Long-term Treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD) With Venglustat

  • End date
    Nov 7, 2025
  • participants needed
  • sponsor
Updated on 23 June 2021


Primary Objective:

-To determine the effect of early versus delayed treatment with venglustat on the total kidney volume (TKV) in participants at risk of rapidly progressive ADPKD.

Secondary Objective:

  • To determine the effect of early versus delayed treatment with venglustat on the renal function (estimated glomerular filtration rate [eGFR]).
  • To characterize the safety profile of venglustat.
  • To evaluate the effect of venglustat on the lens by ophthalmological examination.
  • To evaluate the effect of venglustat on mood using Beck Depression Inventory-II (BDI-II).


Study duration per participant is 25.5 months (maximal). Screening period (when applicable): up to 2 weeks. Core treatment period: 24 months. Follow-up: 30 days after final dose of the investigational medicinal product (IMP) (venglustat).

Condition Congenital Cystic Kidney Disease
Treatment Venglustat GZ402671
Clinical Study IdentifierNCT04705051
Last Modified on23 June 2021

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