STAGED-PKD
Research type
Research Study
Full title
Multicenter, randomized, double-blind, placebo-controlled two stage study to characterize the efficacy, safety, tolerability and pharmacokinetics of GZ/SAR402671 in patients at risk of rapidly progressive Autosomal Dominant Polycystic Kidney Disease (ADPKD)
IRAS ID
245567
Contact name
Albert Chee Meng Ong
Contact email
Sponsor organisation
Genzyme Corporation
Eudract number
2017-004084-12
ISRCTN Number
ISRCTN00000000
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
U1111-1202-0775, WHO universal trial number
Duration of Study in the UK
4 years, 2 months, 17 days
Research summary
Summary of Research
Autosomal dominant polycystic kidney disease (ADPKD) is a life-threatening genetic disease caused by mutations in PKD1 and PKD2 genes characterized by the formation of multiple kidney cysts that enlarge with disease progression to result in end-stage renal disease and dialysis in ~50% of patients.
GZ/SAR402671 is a glucosylceramide synthase (GCS) inhibitor that decreases the synthesis of glucosylceramide (GL-1), a central building block for more complex glycosphingolipids (GSLs).
Human and murine ADPKD is associated with increased GCS activity, leading to a pathogenic accumulation of GSLs such as GL-1, lactosylceramide (GL-2)and GM3. Several structurally distinct GCS inhibitors have significantly reduced cyst growth and preserved renal function in 3 different preclinical ADPKD models.
This is a multicentre, randomized, double-blind, placebo-controlled two stage study to characterize the efficacy, safety, tolerability and pharmacokinetics of GZ/SAR402671 in patients at risk of rapidly progressive ADPKD.
Primary objective is to determine the effect of GZ/SAR402671 on the rate of total kidney volume (TKV) growth in patients at risk of rapidly progressive ADPKD and to determine the effect of GZ/SAR402671 on rate of renal function (eGFR) decline as compared to placebo in patients at risk of rapidly progressive ADPKD.
REC name
North West - Greater Manchester Central Research Ethics Committee
REC reference
18/NW/0792
Date of REC Opinion
28 Jan 2019
REC opinion
Further Information Favourable Opinion