Structure of Miglustat
CAS No.: 72599-27-0
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The BI-3802 was designed by Boehringer Ingelheim and could be obtained free of charge through the Boehringer Ingelheim open innovation portal opnMe.com, associated with its negative control.
Miglustat is an inhibitor of glucosylceramide synthase, primarily to treat Type I Gaucher disease (GD1).
Synonyms: NB-DNJ; N-Butyldeoxynojirimycin; N‐butyldeoxynojirimycin
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Batch number can be found on the product's label following the word 'Batch'.
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Batch number can be found on the product's label following the word 'Batch'.
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Batch number can be found on the product's label following the word 'Batch'.
Search for reports by entering the product batch number.
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CAS No. : | 72599-27-0 |
Formula : | C10H21NO4 |
M.W : | 219.28 |
SMILES Code : | O[C@H]1[C@H](O)[C@@H](CO)N(CCCC)C[C@@H]1O |
Synonyms : |
NB-DNJ; N-Butyldeoxynojirimycin; N‐butyldeoxynojirimycin
|
MDL No. : | MFCD00272581 |
InChI Key : | UQRORFVVSGFNRO-UTINFBMNSA-N |
Pubchem ID : | 51634 |
GHS Pictogram: |
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Signal Word: | Warning |
Hazard Statements: | H361 |
Precautionary Statements: | P501-P202-P201-P280-P308+P313-P405 |
In Vitro:
Concentration | Treated Time | Description | References |
RAW264.7 cells | >50 μM | Assess the inhibitory effect of Miglustat on glucosylceramide synthase (GCS). Results indicated no significant inhibition of GCS at 50 μM concentration. | J Am Chem Soc. 2022 Aug 17;144(32):14819-14827 | |
Pompe disease fibroblasts | 20 μM | 1-3 days | Evaluate the stabilizing effect of Miglustat on recombinant human α-glucosidase (rhGAA). Results showed a two-fold increase in rhGAA activity after 3 days in fibroblasts treated with both rhGAA and Miglustat compared to those treated with rhGAA alone. | J Am Chem Soc. 2022 Aug 17;144(32):14819-14827 |
RAW264.7 cells | 500 μM | 4 days | To study the effect of Miglustat on osteoclast lipid raft organization and NFATc1 nuclear localization, results showed that Miglustat disrupted lipid raft function. | J Clin Invest. 2015 Jun;125(6):2279-92 |
Mouse bone marrow cells | 5, 50, 500 μM | 5 days | To evaluate the effect of Miglustat on osteoclast formation, results showed that Miglustat significantly inhibited osteoclast formation. | J Clin Invest. 2015 Jun;125(6):2279-92 |
Neonatal rat cardiac fibroblasts (NRCFs) | 100 μM and 200 μM | 24 hours | To evaluate the effect of Miglustat on ISO-induced cardiac fibroblast activation. Results showed that Miglustat significantly inhibited ISO-induced NRCFs proliferation and reduced the expression of fibrosis markers (such as PCNA, CTGF, Fn, Col-I, Col-III, and α-SMA). | Mol Med. 2025 Feb 11;31(1):55 |
Primary 409 cells | 100 µM | 72 hours | To evaluate the effect of Miglustat on GSL expression in primary cells, results showed a reduction in GD2 expression | Int J Mol Sci. 2023 Jun 8;24(12):9905 |
A549-ACE2 cells | 100 μM | 72 hours | Evaluate the anti-SARS-CoV-2 activity of Miglustat, showing EC90 of 100 μM | J Enzyme Inhib Med Chem. 2024 Dec;39(1):2289007 |
Human astrocytes | 100 μM | 24 hours | Miglustat suppresses the expression of Pla2g4 and pro-inflammatory genes in activated human astrocytes | Cell. 2019 Dec 12;179(7):1483-1498. e22 |
GBA1-PD iPSC-derived hDA neurons | 100 μM | 3 days | reduce GlcCer accumulation and restore the ratio of α-syn tetramers and related multimers | Proc Natl Acad Sci U S A. 2018 Jan 23;115(4):798-803 |
SH-SY5Y cells | 100 μM | 3 days | reduce intracellular GlcCer levels and restore the ratio of α-syn tetramers and related multimers | Proc Natl Acad Sci U S A. 2018 Jan 23;115(4):798-803 |
SF8628 cells | 100 µM | 72 hours | To analyze the effect of Miglustat on GSL expression, results showed a significant decrease in GD2 expression | Int J Mol Sci. 2023 Jun 8;24(12):9905 |
SF8628 cells | 100 µM | 72 hours | To assess the effect of Miglustat on cell proliferation, results showed no significant impairment in cell viability after 72 hours of treatment | Int J Mol Sci. 2023 Jun 8;24(12):9905 |
In Vivo:
Administration | Dosage | Frequency | Description | References |
C57BL/6 mice | Multiple myeloma model | Oral gavage | 50 mg/mouse/day | Once daily for 21 days | To evaluate the effect of Miglustat on myeloma bone disease, results showed that Miglustat significantly improved bone disease symptoms. | J Clin Invest. 2015 Jun;125(6):2279-92 |
C57BL/6J mice | ISO-induced cardiac fibrosis model | Oral | 150 mg/kg and 300 mg/kg | Twice daily for 7 days | To evaluate the effect of Miglustat on ISO-induced cardiac dysfunction and fibrosis. Results showed that Miglustat improved ISO-induced cardiac dysfunction, reduced cardiac fibrosis areas, and decreased the expression of fibrosis markers. | Mol Med. 2025 Feb 11;31(1):55 |
NOD mice | Experimental autoimmune encephalomyelitis (EAE) | Oral | 600 mg/kg | Daily until EAE progressive phase | Miglustat suppresses NOD EAE progression, reducing axonal loss, demyelination, and recruitment of pro-inflammatory monocytes to the CNS | Cell. 2019 Dec 12;179(7):1483-1498. e22 |
Mouse | Mcoln1−/− mouse model | Intraperitoneal injection | 300 mg/kg body weight | Daily from P10-P21, then three times a week until the end of the study | To evaluate the effects of Miglustat on motor deficits, cerebellar microgliosis, and Purkinje cell loss in the MLIV mouse model. Results showed that Miglustat delays the onset and progression of motor deficits, delays cerebellar Purkinje cell loss, and reduces cerebellar microgliosis characteristic of MLIV disease. | Neurobiol Dis. 2017 Sep;105:257-270 |
Clinical Trial:
NCT Number | Conditions | Phases | Recruitment | Completion Date | Locations |
NCT02612129 | Niemann-Pick Disease, Type C | Phase 2 Phase 3 | Active, not recruiting | October 2020 | United States, California ... More >> UCSF Benioff Children's Hospital Oakland Oakland, California, United States, 94609 United States, Minnesota Mayo Clinic Children's Center Rochester, Minnesota, United States, 55905 Denmark University Hospital Copenhagen (Rigshospitalet) Copenhagen, Denmark, 2100 France CHU de Montpellier Montpellier, France, 34295 Montpellier Cedex 5 Hôpital Trousseau Paris, France, 75571 PARIS Cedex 12 Germany Villa Metabolica Mainz Mainz, Germany, 55131 Dr. von Haunersches Kinderspital der Universität München Munich, Germany, 80337 Italy Ospedale Pediatrico Bambino Gesù Rome, Italy, 00165 Azienda Ospedaliero-Universitaria "Santa Maria della Misericordia" di Udin Udine, Italy, 33100 Poland The Children´s Memorial Istitute Warsaw Warsaw, Poland, 04-730 Spain Hospital Vall D'Hebron Barcelona, Spain, 08035 Switzerland INSELSPITAL University Hospital Bern Bern, Switzerland, CH-3010 United Kingdom Birmingham Children's Hospital Birmingham, United Kingdom, B4 6NH Great Ormond Street Hospital London, United Kingdom, WC1N 3JH Less << |
NCT00000791 | HIV Infections | Phase 2 | Completed | - | United States, Alabama ... More >> Alabama Therapeutics CRS Birmingham, Alabama, United States United States, California USC CRS Los Angeles, California, United States, 90033 Stanford CRS Palo Alto, California, United States Ucsf Aids Crs San Francisco, California, United States, 94110 United States, Colorado University of Colorado Hospital CRS Aurora, Colorado, United States United States, Florida Univ. of Miami AIDS CRS Miami, Florida, United States United States, Illinois Northwestern University CRS Chicago, Illinois, United States, 60611 Rush Univ. Med. Ctr. ACTG CRS Chicago, Illinois, United States, 60612 Weiss Memorial Hosp. Chicago, Illinois, United States, 60640 Cook County Hosp. CORE Ctr. Chicago, Illinois, United States United States, Indiana Indiana Univ. School of Medicine, Infectious Disease Research Clinic Indianapolis, Indiana, United States Methodist Hosp. of Indiana Indianapolis, Indiana, United States United States, Missouri St. Louis ConnectCare, Infectious Diseases Clinic St. Louis, Missouri, United States, 63112 Washington U CRS St. Louis, Missouri, United States United States, New York SUNY - Buffalo, Erie County Medical Ctr. Buffalo, New York, United States, 14260 Beth Israel Med. Ctr. (Mt. Sinai) New York, New York, United States Univ. of Rochester ACTG CRS Rochester, New York, United States United States, North Carolina Unc Aids Crs Chapel Hill, North Carolina, United States, 27514 Wake County Health and Human Services CRS Raleigh, North Carolina, United States United States, Ohio Univ. of Cincinnati CRS Cincinnati, Ohio, United States United States, Pennsylvania Hosp. of the Univ. of Pennsylvania CRS Philadelphia, Pennsylvania, United States United States, Washington University of Washington AIDS CRS Seattle, Washington, United States, 98104 Less << |
NCT02675465 | Pompe Disease | Phase 1 Phase 2 | Recruiting | September 2019 | United States, Arizona ... More >> Neuromuscular Research Centre Recruiting Phoenix, Arizona, United States, 85028 Contact: Kumaraswamy Sivakumar 480-314-1007 ext 6 ksiva@nrcaz.com Contact: Kristy Osgood 480-314-1007 ext 6 kosgood@nrcaz.com Principal Investigator: Kumaraswamy Sivakumar United States, California University of California Irvine Recruiting Orange, California, United States, 92868 Contact: Tahseen Mozaffar 714-456-2332 Mozaffar@uci.edu Contact: Marie Wencel 714-456-2525 mwencel@uci.edu Principal Investigator: Tahseen Mozaffar United States, Florida University of Florida Recruiting Gainesville, Florida, United States, 32610 Contact: Barry J Byrne 352-273-6563 bbyrne@ufl.edu Contact: Lee Kugelmann 352-273-6588 ekugelm@ufl.edu Principal Investigator: Barry Byrne United States, Georgia Emory University Division of Medical Genetics Completed Decatur, Georgia, United States, 30033 United States, Michigan Infusion Associates Recruiting Grand Rapids, Michigan, United States, 49525 Contact: Khan Nedd, MD 616-954-0600 khan.nedd@infusionassociates.com Contact: Katie May 616-818-4473 katie.may@infusionassociates.com Principal Investigator: Khan Nedd United States, Montana Great Falls Clinic, LLP Recruiting Great Falls, Montana, United States, 59405 Contact: Karl Guter 406-771-3311 karl.guter@gfclinic.com Contact: Jacob Decker 406-771-3373 jacob.decker@gfclinic.com Principal Investigator: Karl Guter United States, New Jersey Rutgers New Jersey Medical School Recruiting Newark, New Jersey, United States, 08103 Contact: Xue Ming 973-972-2922 mingxu@njms.rutgers.edu Contact: Eliana Obando-Jaramillo 973-972-5435 emo65@njms.rutgers.edu Principal Investigator: Xue Ming United States, North Carolina Duke University Medical Center Recruiting Durham, North Carolina, United States, 27710 Contact: Priya Kishnani 919-684-2036 priya.kishnani@dm.duke.edu Contact: Ela Stefanescu 919-681-4026 mihaela.stefanescu@duke.edu Principal Investigator: Priya Kishnani United States, Pennsylvania University of Pittsburgh Recruiting Pittsburgh, Pennsylvania, United States, 15213 Contact: Paula Clemens 412-648-9762 clempr@upmc.edu Contact: Gabriela A Niizawa 412-383-9775 niizawaga@upmc.edu Principal Investigator: Paula Clemens United States, Virginia Lysosomal & Rare Disorders Research & Treatment Center (LDRTC) Recruiting Fairfax, Virginia, United States, 22030 Contact: Ozlem Goker-Alpan, MD 571-308-1925 ogoker-alpan@ldrtc.org Contact: Jacqueline Fikry 703-261-6220 ext 105 jfikry@ldrtc.org Principal Investigator: Ozlem Goker-Alpan Australia, South Australia Womens & Childrens Hospital, Adelaide Active, not recruiting North Adelaide, South Australia, Australia, 05006 Germany University Children's Hospital Department of Neuropediatrics and Inborn Metabolic Disorders, St. Josefs-Hospital Recruiting Bochum, Germany, 44791 Contact: Peter Schwenkreis +49 (0) 176 55 12 95 38 peter.schwenkreis@bergmannsheil.de Contact: Rebecca Trost +49 234 3023139 rebecca.trost@bergmannsheil.de Principal Investigator: Schwenkreis Peter Friedrich-Baur-Institure, Dep of Neurology - University Munich Recruiting Munich, Germany, 80336 Contact: Benedikt Schoser +49 (0) 89 4400 57400 benedikt.schoser@med.uni-muenchen.de Contact: Stephan Wenninger +49 (0) 89 4400 57470 Stephan.Wenninger@med.uni-muenchen.de Principal Investigator: Benedikt Schoser Netherlands Erasmus Medical Center Recruiting Rotterdam, Netherlands Contact: A.T. van der Ploeg 31107037254 a.vanderploeg@erasmusmc.nl Contact: Jacqueline Hardon 31107037047 j.hardon@erasmusmc.nl Principal Investigator: A.T. van der Ploeg United Kingdom University Hospital Birmingham NHS Foundation Trust, Queen Elizabeth Medical Center Completed Birmingham, United Kingdom, B15 2TH Salford Royal NHS Foundation Trust Recruiting Salford, United Kingdom, M6 8HD Contact: Mark Roberts 0161-206-4365 mark.roberts@srft.nhs.uk Contact: Catherine Wilson 44- 01612064851 Catherine.Wilson@srft.nhs.uk Principal Investigator: Mark Roberts Less << |
NCT03729362 | Pompe Disease (Late-onset) | Phase 3 | Recruiting | January 2021 | United States, Arizona ... More >> Neuromuscular Research Center Not yet recruiting Phoenix, Arizona, United States, 85028 United States, California University of California, Irvine Recruiting Irvine, California, United States, 92868 United States, Virginia Lysosomal and Rare Disorders Research Recruiting Fairfax, Virginia, United States, 22030 Less << |
NCT00316498 | Niemann Pick Diseases | Phase 1 | Completed | - | United States, Maryland ... More >> National Institutes of Health Clinical Center, 9000 Rockville Pike Bethesda, Maryland, United States, 20892 United States, New York Columbia University New York, New York, United States, 10032-3784 United Kingdom Oxford GlycoSciences Ltd. Abingdon, United Kingdom Less << |
NCT00001993 | HIV Infections | Phase 2 | Completed | - | United States, Florida ... More >> Univ of Miami School of Medicine Miami, Florida, United States, 331361013 United States, Illinois Rush Presbyterian - Saint Luke's Med Ctr Chicago, Illinois, United States, 60612 United States, Indiana Infectious Diseases Research Clinic / Indiana Univ Hosp Indianapolis, Indiana, United States, 46202 United States, Ohio Ohio State Univ Hosp Clinic Columbus, Ohio, United States, 432101228 United States, South Carolina Julio Arroyo West Columbia, South Carolina, United States, 29169 United States, Washington Univ of Washington Seattle, Washington, United States, 981224304 Less << |
NCT00000692 | HIV Infections | Phase 1 | Completed | - | United States, California ... More >> Univ of California / San Diego Treatment Ctr San Diego, California, United States, 921036325 Stanford Univ School of Medicine Stanford, California, United States, 94305 United States, Florida Univ of Miami School of Medicine Miami, Florida, United States, 331361013 United States, Illinois Rush Presbyterian - Saint Luke's Med Ctr Chicago, Illinois, United States, 60612 United States, Massachusetts Harvard (Massachusetts Gen Hosp) Boston, Massachusetts, United States, 02114 United States, South Carolina Julio Arroyo West Columbia, South Carolina, United States, 29169 Less << |
NCT00041535 | Gaucher Disease | PHASE2 | COMPLETED | 2007-03-21 | National Institutes of Health ... More >>Clinical Center, 9000 Rockville Pike, Bethesda, Maryland, 20892, United States|Institute of Child Health, London, United Kingdom Less << |
NCT00194649 | Contraception | Phase 4 | Completed | - | United States, Washington ... More >> University of Washington Seattle, Washington, United States, 98195 Less << |
NCT01451879 | - | Completed | - | United States, Florida ... More >> University of Florida Gainesville, Florida, United States, 32610 Less << | |
NCT00002079 | HIV Infections | Phase 2 | Completed | - | United States, California ... More >> Dr Marcus Conant San Francisco, California, United States, 94115 Shared Med Research Foundation Tarzana, California, United States, 91356 United States, Florida Stratogen of South Florida Miami Beach, Florida, United States, 33140 Saint Joseph's Hosp / Infectious Disease Rsch Institute Tampa, Florida, United States, 33614 United States, Georgia West Paces Clinical Research Incoporated Atlanta, Georgia, United States, 30327 United States, Pennsylvania Dr Samuel W Golden Pittsburgh, Pennsylvania, United States, 15218 United States, Texas Dallas Veterans Administration Med Ctr Dallas, Texas, United States, 75216 Dr Daniel Barbero Fort Worth, Texas, United States, 76104 Park Plaza Hosp Houston, Texas, United States, 77004 Less << |
NCT00537602 | Cystic Fibrosis | Phase 2 | Terminated(Methodology applied... More >> did not meet all criteria required per guidelines) Less << | - | Spain ... More >> Corporacio Parc Tauli / Parc Tauli Hospital Barcelona, Spain Less << |
NCT00672022 | GM2 Gangliosidoses ... More >> Tay-Sachs Sandhoff Disease Less << | Phase 3 | Completed | - | United States, District of Col... More >>umbia Children's National Medical Center Washington, District of Columbia, United States, 20010 Less << |
NCT00945347 | Cystic Fibrosis | Phase 2 | Completed | - | Belgium ... More >> Cliniques Universitaires St Luc (Université Catholique de Louvain) 10 avenue Hippocrate Brussels, Belgium, 1200 Less << |
NCT01822028 | Diarrhea | Phase 1 | Completed | - | Canada, Ontario ... More >> BioPharma Services Inc. Toronto, Ontario, Canada, M9L 3A2 Less << |
NCT02030015 | GM1 Gangliosidoses ... More >> GM2 Gangliosidoses Tay-Sachs Disease Sandhoff Disease Less << | Phase 4 | Recruiting | August 31, 2019 | United States, Minnesota ... More >> University of Minnesota Recruiting Minneapolis, Minnesota, United States, 55455 Contact: Jeanine R. Jarnes, PharmD 612-626-5131 utzx0002@umn.edu Principal Investigator: Jeanine R. Jarnes, PharmD Sub-Investigator: Chester B. Whitley, MD, PhD Less << |
NCT00418847 | Gangliosidoses GM2 | Phase 2 | Completed | - | Canada, Ontario ... More >> The Hospital for Sick Children Toronto, Ontario, Canada, M5G 1X8 Less << |
NCT00319046 | Gaucher Disease Type 1 | Phase 3 | Completed | - | - |
NCT00319046 | - | Completed | - | - | |
NCT00742092 | Cystic Fibrosis | Phase 2 | Completed | - | Belgium ... More >> Universite Catholique de Louvain Brussels, Belgium, B-1200 Less << |
NCT02185651 | Pompe Disease ... More >> Hypersensitivity Reaction Less << | Phase 1 | Terminated(Participants not in... More >>terested in enrolling.) Less << | - | - |
NCT00517153 | Niemann-Pick Type C Disease | Phase 2 | Completed | - | United States, New York ... More >> Columbia University Medical Center New York, New York, United States, 10032 Less << |
NCT01760564 | Niemann-Pick Disease Type C | Phase 3 | Completed | - | Taiwan ... More >> National Taiwan University Hospital Taipei, Taiwan Less << |
NCT02520934 | Gaucher Disease | Not Applicable | Active, not recruiting | December 2018 | Taiwan ... More >> National Taiwan University Hospital Taipei, Taiwan, 10041 Less << |
NCT02325362 | Cystic Fibrosis | Phase 2 Phase 3 | Completed | - | France ... More >> Assistance publique-Hôpitaux de Paris, Hôpital Cochin Paris, France, 75014 Less << |
Bio Calculators | ||||
Preparing Stock Solutions | ![]() |
1mg | 5mg | 10mg |
1 mM 5 mM 10 mM |
4.56mL 0.91mL 0.46mL |
22.80mL 4.56mL 2.28mL |
45.60mL 9.12mL 4.56mL |