Sanofi begins global clinical trial to evaluate therapy for patients at risk of rapidly progressing ADPKD
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  .
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 .
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.
For more information on this trial, please visit Clinical Trials.gov
- 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
- 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.
- Natoli TA, Smith LA, Rogers KA, et al. Inhibition of glucosylceramide accumulation results in effective blockade of polycystic