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Pediatric Trials Network

Pediatric Trials Network. March 27, 2014. A project of the Best Pharmaceuticals for Children Act. Why are studies in children difficult?. Limited number of patients with the disease No “healthy child/baby volunteer” Low rates of parental informed consent Perceived study risks

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Pediatric Trials Network

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  1. Pediatric Trials Network March 27, 2014 A project of the Best Pharmaceuticals for Children Act

  2. Why are studies in children difficult? • Limited number of patients with the disease • No “healthy child/baby volunteer” • Low rates of parental informed consent • Perceived study risks • Limited blood volume and timed sampling • Sick population – increases variability • Lack of clinical pharmacology expertise • Lack of pediatric PK/PD modeling expertise

  3. How Did The PTN Start? “Create an infrastructure for investigators to conduct trials that improve pediatric labeling and child health.” • Sponsored by the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) • Network for studying drug product formulation, age-appropriate drug dosing, efficacy, safety, and device validation • Success: Completed trials that improve dosing, safety information, labeling, and ultimately child health

  4. Pediatric Trials Network • PI: Benjamin • Since 2010, ~25 ongoing projects, 12 clinical trials • Phase 1 and 2 studies • All therapeutic areas • 5 CSRs submitted to FDA • 4-6 potential label changes • Training of fellows/junior faculty

  5. PTN Structure

  6. PTN Administrative Core

  7. PTN Fellows

  8. Pediatric Trials Network

  9. PTN Site Participation • 255 investigators at 138 sites expressed interest in participating • Anticipate ~60 sites actively enrolling in trials conducted in 2014 • Growth of the “rapid start network”, a pediatric clinical trial consortium affiliated with PTN to a total of approximately 100 sites

  10. PTN Update • Metronidazole: 3 sites, 24 premature infants at risk for NEC. Protocol chair: Michael Cohen-Wolkowiez. Enrollment and analysis complete, CSR submitted to FDA, published in PIDJ • Acyclovir: 3 sites, 32 preterm and term infants with suspected HSV. Protocol chair: Brian Smith. Enrollment and analysis complete, CSR submitted to FDA, published in PIDJ • TAPE weight estimate device: 3 sites, 625 children. Protocol chair: Sue Rahman, Children’s Mercy Hospital, Kansas City, MO. Enrollment and analysis complete. CSR submitted to FDA, published Ann of Emer Med • Hydroxyurea: 6 sites, 40 children. Protocol chair: Kathleen Neville, Children’s Mercy Hospital, Kansas City, MO. Enrollment and analysis complete. CSR submitted to FDA • Opportunistic (POPS I and POPD II):36sites, 1200 children. Protocol chair: Michael Cohen-Wolkowiez. Enrollment ongoing (>1000 enrolled to date) • Lisinopril PK: 8 sites, 24 children with kidney transplants and HTN. Protocol chair: Howard Trachtman, NYU Langone Medical Center. Enrollment complete. Analysis ongoing

  11. PTN Update • Midazolam meta-analysis: Support change of current midazolam labeling to include the treatment of seizures in children ages 2 years and older using existing PK and safety data. Protocol chair: Brian Smith. Analysis in progress. • Ampicillin meta-analysis: PK analysis in combination with 2 retrospective cohort studies of infants with sepsis and/or meningitis. 1 site, 64 infants. Protocol chair: Michael Cohen-Wolkowiez. Data collection and analysis complete, CSR submitted to FDA. PAS poster. • Obesity informatics: Literature search to create a database with published PK studies for the NIH Priority Drug List relevant to pediatric obesity. Protocol chair: Kevin Watt. Literature review complete. PAS poster. • Staph microtrials: 9 sites, 96 infants, 3 anti-staph drugs. Protocol chair: Matt Laughon, UNC. Enrollment ongoing. • Sildenafil: 5 sites, up to 24 infants with pulmonary arterial hypertension or lung disease. Protocol chair: Matt Laughon, UNC. Enrollment ongoing. • Clindamycin obesity: 6 sites, up to 32 children. Safety/PK study of multiple-dose IV and oral clindamycin in obese children. Protocol chair: Janice Sullivan, University of Louisville, KY. Enrollment ongoing. Federated IRB.

  12. PTN Update • Methadone PICU: 4 sites, up to 36 patients. Protocol chair: Kevin Watt. Enrollment ongoing. • Diuretics in NICU: retrospective review: 2 sites, 700 pts. Protocol Chair: Matt Laughon, UNC. Data collection ongoing. • Piperacillin-tazobactam, clindamycin, metronidazole, ampicillin safety in infants with complicated intraabdominal infections (SCAMP): 50 sites, 374 subjects, Protocol Chair: Michael Cohen-Wolkowiez. Site start up • Pantoprazole obesity: 3 sites and 60 children. Study to determine effect of obesity on the PK/PD of pantoprazole in children. Protocol chair: Greg Kearns, Children’s Mercy Hospital, Kansas City, MO. Site start up. • Pediatrix meta-analysis of safety of 11 drugs: Retrospective data analysis; database to include >300 sites and >800,000 infants and data from FDA. Protocol chair: Brian Smith. Data analysis. • Fluconazole safety meta-analysis: 33 sites, 361 infants enrolled in an earlier RCT. Protocol chair: Brian Smith. Data analysis.

  13. PTN Resources • How to get involved: https://pediatrictrials.org/how-to-get-involved • How to develop a concept sheet: https://pediatrictrials.org/how-to-get-involved/for-healthcare-professionals

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