis
Uncomplicated Urinary Tract Infections
Inhalational Anthrax (Post-Exposure)
Drug: Ampicillin

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 ] [ 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.

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 ] [ 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

whole blood

Estimated Enrollment: 2000
Study Start Date: November 2011
Estimated Study Completion Date: August 2015
Estimated Primary Completion Date: August 2015 (Final data collection date for primary outcome measure)
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
Genders Eligible for Study:   Both
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: Michelle Hart    501-364-1418   
Principal Investigator: Laura James, MD         
United States, California
University of California at San Diego Medical Center Recruiting
La Jolla, California, United States, 92093
Contact: Nancy Tang    858-246-0012   
Principal Investigator: Adriana Tremoulet, MD         
Axis Clinical Trials Terminated
Los Angeles, California, United States, 90036
University of California, Los Angeles Medical Center Not yet recruiting
Los Angeles, California, United States, 90095
Contact: Kavita Shankar    310-794-6780   
Principal Investigator: Jaime Deville, MD         
United States, Colorado
The Children's Hospital Colorado Recruiting
Aurora, Colorado, United States, 80045
Contact: Theresa Wilson    720-777-7351   
Principal Investigator: Peter Mourani, MD         
United States, Connecticut
Yale New Haven Children's Hospital Recruiting
New Haven, Connecticut, United States, 06504
Contact: Monica Konstantino    203-688-2320   
Principal Investigator: Matthew Bizzarro, MD         
United States, Delaware
Alfred I. DuPont Hospital for Children Recruiting
Wilmington, Delaware, United States, 19803
Contact: Kimberly Klipner    302-651-4088   
Principal Investigator: Glenn Stryjewski, MD         
United States, District of Columbia
Children's National Medical Center Recruiting
Washington, District of Columbia, United States, 20010
Contact: Elaine Williams    202-884-2245   
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: Kathleen Thoma    904-244-3059   
Principal Investigator: Mobeen Rathore, MD         
United States, Illinois
Childrens Memorial Hospital Recruiting
Chicago, Illinois, United States, 60614
Contact: Carol Nielson    312-227-8272   
Principal Investigator: Ram Yogev, MD         
United States, Indiana
Riley Hosptial for Children at Indiana University Recruiting
Indianapolis, Indiana, United States, 46202
Contact: Leslie Wilson    317-274-8255   
Principal Investigator: Brenda Poindexter, MD, MS         
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
Kosair Children's Hospital Recruiting
Louisville, Kentucky, United States, 40202
Contact: Tressa C Bratton    502-629-5604   
Principal Investigator: Janice Sullivan, MD, FAAP, FCCM, CPI, CIP         
United States, Maryland
University of Maryland Recruiting
Baltimore, Maryland, United States, 21201
Contact: Donna Cannonier    410-328-2438   
Principal Investigator: Susan Mendley, MD         
United States, Michigan
Children's Hospital of Michigan Recruiting
Detroit, Michigan, United States, 48201
Contact: Chantel Karmo    248-737-5939   
Principal Investigator: Edward Dabrowski, MD         
United States, Missouri
Children's Mercy Hospitals and Clinics Active, not recruiting
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 Recruiting
Durham, North Carolina, United States, 27710
Contact: Samantha Wrenn    919-681-2554   
Contact: Christie Milleson    919-681-2401   
Principal Investigator: Kevin Watt, MD         
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         
Case Western University Hospitals of Cleveland Recruiting
Cleveland, Ohio, United States, 44106
Contact: Catherine TAsi    216-844-3312   
Contact: Sue Bergant   
Principal Investigator: David Speicher, MD         
Akron Children's Hospital Recruiting
Cleveland, Ohio, United States, 44313
Contact: Eloise Lemon, RN    330-543-3193   
Principal Investigator: Michael Reed, PharmD         
United States, Oregon
Oregon Health and Science University Recruiting
Portland, Oregon, United States, 97201-2701
Contact: Kira Clark    503-494-3044   
Principal Investigator: Amira Al-Uzri, MD         
United States, South Carolina
Medical University of South Carolina Recruiting
Charleston, South Carolina, United States, 29425
Contact: Patricia Infinger    843-792-7857   
Principal Investigator: Andrew Atz, MD         
United States, Utah
University of Utah Hospitals and Clinics Recruiting
Salt Lake City, Utah, United States, 84108
Contact: Tiffanie Hales    801-213-3367   
Contact: Melissa Bolton    801-213-3909   
Principal Investigator: Michael Spigarelli, MD         
United States, Virginia
University of Virginia Children's Hospital Active, not recruiting
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   
Principal Investigator: Joseph Flynn, MD         
United States, Wisconsin
Medical College of Wisconsin Not yet recruiting
Milwaukee, Wisconsin, United States, 53226
Contact: Trish Barribeau    414-955-2815   
Principal Investigator: Beth Drolet, MD         
Canada, Manitoba
Manitoba Institute of Child Health Not yet 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 Not yet recruiting
Ottawa, Ontario, Canada, K1H 8L1
Contact: Barbara Murchison    1-613-737-7600 ext 4133   
Principal Investigator: Thierry Lacaze, MD         
The Hospital for Sick Children Not yet recruiting
Toronto, Ontario, Canada, M5G 1X8
Contact: Yaron Finkelstein, MD    1-416-813-5317   
Principal Investigator: Yaron Finkelstein, MD         
Canada, Quebec
Hospital Sainte-Justine Not yet recruiting
Montreal, Quebec, Canada, T3T 1C5
Contact: Christine Massicotte    1-514-345-4931 ext 3209   
Contact: Catherine Litalien, MD    1-514-345-4931 ext 2571   
Principal Investigator: Catherine Litalien, MD         
Hadassah University Hospital Not yet recruiting
Ein-Kerem, Jerusalem, Israel, 91120
Contact: Dan Engelhard, MD    011-972-2-6778960   
Principal Investigator: Dan Engelhard, MD         
Assaf Harofeh Medical Center Not yet 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 Not yet recruiting
Petah Tikva, Israel, 49202
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 Not yet recruiting
Southampton, Hampshire, United Kingdom, SO16 6YD
Contact: Macintosh Iain, MD    011-44-0-23-8120-8328   
Contact: Filipa Martins    011-44-0-23-8120-5234   
Principal Investigator: Iain Macintosh, MD         
Alder Hey Children's Hospital Not yet recruiting
Liverpool, Merseyside, United Kingdom, L12 2AP
Contact: Charlie Orton    011-44-151-252-5570   
Contact: Dan Hawcutt, MD    011-44-1512284811 ext 2441   
Principal Investigator: Dan Hawcutt, MD         
University of Bristol Not yet recruiting
Bristol, United Kingdom, BS8 1TH
Contact: Adam Finn, MD    011-44-0-117-34-20172   
Principal Investigator: Adam Finn, MD         
Sponsors and Collaborators
Daniel Benjamin
The EMMES Corporation
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, IND 114369, IND 114531, IND 114892, IND 115226, IND 118329, IND 118358
Study First Received: August 17, 2011
Last Updated: July 30, 2014
Health Authority: United States: Institutional Review Board
United States: Food and Drug Administration

Keywords provided by Duke University:
opioid addiction
cyanide poisoning
cardiac arrest
cardiac arrhythmia
Rocky Mountain Spotted Fever
Q Fever
Heterozygous Familial Hypercholesterolemia (HeFH)
Primary Prevention of Coronary Events
Acute pulmonary edema

Additional relevant MeSH terms:
Autistic Disorder
Bipolar Disorder
Communicable Diseases
Heart Failure
Urinary Tract Infections
Affective Disorders, Psychotic
Cardiovascular Diseases
Child Development Disorders, Pervasive
Heart Diseases
Kidney Diseases
Lipid Metabolism Disorders
Lung Diseases
Mental Disorders
Mental Disorders Diagnosed in Childhood
Metabolic Diseases
Mood Disorders
Nephritis, Interstitial
Pathologic Processes
Pyelitis processed this record on March 01, 2015