Drugs / Lucerastat
last change Sep 2021 re-read 2 minutes ago

Lucerastat

Small molecule targets UGCG via inhibition

Developed for
Fabry disease
Investigated by
Idorsia Pharmaceuticals Ltd.

Trials 7 · a red edge is where a trial was stopped

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PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 15 trials
Phase 1 NCT03832452 Feb → Apr 2019 Idorsia Pharmaceuticals Ltd. Completed No outcome recorded
Phase 1 NCT03380455 Jan 2018 Idorsia Pharmaceuticals Ltd. Completed No outcome recorded
Phase 1 NCT02930655 Feb 2015 → Feb 2016 Fabry disease Idorsia Pharmaceuticals Ltd. Completed No outcome recorded
Phase 1 NCT02944474 Dec 2003 → May 2004 Idorsia Pharmaceuticals Ltd. Completed No outcome recorded
Phase 1 NCT02944487 Oct → Dec 2002 Idorsia Pharmaceuticals Ltd. Completed No outcome recorded
Phase 32 trials
Phase 3 NCT03737214 Dec 2018 → Aug 2029 expected Fabry disease Idorsia Pharmaceuticals Ltd. Active not recruiting No outcome recorded
Phase 3 NCT03425539 Jun 2018 → Aug 2021 Fabry disease Idorsia Pharmaceuticals Ltd. Completed No outcome recorded

Evidence & citations 8 cited values

Every value below carries the sentence it was read from. 9 sources stand behind the page.

FieldValueCited text
Known as Lucerastat ClinicalTrials.gov intervention name — accepted as the source's own label NCT02944474
6

NCT03832452

NCT03737214

NCT02930655

NCT03425539

NCT02944487

NCT03380455

Known as ACT-434964 ChEMBL registry synonym — accepted as the source's own label CHEMBL1086997
Known as N-butyldeoxygalactonojirimycin ChEMBL registry synonym — accepted as the source's own label CHEMBL1086997
Known as N-n-butyl deoxygalactonojirimycin ChEMBL registry synonym — accepted as the source's own label CHEMBL1086997
Action Inhibit “Lucerastat, an inhibitor of glucosylceramide synthase” PMID 28088251 Jan 2017
1

“Ceramide glucosyltransferase inhibitor” CHEMBL1086997

Modality Small molecule “Lucerastat, an iminosugar with potential as substrate reduction therapy for glycolipid storage disorders” PMID 28088251 Jan 2017
Route Oral “Ten subjects with Fabry Disease received 1000 mg of oral lucerastat twice daily for 12 weeks” NCT02930655
Target UGCG “Ceramide glucosyltransferase inhibitor” CHEMBL1086997