1 / 26

Pediatric Trials Network

Pediatric Trials Network. What Is The Pediatric Trials Network PTN ?. Sponsored by the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD ) T he primary objective of the Pediatric Trials Network:

kyran
Download Presentation

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

  2. What Is The Pediatric Trials Network PTN? • Sponsored by the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) • The primary objective of the Pediatric Trials Network: Create an infrastructure for investigators to conduct trials that improve pediatric labeling and child health. • PTN is studying product formulation, drug dose, efficacy, safety, and device validation • Evidence of success will be completed trials that improve dosing, safety information, labeling, and ultimately child health

  3. Structure of PTN

  4. Pediatric Trials Network (PTN) 2011

  5. Protocol: Metronidazole Protocol Chair: Cohen-Wolkowiez • Protocol Title: Safety and Pharmacokinetics of Multiple Dose Metronidazole in Premature Infants • Objective: Evaluate the safety, PK, and surrogate PD of intravenous metronidazole in premature infants with suspected serious infection • Study Population:16 to 32 participants <32 weeks gestational age with suspected serious infection. Participants will be divided into 2 groups based on postnatal age. • Study Duration: original target 18 months (finished in 12); each participant will participate in the study for up to 15 days: 2-5 days of study drug administration followed by 10 days of adverse events monitoring. • Number of Sites: 3 • October, 2011 Enrollment Complete

  6. Demographic Distribution: These data have not been peer-reviewed

  7. Metronidazole Individual EBE PK Parameter Estimates by Post Natal Age Group: These data have not been peer-reviewed

  8. Concentration vs. Time: These data have not been peer-reviewed

  9. Clearance vs. Post Menstrual Age: These data have not been peer-reviewed

  10. Protocol: Acyclovir Protocol Chair: Smith • Protocol Title: An Open Label Study to Describe the Pharmacokinetics of Acyclovir in Premature Infants • Objective: To evaluate the safety and PK of IV acyclovir in premature infants with suspected systemic infections. • Study Population: 20 Infants < 45 days postnatal age, suspected to have a systemic infection divided into groups by gestational and postnatal age • Study Duration: each infant will be in the study for up to 13 days; goal is to provide final component of PK data for subsequent efficacy trial • Number of Sites: 3 • First Patient Enrolled: September 19, 2011; target March 2012

  11. Protocol: HydroxyureaProtocol Chair: Neville • Protocol Title: PK & Relative Bioavailability of a Liquid Formulation of Hydroxyurea in Pediatric Patients with Sickle Cell Anemia • Objective: relative bioavailabilty study and bioequivalence study with new formulation • Study Population: 40 children ages 2-17 with sickle cell anemia or sickle beta-zero thalassemia; two-armed study with older children (bioequivalence) enrolled first • Study duration: a subset of patients in each age cohort will receive single dose and a subset will receive multiple doses • Number of Sites: Six • First Patient: December, 2011

  12. Protocol: POPS Pediatric Opportunistic PK Study • Protocol Title: Pharmacokinetics of Understudied Drugs Administered to Children per Standard of Care • Objectives: • Evaluate the PK of understudied drugs currently being administered to children. • Study Population: 500 children (birth-20 years) who are receiving understudied drugs of interest per standard of care as prescribed by their treating caregiver • Study Duration: each child will participate in the study for up to 90 days per drug; study conduct for 3 years • Number of Sites: 15 • First Patient Enrolled: November, 2011

  13. Protocol: LisinoprilProtocol Chair: Trachtman • Protocol Title: Safety and Pharmacokinetics of Lisinopril in Pediatric Kidney Transplant Recipients • Objective: initial description of the PK-PD and safety of lisinopril following transplantation • Study population: 24 children ages 2-18 with kidney transplant and stable allograft function • Study participation: Up to 51 days; enrolled children will receive multiple doses with multiple assessments for potential endpoints for subsequent efficacy trial • Number of Sites: 8 • Target to Enroll First Patient: January 2012

  14. Protocol: TAPEProtocol Chair: Rahman • Protocol Title: Taking the Guesswork out of Pediatric Weight Estimation (TAPE): Validation of the Mercy TAPE • Objective: device trial to provide more accurate, rapid estimation of weight in the acute care setting—e.g., use in emergency setting or resource poor countries for quick dosing calculations • Study population: 625 children 2months to 16 years old enrolled into 17 strata • Protocol Final and target first enrollment January 2012

  15. Other Task Orders • Midazolam • Analysis of previously collected data • Provide supplemental data to support of the current prescription labeling to include the treatment of seizures • Ampicillin • Original written request, PK study and efficacy study in infants • PPRU (Pediatric Pharmacology Research Unit) collected samples • Obesity • Analysis to provide preliminary data and hand held application for dosing in obese children

  16. Lessons Learned Main Contract Timelines • Meropenem RFP NIH-NICHD-2005-18 released August 2005, submitted October 2005 • Signed September 2007 (24 months) • Protocol in written as part of application; IND granted March 2008 (31 months) • Site contracts, IRB, investigators meeting • First patient June 2008 • 200 infants; last infant enrolled September 2009

  17. Lessons Learned Main Contract Timelines • Meropenem RFP release to signature 24 months • IND 31 months • First patient 34 months • Last infant 48 months • Clinical Study Report 60 months from RFP release • Pediatric Trials Network RFP 3/2010, signature 6 months • IND 7 months • First patient 9 months • Last patient 18 months • Clinical study report 22 months (anticipated)

  18. Differences in timelines • IRB vs. Contracts • Single entity of PTN mitigates • Risk to NIH • Risk to investigators • Contracts with sites • Opportunistic study • Contracts with vendors

  19. Comparison of output and efficiency Legacy Trials vs PTN Detailed Comparison • Legacy Trials Timeline 8 years • PTN 12–18 months • Similar number of patients • PTN more INDs • By 24 months: studies enrolling more patients, in more sites, under more INDs

  20. Pricing differential • Per patient pricing reduced 30–50% • Faculty (thought leadership) • Winning a grant, conduct of the grant • Junior faculty • K23 awardees and young investigators • Operations (staff) efficiency

  21. Pediatric Trials Network (PTN) 2012 tentative

  22. How Do I Participate in the PTN? • The POPS study: • children interact with the health care system (e.g., admitted to the PICU or seen in the ER) • on a prioritized off-patent therapeutic that has insufficient dosing information in their clinical stratum age-based: e.g., premature neonates acuity based: e.g., resuscitation meds clinical-based: e.g., ethnicity, obesity • ask for consent to take blood at pre-specified times based on dosing interval (Q4 vs. Q24)

  23. PTN and POPS Continued • 15 or more therapeutics bundled into one protocol • Samples stored locally and sent in batch • Flexibility to add molecules • Provide preliminary and supportive data for subsequent trials • Compare to epi-data • Metronidazole example • Provide a testing ground for sites—enrollment • Facilitate contracts and infrastructure—enrollment in between more traditional trials

  24. Contacting the PTN for the POPS trial • POPS Protocol Chair: Micky Cohen-Wolkowiez michael.cohenwolkowiez@duke.edu • POPS project lead: Barrie Harper barrie.harper@duke.edu • www.pediatrictrials.org

  25. Limits of the mechanism Opportunistic PK and PK-PD Safety Efficacy

More Related