Trials Annotator

Trial NCT03523728

Org. Study IDs: EFC15392
Secondary IDs: 2017-004084-12 U1111-1202-0775

Last trial update was posted on 2023-02-03

MeSH Interventions

Venglustat

MeSH Conditions

Polycystic Kidney Diseases Polycystic Kidney, Autosomal Dominant

Other Conditions

Stopping Reasons

Interim analysis for futility of the Stage 1 of the EFC15392 study met the protocol specified stopping rule based on the primary endpoint. EFC15392 study was stopped for futility based on prespecified criteria and recommendation from DMC

Limitations And Caveats

Following decision to discontinue EFC15392 based on the futility analysis, the two-steps analysis initially planned were not applicable, therefore only the final analysis of all safety endpoints (laboratory, vital sign, ECG, physical examination, BDI-II, and lens opacity) were performed on extended combined Stage 1 and Stage 2 safety population only.

Result Publications

Gansevoort RT, Hariri A, Minini P, Ahn C, Chapman AB, Horie S, Knebelmann B, Mrug M, Ong ACM, Pei YPC, Torres VE, Modur V, Antonshchuk I, Perrone RD. Venglustat, a Novel Glucosylceramide Synthase Inhibitor, in Patients at Risk of Rapidly Progressing ADPKD: Primary Results of a Double-Blind, Placebo-Controlled, Phase 2/3 Randomized Clinical Trial. Am J Kidney Dis. 2023 May;81(5):517-527.e1. doi: 10.1053/j.ajkd.2022.10.016. Epub 2022 Dec 17. [PMID: 36535535] [DOI: 10.1053/j.ajkd.2022.10.016]

Annotations

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Intervention Annotations

#	Outdated	Intervention				Condition
#	Outdated	Name		Route	DrugBank ID	Condition