BACKGROUND: PEDS-C is the first multicenter placebo-controlled trial for the treatment of chronic hepatitis C (HCV) in childhood that has ever been conducted in the United States (USA). Establishment of the research team, protocol, administrative infrastructure, and ancillary contributors for the pediatric trial took years of planning. PURPOSE: To study the safety and efficacy of pegylated-interferon alpha (PEG-2a) plus ribavirin (RV) with PEG-2a monotherapy in children aged 5 years through 18 years. To assess the health-related quality of life and growth and body composition in children with chronic hepatitis C infection, before, during, and after treatment. METHODS:Eleven centers of pediatric hepatobiliary clinical research were united in a National Institutes of Diabetes and Digestive and Kidney Diseases (NIDDK) funded grant with financial support from the Food and Drug Administration (FDA) and a corporate sponsor to conduct the treatment trial. LIMITATIONS: The most important initial limitation in the design of this complex study was securing the financial support and infrastructural organization, a process that took several years. Challenges faced by the study group included identifying the optimal study design given the limited study population, and determining what ancillary studies could be incorporated into the treatment trial. CONCLUSIONS: In this article the process taken to design the study and administrative infrastructure, the lessons learned, and the controversial issues deliberated during the planning process are discussed. The evolution of the study and the considerations taken in the development of the protocol are valuable tools which can be applied to pediatric clinical trials in general.
RCT Entities:
BACKGROUND: PEDS-C is the first multicenter placebo-controlled trial for the treatment of chronic hepatitis C (HCV) in childhood that has ever been conducted in the United States (USA). Establishment of the research team, protocol, administrative infrastructure, and ancillary contributors for the pediatric trial took years of planning. PURPOSE: To study the safety and efficacy of pegylated-interferon alpha (PEG-2a) plus ribavirin (RV) with PEG-2a monotherapy in children aged 5 years through 18 years. To assess the health-related quality of life and growth and body composition in children with chronic hepatitis C infection, before, during, and after treatment. METHODS: Eleven centers of pediatric hepatobiliary clinical research were united in a National Institutes of Diabetes and Digestive and Kidney Diseases (NIDDK) funded grant with financial support from the Food and Drug Administration (FDA) and a corporate sponsor to conduct the treatment trial. LIMITATIONS: The most important initial limitation in the design of this complex study was securing the financial support and infrastructural organization, a process that took several years. Challenges faced by the study group included identifying the optimal study design given the limited study population, and determining what ancillary studies could be incorporated into the treatment trial. CONCLUSIONS: In this article the process taken to design the study and administrative infrastructure, the lessons learned, and the controversial issues deliberated during the planning process are discussed. The evolution of the study and the considerations taken in the development of the protocol are valuable tools which can be applied to pediatric clinical trials in general.
Authors: S Zeuzem; S V Feinman; J Rasenack; E J Heathcote; M Y Lai; E Gane; J O'Grady; J Reichen; M Diago; A Lin; J Hoffman; M J Brunda Journal: N Engl J Med Date: 2000-12-07 Impact factor: 91.245
Authors: D L Musselman; D H Lawson; J F Gumnick; A K Manatunga; S Penna; R S Goodkin; K Greiner; C B Nemeroff; A H Miller Journal: N Engl J Med Date: 2001-03-29 Impact factor: 91.245
Authors: L Comanor; J Minor; H S Conjeevaram; E A Roberts; F Alvarez; E M Bern; P Goyens; P Rosenthal; A Lachaux; M Shelton; J Sarles; E M Sokal Journal: J Viral Hepat Date: 2001-03 Impact factor: 3.728
Authors: Kathleen B Schwarz; Parvathi Mohan; Michael R Narkewicz; Jean Pappas Molleston; S Russell Nash; Sylvia Hu; Ka Wang; Jean Michel Gries Journal: J Pediatr Gastroenterol Nutr Date: 2006-10 Impact factor: 2.839
Authors: M P Manns; J G McHutchison; S C Gordon; V K Rustgi; M Shiffman; R Reindollar; Z D Goodman; K Koury; M Ling; J K Albrecht Journal: Lancet Date: 2001-09-22 Impact factor: 79.321
Authors: Kathleen B Schwarz; Regino P Gonzalez-Peralta; Karen F Murray; Jean P Molleston; Barbara A Haber; Maureen M Jonas; Philip Rosenthal; Parvathi Mohan; William F Balistreri; Michael R Narkewicz; Lesley Smith; Steven J Lobritto; Stephen Rossi; Alexandra Valsamakis; Zachary Goodman; Patricia R Robuck; Bruce A Barton Journal: Gastroenterology Date: 2010-10-28 Impact factor: 22.682
Authors: Jean P Molleston; William Mellman; Michael R Narkewicz; William F Balistreri; Regino P Gonzalez-Peralta; Maureen M Jonas; Steven J Lobritto; Parvathi Mohan; Karen F Murray; Dolores Njoku; Philip Rosenthal; Bruce A Barton; Monica V Talor; Irene Cheng; Kathleen B Schwarz; Barbara A Haber Journal: J Pediatr Gastroenterol Nutr Date: 2013-03 Impact factor: 2.839
Authors: Maureen M Jonas; William Balistreri; Regino P Gonzalez-Peralta; Barbara Haber; Steven Lobritto; Parvathi Mohan; Jean P Molleston; Karen F Murray; Michael R Narkewicz; Philip Rosenthal; Kathleen B Schwarz; Bruce A Barton; John A Shepherd; Paul D Mitchell; Christopher Duggan Journal: Hepatology Date: 2012-07-06 Impact factor: 17.425
Authors: James R Rodrigue; William Balistreri; Barbara Haber; Maureen M Jonas; Parvathi Mohan; Jean P Molleston; Karen F Murray; Michael R Narkewicz; Philip Rosenthal; Lesley J Smith; Steven J Lobritto; Kathleen B Schwarz; Patricia R Robuck; Bruce Barton; Regino P González-Peralta Journal: Hepatology Date: 2011-05 Impact factor: 17.425
Authors: Parvathi Mohan; Bruce A Barton; Michael R Narkewicz; Jean P Molleston; Regino P Gonzalez-Peralta; Philip Rosenthal; Karen F Murray; Barbara Haber; Kathleen B Schwarz; Zachary D Goodman Journal: Hepatology Date: 2013-09-30 Impact factor: 17.425
Authors: James R Rodrigue; William Balistreri; Barbara Haber; Maureen M Jonas; Parvathi Mohan; Jean P Molleston; Karen F Murray; Michael R Narkewicz; Philip Rosenthal; Lesley J Smith; Kathleen B Schwarz; Patricia Robuck; Bruce Barton; Regino P González-Peralta Journal: J Pediatr Gastroenterol Nutr Date: 2009-03 Impact factor: 2.839