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Sanofi begins global clinical trial to evaluate therapy for patients at risk of rapidly progressing ADPKD

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Sanofi has begun a pivotal global clinical trial to study the safety, efficacy, and tolerability of a drug called venglustat in ADPKD and is enrolling patients who are at risk of rapidly progressive ADPKD.

Drug targets cyst growth factors

ADPKD is caused by a mutation in the PKD1 or PKD2 gene that leads to a build-up of complex substances called glycosphingolipids in the kidneys. Glycosphingolipid accumulation is thought to be an important driver of cyst growth [1] [2]. 

Venglustat is designed to inhibit the abnormal accumulation of a substance in the body called glucosylceramide (GL-1), which has a role in production of glycosphingolipids. In genetic mouse models of ADPKD, inhibition of glycosphingolipid production has been shown to reduce kidney cyst growth [3].

If you are interested in taking part

In the UK, patients will be enrolled for the trial at the following hospital. You don't have to be a patient at this hospital to take part. Just ask your kidney doctor.

  • Sheffield Northern General - Professor Albert Ong is the Principal Investigator. Contact either This email address is being protected from spambots. You need JavaScript enabled to view it. or This email address is being protected from spambots. You need JavaScript enabled to view it..

For more information on this trial, please visit Clinical Trials.gov


  1. Chatterjee S, Shi WY, Wilson P, Mazumdar A. Role of lactosylceramide and MAP kinase in the proliferation of proximal tubular cells in human polycystic kidney disease. J Lipid Res. 1996;37(6):1334-44
  2. Deshmukh GD, Radin NS, Gattone VH, 2nd, Shayman JA. Abnormalities of glycosphingolipid, sulfatide, and ceramide in the polycystic (cpk/cpk) mouse. J Lipid Res. 1994;35(9):1611-8.
  3. Natoli TA, Smith LA, Rogers KA, et al. Inhibition of glucosylceramide accumulation results in effective blockade of polycystic