Evaluate the Safety, Pharmacodynamics, Pharmacokinetics, and Exploratory Efficacy of GZ/SAR402671 in Treatment-naïve Adult Male Patients With Fabry Disease

Phase 2 Completed 11 enrolled Genzyme, a Sanofi Company

NaSingle-groupOpen-labelTreatment

Summary

Primary Objective: To assess the safety, pharmacokinetics (PK), pharmacodynamics (PD), and exploratory efficacy of GZ/SAR402671 in enzyme replacement therapy treatment-naïve adult male participants diagnosed with Fabry disease.

Timeline

Start: 2014-11
Primary completion: 2016-09
Completion: 2016-09

Outcome

Missed primary endpoint

registry analysis (other test); GZ/SAR402671; p = 0.3173; McNemar Test; note: Threshold for significance at 0.05 level. NCT02228460 ↗

registry analysis (other test); GZ/SAR402671; p = 0.625; Wilcoxon signed rank test; note: Threshold for significance at 0.05 level. NCT02228460 ↗

Publications

Deegan PB, Goker-Alpan O, Geberhiwot T, Hopkin RJ, Lukina E, Tylki-Szymanska A, Zaher A, Sensinger C, Gaemers SJM, Modur V, Thurberg BL, Sharma J, Najafian B, Mauer M, DasMahapatra P, Wilcox WR, Germain DP. Venglustat, an orally administered glucosylceramide synthase inhibitor: Assessment over 3 years in adult males with classic Fabry disease in an open-label phase 2 study and its extension study. Mol Genet Metab. 2023 Feb;138(2):106963. doi: 10.1016/j.ymgme.2022.11.002. Epub 2022 Nov 9.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Venglustat	Small molecule	15 mg	Oral