Efficacy and tolerability of a new formulation of pancrelipase delayed-release capsules in children aged 7 to 11 years with exocrine pancreatic insufficiency and cystic fibrosis: A multicenter, randomized, double-blind, placebo-controlled, two-period crossover, superiority study

Clinical Therapeutics

Volumn 32, Issue 1, 2010, Pages 89-103

  Graff, Gavin R ^a   Maguiness, Karen ^b   McNamara, John ^c   Morton, Ronald ^d   Boyd, David ^e   Beckmann, Katrin ^f   Bennett, Djenane ^e  

^a PENN STATE MILTON S HERSHEY MEDICAL CENTER   (United States)

^b INDIANA UNIVERSITY SCHOOL OF MEDICINE   (United States)

^c CHILDREN S HOSPITALS AND CLINICS OF MINNESOTA   (United States)

^d University of Louisville School of Medicine   (United States)

^e Solvay Pharmaceuticals Inc   (Georgia)

^f Solvay Pharmaceuticals GmbH   (Germany)

Author keywords

coefficient of fat absorption; cystic fibrosis; exocrine pancreatic insufficiency; pancreatic enzyme replacement therapy; pancrelipase; pediatric

Indexed keywords

FAT; GASTROINTESTINAL AGENT; NITROGEN; PANCRELIPASE; PLACEBO;

ARTICLE; CHILD; CLINICAL TRIAL; CONTROLLED CLINICAL TRIAL; CONTROLLED STUDY; CROSSOVER PROCEDURE; CYSTIC FIBROSIS; DELAYED RELEASE FORMULATION; DOUBLE BLIND PROCEDURE; DRUG EFFECT; ENZYME REPLACEMENT; FECES; FEMALE; HUMAN; INTESTINE ABSORPTION; MALE; METABOLISM; MICROCAPSULE; MULTICENTER STUDY; PANCREAS EXOCRINE INSUFFICIENCY; RANDOMIZED CONTROLLED TRIAL; STEATORRHEA; TREATMENT OUTCOME;

CAPSULES; CHILD; CROSS-OVER STUDIES; CYSTIC FIBROSIS; DELAYED-ACTION PREPARATIONS; DOUBLE-BLIND METHOD; ENZYME REPLACEMENT THERAPY; EXOCRINE PANCREATIC INSUFFICIENCY; FATS; FECES; FEMALE; GASTROINTESTINAL AGENTS; HUMANS; INTESTINAL ABSORPTION; MALE; NITROGEN; PANCRELIPASE; PLACEBOS; STEATORRHEA; TREATMENT OUTCOME;

MLCS; MLOWN;

EID: 77952030011     PISSN: 01492918     EISSN: 1879114X     Source Type: Journal    
DOI: 10.1016/j.clinthera.2010.01.012     Document Type: Article

References (33)

1. Baker, SS, Delayed release pancrelipase for the treatment of pancreatic exocrine insufficiency associated with cystic fibrosis. Ther Clin Risk Manag. 4 (2008), 1079–1084.
2. Borowitz, D, Durie, PR, Clarke, LL, et al. Gastrointestinal outcomes and confounders in cystic fibrosis. J Pediatr Gastroenterol Nutr. 41 (2005), 273–285.
3. Littlewood, JM, Wolfe, SP, Conway, SP, Diagnosis and treatment of intestinal malabsorption in cystic fibrosis. Pediatr Pulmonol. 41 (2006), 35–49.
4. DiMagno, MJ, DiMagno, EP, Chronic pancreatitis. Curr Opin Gastroenterol. 22 (2006), 487–497.
5. Layer, P, Holtmann, G, Pancreatic enzymes in chronic pancreatitis. Int J Pancreatol. 15 (1994), 1–11.
6. Abdel Aziz, AM, Lehman, GA, Current treatment options for chronic pancreatitis. Curr Treat Options Gastroenterol. 10 (2007), 355–368.
7. Ratjen, F, Döring, G, Cystic fibrosis. Lancet. 361 (2003), 681–689.
8. Couper, RT, Corey, M, Moore, DJ, et al. Decline of exocrine pancreatic function in cystic fibrosis patients with pancreatic sufficiency. Pediatr Res. 32 (1992), 179–182.
9. Borowitz, D, Baker, RD, Stallings, V, Consensus report on nutrition for pediatric patients with cystic fibrosis. J Pediatr Gastroenterol Nutr. 35 (2002), 246–259.
10. Bronstein, MN, Sokol, RJ, Abman, SH, et al. Pancreatic insufficiency, growth, and nutrition in infants identified by newborn screening as having cystic fibrosis. J Pediatr. 120 (1992), 533–540.
11. FitzSimmons, SC, The changing epidemiology of cystic fibrosis. J Pediatr. 122 (1993), 1–9.
12. Stallings, VA, Stark, LJ, Robinson, KA, et al., Ad Hoc Working Group. Evidence-based practice recommendations for nutrition-related management of children and adults with cystic fibrosis and pancreatic insufficiency: Results of a systematic review. J Am Diet Assoc. 108 (2008), 832–839.
13. Konstan, MW, Butler, SM, Wohl, ME, et al., Investigators and Coordinators of the Epidemiologic Study of Cystic Fibrosis. Growth and nutritional indexes in early life predict pulmonary function in cystic fibrosis. J Pediatr. 142 (2003), 624–630.
14. Beker, LT, Russek-Cohen, E, Fink, RJ, Stature as a prognostic factor in cystic fibrosis survival. J Am Diet Assoc. 101 (2001), 438–442.
15. Konstan, MW, Stern, RC, Trout, JR, et al. Ultrase MT12 and Ultrase MT20 in the treatment of exocrine pancreatic insufficiency in cystic fibrosis: Safety and efficacy. Aliment Pharmacol Ther. 20 (2004), 1365–1371.
16. Borowitz, D, Goss, CH, Limauro, S, et al. Study of a novel pancreatic enzyme replacement therapy in pancreatic insufficient subjects with cystic fibrosis. J Pediatr. 149 (2006), 658–662.
17. Borowitz, D, Goss, CH, Stevens, C, et al. Safety and preliminary clinical activity of a novel pancreatic enzyme preparation in pancreatic insufficient cystic fibrosis patients. Pancreas. 32 (2006), 258–263.
18. Brady, MS, Garson, JL, Krug, SK, et al. An enteric-coated high-buffered pancrelipase reduces steatorrhea in patients with cystic fibrosis: A prospective, randomized study. J Am Diet Assoc. 106 (2006), 1181–1186.
19. Safdi, M, Bekal, PK, Martin, S, et al. The effects of oral pancreatic enzymes (Creon 10 capsule) on steatorrhea: A multicenter, placebocontrolled, parallel group trial in subjects with chronic pancreatitis [published correction appears in Pancreas. 2007;34:174]. Pancreas 33 (2006), 156–162.
20. Santini, B, Antonelli, M, Battistini, A, et al. Comparison of two enteric coated microsphere preparations in the treatment of pancreatic exocrine insufficiency caused by cystic fibrosis. Dig Liver Dis. 32 (2000), 406–411.
21. Stern, RC, Eisenberg, JD, Wagener, JS, et al. A comparison of the efficacy and tolerance of pancrelipase and placebo in the treatment of steatorrhea in cystic fibrosis patients with clinical exocrine pancreatic insufficiency. Am J Gastroenterol. 95 (2000), 1932–1938.
22. US Food and Drug Administration. TFDA requires pancreatic extract manufacturers to submit marketing applications. http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/2004/ucm108289.htm Accessed December 2, 2009.
23. Trapnell, BC, Maguiness, K, Graff, GR, et al. Efficacy and safety of Creon 24,000 in subjects with exocrine pancreatic insufficiency due to cystic fibrosis. J Cyst Fibros. 8 (2009), 370–377.
24. Code of Federal Regulations. Title 21—Food and Drugs. http://www.accessdata.fda.gov/SCRIPTs/cdrh/cfdocs/cfcfr/CFRSearch.cfm Accessed December, 2, 2009.
25. International Conference on Harmonisation. Guideline for Industry. Structure and contents of clinical study reports (ICH E3). http://www.fda.gov/downloads/RegulatoryInformation/Guidances/UCM129456.pdf Accessed December 14, 2009.
26. International Conference on Harmonisation. ICH Harmonised Tripartite Guideline. Statistical principles for clinical trials (E9). http://www.ich.org/LOB/media/MEDIA485.pdf Accessed December 14, 2009.
27. International Conference on Harmonisation. ICH Harmonised Tripartite Guideline. Guideline for good clinical practice E6(R1). http://www.ich.org/LOB/media/MEDIA482.pdf Accessed December 14, 2009.
28. Sinaasappel, M, Stern, M, Littlewood, J, et al. Nutrition in patients with cystic fibrosis: A European Consensus. J Cyst Fibros. 1 (2002), 51–75.
29. Van De Kamer, JH, Ten Bokkel Huinink, H, Weyers, HA, Rapid method for the determination of fat in feces. J Biol Chem. 177 (1949), 347–355.
30. Kjeldahl, J, A new method for the determination of nitrogen in organic matter. Z Anal Chem. 22 (1883), 366–382.
31. Borowitz, D, Konstan, MW, O'Rourke, MW, et al. Coefficients of fat and nitrogen absorption in healthy subjects and individuals with cystic fibrosis. J Pediatr Phamacol Ther. 12 (2007), 47–52.
32. Colombo, C, Fredella, C, Russo, MC, et al. Efficacy and tolerability of Creon for Children in infants and toddlers with pancreatic exocrine insufficiency caused by cystic fibrosis: An open-label, single-arm, multicenterstudy. Pancreas. 38 (2009), 693–699.
33. Sander-Struckmeier, S, Boyd, D, Caras, SD, CREON: Efficacy and safety in children less than 7 years old with exocrine pancreatic insufficiency due to cystic fibrosis. J Cyst Fibros, 8(Suppl 2), 2009, S80 Abstract P324.