A double-blind, randomized, placebo-controlled trial studying the effects of Saccharomyces boulardii on the gastrointestinal tolerability, safety, and pharmacokinetics of miglustat

Orphanet Journal of Rare Diseases

Volumn 10, Issue 1, 2015, Pages

  Remenova, Tatiana ^a   Morand, Olivier ^a   Amato, Dominick ^b   Chadha Boreham, Harbajan ^a   Tsurutani, Scott ^c   Marquardt, Thorsten ^d  

^a ACTELION PHARMACEUTICALS LTD   (Switzerland)

^b MOUNT SINAI HOSPITAL   (Canada)

^c Actelion Pharmaceuticals US Inc   (United States)

^d UNIVERSITY HOSPITAL MÜNSTER   (Germany)

Author keywords

Diarrhea; Miglustat; Niemann Pick disease type C; S. boulardii; Type 1 Gaucher disease

Indexed keywords

MIGLUSTAT; PLACEBO; PROBIOTIC AGENT; 1 DEOXYNOJIRIMYCIN; ENZYME INHIBITOR;

ABDOMINAL DISCOMFORT; ABDOMINAL PAIN; ADULT; ARTICLE; BLOATING; CONTROLLED STUDY; CROSSOVER PROCEDURE; DIARRHEA; DOUBLE BLIND PROCEDURE; DRUG BLOOD LEVEL; DRUG SAFETY; DRUG TOLERABILITY; DYSPEPSIA; ERUCTATION; FEMALE; FLATULENCE; GASTROINTESTINAL SYMPTOM; HUMAN; HUMAN EXPERIMENT; INCIDENCE; INDIGESTION; LOOSE FECES; MALE; NAUSEA; NONHUMAN; NORMAL HUMAN; PHASE 1 CLINICAL TRIAL; RANDOMIZED CONTROLLED TRIAL; SACCHAROMYCES BOULARDII; STOMACH DISTENSION; ANALOGS AND DERIVATIVES; DRUG EFFECTS; GASTROINTESTINAL TRACT; MIDDLE AGED; SACCHAROMYCES;

1-DEOXYNOJIRIMYCIN; ADULT; CROSS-OVER STUDIES; DOUBLE-BLIND METHOD; ENZYME INHIBITORS; FEMALE; GASTROINTESTINAL TRACT; HUMANS; MALE; MIDDLE AGED; PLACEBOS; SACCHAROMYCES;

EID: 84937206815     PISSN: None     EISSN: 17501172     Source Type: Journal    
DOI: 10.1186/s13023-015-0297-7     Document Type: Article

References (30)

1. Actelion. Zavesca® (miglustat capsules) prescribing information. Available at: http://www.zavesca.com/. Accessed 30 April, 2015.
2. Miglustat (Zavesca®) Summary of Product Characteristics. Available at: http://www.ema.europa.eu/ema/index.jsp?curl=pages/medicines/human/medicines/000435/human-med-001171.jsp&murl=menus/medicines/medicines.jsp&mid=WC0b01ac058001d125. Accessed 30 April, 2015.
3. Elstein D, Hollak C, Aerts JM, van Weely S, Maas M, Cox TM, et al. Sustained therapeutic effects of oral miglustat (Zavesca, N-butyldeoxynojirimycin, OGT 918) in type I Gaucher disease. J Inherit Metab Dis. 2004;27:757-66.
4. Elstein D, Dweck A, Attias D, Hadas-Halpern I, Zevin S, Altarescu G, et al. Oral maintenance clinical trial with miglustat for type I Gaucher disease: switch from or combination with intravenous enzyme replacement. Blood. 2007;110:2296-301.
5. Heitner R, Elstein D, Aerts J, Weely S, Zimran A. Low-dose N-butyldeoxynojirimycin (OGT 918) for type I Gaucher disease. Blood Cells Mol Dis. 2002;28:127-33.
6. Cox T, Lachmann R, Hollak C, Aerts J, van Weely S, Hrebicek M, et al. Novel oral treatment of Gaucher's disease with N-butyldeoxynojirimycin (OGT 918) to decrease substrate biosynthesis. Lancet. 2000;355:1481-5.
7. Patterson MC, Vecchio D, Prady H, Abel L, Wraith JE. Miglustat for treatment of Niemann-Pick C disease: a randomised controlled study. Lancet Neurol. 2007;6:765-72.
8. Patterson MC, Vecchio D, Jacklin E, Abel L, Chadha-Boreham H, Luzy C, et al. Long-term miglustat therapy in children with Niemann-Pick disease type C. J Child Neurol. 2010;25:300-5.
9. Wraith JE, Vecchio D, Jacklin E, Abel L, Chadha-Boreham H, Luzy C, et al. Miglustat in adult and juvenile patients with Niemann-Pick disease type C: long-term data from a clinical trial. Mol Genet Metab. 2010;99:351-7.
10. Pineda M, Wraith JE, Mengel E, Sedel F, Hwu WL, Rohrbach M, et al. Miglustat in patients with Niemann-Pick disease Type C (NP-C): a multicenter observational retrospective cohort study. Mol Genet Metab. 2009;98:243-9.
11. Belmatoug N, Burlina A, Giraldo P, Hendriksz CJ, Kuter DJ, Mengel E, et al. Gastrointestinal disturbances and their management in miglustat-treated patients. J Inherit Metab Dis. 2011;34:991-1001.
12. Hollak CE, Hughes D, van Schaik IN, Schwierin B, Bembi B. Miglustat (Zavesca) in type 1 Gaucher disease: 5-year results of a post-authorisation safety surveillance programme. Pharmacoepidemiol Drug Saf. 2009;18:770-7.
13. Andersson U, Butters TD, Dwek RA, Platt FM. N-butyldeoxygalactonojirimycin: a more selective inhibitor of glycosphingolipid biosynthesis than N-butyldeoxynojirimycin, in vitro and in vivo. Biochem Pharmacol. 2000;59:821-9.
14. Amiri M, Naim HY. Long term differential consequences of miglustat therapy on intestinal disaccharidases. J Inherit Metab Dis. 2014;37:929-37.
15. Amiri M, Naim HY. Miglustat-induced intestinal carbohydrate malabsorption is due to the inhibition of alpha-glucosidases, but not beta-galactosidases. J Inherit Metab Dis. 2012;35:949-54.
16. Butters TD, van den Broek LAGM, Fleet GWJ, Krulle TM, Wormald MR, Dwek RA, et al. Molecular requirements of imino sugars for the selective control of N-linked glycosylation and glycosphingolipid biosynthesis. Tetrahedron Asymmetry. 2000;11:113-24.
17. Schiweck S, Clarke M, Pollach G. Ullmann's Encyclopedia of Inustrial Chemistry. Weinheim: Wiley-VCH Verlag GmbH & Co. KGaA; 2012.
18. Dechelotte P. Type 1 Gaucher's disease in the adult. Nutritional management during initiation of treatment with miglustat. Presse Med. 2004;33:494-6.
19. Cox TM, Aerts JM, Andria G, Beck M, Belmatoug N, Bembi B, et al. The role of the iminosugar N-butyldeoxynojirimycin (miglustat) in the management of type I (non-neuronopathic) Gaucher disease: a position statement. J Inherit Metab Dis. 2003;26:513-26.
20. Pastores GM, Barnett NL, Kolodny EH. An open-label, noncomparative study of miglustat in type I Gaucher disease: efficacy and tolerability over 24 months of treatment. Clin Ther. 2005;27:1215-27.
21. Giraldo P, Latre P, Alfonso P, Acedo A, Alonso D, Barez A, et al. Short-term effect of miglustat in every day clinical use in treatment-naive or previously treated patients with type 1 Gaucher's disease. Haematologica. 2006;91:703-6.
22. Giraldo P, Alfonso P, Atutxa K, Fernandez-Galan MA, Barez A, Franco R, et al. Real-world clinical experience with long-term miglustat maintenance therapy in type 1 Gaucher disease: the ZAGAL project. Haematologica. 2009;94:1771-5.
23. Champion H, Ramaswami U, Imrie J, Lachmann RH, Gallagher J, Cox TM, et al. Dietary modifications in patients receiving miglustat. J Inherit Metab Dis. 2010;33 Suppl 3:S379-83.
24. Treem WR, McAdams L, Stanford L, Kastoff G, Justinich C, Hyams J. Sacrosidase therapy for congenital sucrase-isomaltase deficiency. J Pediatr Gastroenterol Nutr. 1999;28:137-42.
25. Lucke T, Keiser M, Illsinger S, Lentze MJ, Naim HY, Das AM. Congenital and putatively acquired forms of sucrase-isomaltase deficiency in infancy: effects of sacrosidase therapy. J Pediatr Gastroenterol Nutr. 2009;49:485-7.
26. Harms HK, Bertele-Harms RM, Bruer-Kleis D. Enzyme-substitution therapy with the yeast Saccharomyces cerevisiae in congenital sucrase-isomaltase deficiency. N Engl J Med. 1987;316:1306-9.
27. Buts JP, De Keyser N, Marandi S, Hermans D, Sokal EM, Chae YH, et al. Saccharomyces boulardii upgrades cellular adaptation after proximal enterectomy in rats. Gut. 1999;45:89-96.
28. Buts JP, Bernasconi P, Van Craynest MP, Maldague P, De Meyer R. Response of human and rat small intestinal mucosa to oral administration of Saccharomyces boulardii. Pediatr Res. 1986;20:192-6.
29. Arends M, van Dussen L, Biegstraaten M, Hollak CE. Malignancies and monoclonal gammopathy in Gaucher disease; a systematic review of the literature. Br J Haematol. 2013;161:832-42.
30. Lewis SJ, Heaton KW. Stool form scale as a useful guide to intestinal transit time. Scand J Gastroenterol. 1997;32:920-4.