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Principal Investigator: KANECIA ZIMMERMAN, MD
Organization: DUKE UNIVERSITY
Fiscal Year: 2020
Award: $5,000,000
Funding agency: Eunice Kennedy Shriver National Institute of Child Health and Human Development
For the last 25 years the scientific community, including academia, the National Institutes of
Health (NIH), the Food and Drug Administration (FDA) and the pharmaceutical industry have worked to
improve the knowledge of medications used in children. The responsibility for the implementation
and oversight for improving drug development has been delegated primarily to the FDA (for on-patent
drugs) and to the NIH (for off-patent drugs). The Pediatric Trials Network (PTN) has been
established and funded under the Best Pharmaceuticals for Children Act (BPCA) Clinical Program
implemented by the Eunice Kennedy Shriver National Institute of Child Health and Human Development
(NICHD).
Many PTN studies historically capitalize on standard of care (SOC) procedures, such as data and
biological-sample collection from infants and children already receiving drugs of interest (DOIs),
and have produced meaningful pharmacokinetic (PK) data resulting in improved dosing recommendations
in infants and children. These studies do not prescribe or administer drugs to children, but rather
collected samples from children who were already receiving drugs per SOC as prescribed by their
treating provider. In addition, preliminary data obtained through opportunistic studies have served
to design phase 1 through 3 trials in children as well as support applications for extramural
research.
The coronavirus disease 2019 (COVID-19) pandemic is caused by the novel severe acute respiratory
syndrome coronavirus 2 (SARS-CoV-2). The infection may range from the patient having no symptoms to
causing a wide range of symptoms, such as mild cold like symptom to life-threatening pneumonias,
sepsis, and multi-organ system failure. COVID-19 first emerged in December 2019 when a cluster of
patients in Wuhan, China developed severe pneumonias of unknown cause. [Wiersinga et al, JAMA
doi:10.1001/jama2020.12839]. As of August 11, 2020, SARS-CoV-2 has affected more than 200
countries, resulting in 20,124,437 million identified cases with over 737K confirmed deaths
[https://www.cdc.gov/coronavirus/2019-ncov]. In April 2020 at the peak of the COVID-19 outbreak,
there was recognized a new onset of hyperinflammatory symptoms in children who either had active
disease or in children who had been previously exposed to COVID-19 with or without symptoms. This
syndrome was named multisystem inflammatory syndrome in children (MIS-C) by the CDC in May 2020. As
of July 29, 2020, 570 patients have met the criteria for MIS-C in the US
[https://www.cdc.gov/mmwr/volumes/69/wr/mm6932e2.htm]. As the pandemic continues to spread
throughout the US and the world, the scientific community anticipates that the number of children
infected and affected will continue to rise. There must be an organized concerted collaborative
effort between public health, patient care, and research in order for the appropriate prevention,
identification and treatment of children impacted by COVID-19.
Since COVID-19 has ravaged the world with inordinate high infection rates and death, the need
for preventive, research and clinical data have become paramount and top priority for NIH as a
research and funding institution. Furthermore, NIH, academia, clinicians, and others in the
scientific community are concluding that the harmonization of all epidemiology and clinical related
data as well as clinically relevant outcomes vital to identification and treatment of patients
at-risk for COVID-related illnesses, such as the multi-system inflammatory syndrome in children
(MIS-C), are critical to successfully managing the pandemic.
These COVID-19 related studies will leverage the expertise and existing data within the PTN
infrastructure of the BPCA program (BPCA PTN and Data Coordinating Center) to expand the
research on SARS-CoV-2 (COVID-19) in children.
Terms: <0-11 years old><2019 novel coronavirus><2019-nCoV><Academia><Affect><Best Pharmaceuticals for Children Act><Biological><Biological Markers><CDC><COVID><COVID-19><COVID-19 epidemic><COVID-19 pandemic><COVID19><COVID19 epidemic><COVID19 pandemic><Centers for Disease Control><Centers for Disease Control and Prevention><Centers for Disease Control and Prevention (U.S.)><Cessation of life><Child><Child Youth><Childhood><Children (0-21)><China><Clinical><Clinical Data><Clinical Trials Design><CoV disease><Communities><Coronaviridae><Coronavirus><Country><Data><Data Coordinating Center><Data Coordination Center><Death><Diagnosis><Disease><Disease Outbreaks><Disorder><Dose><Drug Industry><Drug Kinetics><Drugs><EXTMR><Effectiveness><Epidemiology><Exposure to><Extramural><Extramural Activities><Failure><Food and Drug Administration><Funding><Future><Infant><Infection><Inflammatory><Institution><Intervention><Intervention Strategies><Knowledge><Legal patent><Life><Mainland China><Medication><NICHD><NIH><Names><National Institute of Child Health and Human Development><National Institute of Children's Health and Human Development><National Institutes of Health><Network Infrastructure><Outbreaks><Outcome><Patents><Patient Care><Patient Care Delivery><Patients><Pharmaceutic Industry><Pharmaceutic Preparations><Pharmaceutical Industry><Pharmaceutical Preparations><Pharmacokinetics><Pneumonia><Population><Prevention><Preventive><Procedures><Provider><Public Health><Recommendation><Reporting><Research><Risk><Risk Factors><SARS-CoV-2><SARS-CoV2><SARS-associated coronavirus 2><SARS-coronavirus-2><SARS-related coronavirus 2><Safety><Sampling><Science><Sepsis><Severe acute respiratory syndrome coronavirus 2><Symptoms><Syndrome><System><Therapeutic><USFDA><United States Centers for Disease Control><United States Centers for Disease Control and Prevention><United States Food and Drug Administration><United States National Institutes of Health><Work><Wuhan coronavirus><bio-markers><biologic marker><biomarker><blood infection><bloodstream infection><children><childrens'><clinical relevance><clinically relevant><corona virus><corona virus disease><corona virus disease 2019><corona virus disease 2019 epidemic><corona virus disease 2019 pandemic><coronavirus disease><coronavirus disease 2019><coronavirus disease 2019 epidemic><coronavirus disease 2019 pandemic><data harmonization><design><designing><disease diagnosis><drug development><drug/agent><epidemiologic><epidemiological><harmonized data><improved><infection rate><interest><interventional strategy><novel><off-patent><pandemic><pandemic disease><pediatric><phase 1 designs><phase I designs><programs><rate of infection><sample collection><specimen collection><standard of care><youngster>