1 / 13

Advancing Pediatric Clinical Trials: Challenges and Achievements in the Pediatric Trials Network

The Pediatric Trials Network (PTN) addresses critical challenges in studying drugs for children, such as limited patient populations and the need for informed parental consent. Established by the Eunice Kennedy Shriver National Institute of Child Health and Human Development, the PTN facilitates trials focused on pediatric drug formulations, dosing, efficacy, and safety. Since its inception in 2010, the network has seen considerable success, completing numerous trials and submitting reports to the FDA, thus improving child health outcomes and medication labeling.

randall-hayes
Télécharger la présentation

Advancing Pediatric Clinical Trials: Challenges and Achievements in the Pediatric Trials Network

An Image/Link below is provided (as is) to download presentation Download Policy: Content on the Website is provided to you AS IS for your information and personal use and may not be sold / licensed / shared on other websites without getting consent from its author. Content is provided to you AS IS for your information and personal use only. Download presentation by click this link. While downloading, if for some reason you are not able to download a presentation, the publisher may have deleted the file from their server. During download, if you can't get a presentation, the file might be deleted by the publisher.

E N D

Presentation Transcript

  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

More Related