The Pediatric Trials Network (PTN) is a consortium of clinical research sites located around the United States that are cooperating in the design and conduct of clinical trials to improve health care for young patients. The network is sponsored by the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD).
The work of the PTN addresses a critical lack of information regarding the impact of therapeutic agents on infants and children. Developing organs and changes in metabolism throughout infancy and childhood affect how drugs are processed by immature or maturing bodies; thus, age-dependent adjustments in doses are required to ensure that such therapies are used safely and effectively. Unfortunately, only a small percentage of drugs and devices approved by the Food and Drug Administration (FDA) have actually been studied in children and are labeled for pediatric use. Pediatricians, consequently, are often forced to prescribe medical therapies "off-label," or according to their best guess based on adult studies.
To fill this knowledge gap, the PTN is studying the formulation, dosing, efficacy, and safety of drugs, as well as the development of medical devices, used in pediatric patients. In keeping with the goals of the Best Pharmaceuticals for Children Act (BPCA), data collected from PTN trials are helping regulators to revise FDA labels for safer and more effective use in infants and children.
A map of sites currently participating in PTN trials may be viewed on the network's website [1].
Maps, Directions, and Place Reviews
Background
Pediatric clinical research faces unique challenges: low study consent rates among parents of sick children, limited blood volume available for the conduct of pharmacokinetic studies, and a relative lack of pediatric analytical expertise in pharmacokinetics and pharmacodynamics, to name a few. Because of such obstacles, before 1998, pharmaceutical companies were not required by the government to test their drugs in infants and children, even if the drugs were commonly given to those populations.
The Food and Drug Administration Modernization Act (1997) and BPCA (2002, amended in 2007 and renewed in 2012) offer financial incentives in the form of patent extensions for companies that voluntarily test their drugs in pediatric patients. BPCA also provides a mechanism by which off-patent therapeutics might be studied through a collaboration between the FDA and National Institutes of Health. The NICHD is responsible for funding these studies from its annual budget.
Since the BPCA was first enacted, the NICHD has awarded numerous projects to organizations and institutions for the purpose of gathering information to improve pediatric drug labeling. One of these projects is the PTN, which is creating a scientific, technical, and administrative infrastructure that, in strategic partnership with the NICHD, is studying critical drugs and diagnostic devices in children to improve labeling for pediatric use.
Clinical Trials Kansas City Video
Current Projects
The PTN is conducting a number of pediatric trials spanning several therapeutic areas, including cardiovascular diseases, cancer, infectious diseases, gastroenterology, respiratory diseases, neonatology, and medical devices. Below is a summary of PTN projects as of October 2013.
Published Results
- Gonzalez D, Melloni C, Yogev R, et al. (October 2014). "Use of opportunistic clinical data and a population pharmacokinetic model to support dosing of clindamycin for premature infants to adolescents". Clinical Pharmacology and Therapeutics. 96 (4): 429-37. PMC 4169790 . PMID 24949994. doi:10.1038/clpt.2014.134.
- Tremoulet A, Le J, Poindexter B, et al. (June 2014). "Characterization of the population pharmacokinetics of ampicillin in neonates using an opportunistic study design". Antimicrobial Agents and Chemotherapy. 58 (6): 3013-20. PMC 4068432 . PMID 24614374. doi:10.1128/AAC.02374-13.
- Benjamin DK, Hudak ML, Duara S, et al. (May 2014). "Effect of fluconazole prophylaxis on candidiasis and mortality in premature infants: a randomized clinical trial". JAMA. 311 (17): 1742-9. PMC 4110724 . PMID 24794367. doi:10.1001/jama.2014.2624.
- Sampson MR, Bloom BT, Lenfestey RW, et al. (January 2014). "Population pharmacokinetics of intravenous acyclovir in preterm and term infants". The Pediatric Infectious Disease Journal. 33 (1): 42-9. PMC 3904301 . PMID 24346595. doi:10.1097/01.inf.0000435509.75114.3d.
- Abdel-Rahman SM, Paul IM, James LP, Lewandowski A (October 2013). "Evaluation of the Mercy TAPE: performance against the standard for pediatric weight estimation". Annals of Emergency Medicine. 62 (4): 332-339.e6. PMC 4039210 . PMID 23602655. doi:10.1016/j.annemergmed.2013.02.021.
- Cohen-Wolkowiez M, Sampson M, Bloom BT, et al. (September 2013). "Determining population and developmental pharmacokinetics of metronidazole using plasma and dried blood spot samples from premature infants". The Pediatric Infectious Disease Journal. 32 (9): 956-61. PMC 3769518 . PMID 23587979. doi:10.1097/INF.0b013e3182947cf8.
- Hornik CP, Herring AH, Benjamin DK, et al. (July 2013). "Adverse events associated with meropenem versus imipenem/cilastatin therapy in a large retrospective cohort of hospitalized infants". The Pediatric Infectious Disease Journal. 32 (7): 748-53. PMC 3708263 . PMID 23838776. doi:10.1097/INF.0b013e31828be70b.
- Cohen-Wolkowiez M, Poindexter B, Bidegain M, et al. (December 2012). "Safety and effectiveness of meropenem in infants with suspected or complicated intra-abdominal infections". Clinical Infectious Diseases. 55 (11): 1495-502. PMC 3491861 . PMID 22955430. doi:10.1093/cid/cis758.
- Smith PB, Cohen-Wolkowiez M, Castro LM, et al. (October 2011). "Population pharmacokinetics of meropenem in plasma and cerebrospinal fluid of infants with suspected or complicated intra-abdominal infections". The Pediatric Infectious Disease Journal. 30 (10): 844-9. PMC 3173561 . PMID 21829139. doi:10.1097/INF.0b013e31822e8b0b.
- Laughon MM, Benjamin DK, Capparelli EV, et al. (September 2011). "Innovative clinical trial design for pediatric therapeutics". Expert Review of Clinical Pharmacology. 4 (5): 643-52. PMC 3184526 . PMID 21980319. doi:10.1586/ecp.11.43.
PTN Core Leadership
- PTN Program Management and Clinical Operations Core; Mentorship Core: Daniel K. Benjamin Jr., Duke Clinical Research Institute [21], Durham, NC
- Clinical Pharmacology Core: Gregory L. Kearns, Children's Mercy Hospital [22], Kansas City, MO
- Pharmacometrics Core: Edmund Capparelli, University of California-San Diego [23], San Diego, CA
- Safety and Ethics Core: Ross McKinney, Duke University Medical Center [24], Durham, NC
- Devices Core: Andre Muelenaer, Virginia Tech Carilion School of Medicine [25], Roanoke, VA
PTN Administrative Core Committee
- Jeffrey Barrett, Children's Hospital of Philadelphia [26], Philadelphia, PA
- Michael Cohen-Wolkowiez, Duke Clinical Research Institute [27], Durham, NC
- Matthew Laughon, University of North Carolina at Chapel Hill [28], Chapel Hill, NC
- T. Michael O'Shea, Wake Forest Baptist Medical Center [29], Winston-Salem, NC
- Ian M. Paul, Penn State College of Medicine [30], Hershey, PA
- P. Brian Smith, Duke Clinical Research Institute [31], Durham, NC
- John van den Anker, George Washington University School of Medicine and Health [32], Washington, DC
- Kelly Wade, Children's Hospital of Philadelphia [33], Philadelphia, PA
- The Eunice Kennedy Shriver National Institute of Child Health and Human Development [34]: David Siegel, Perdita Taylor-Zapata, Anne Zajicek
- The EMMES Corporation (data coordinating center) [35]: Ravinder Anand, Traci Clemons
Source of the article : Wikipedia
EmoticonEmoticon