STAGED-PKD-EXT
Research type
Research Study
Full title
Multicenter, open-label, extension study to characterize the long-term efficacy and safety of early versus delayed treatment with venglustat (GZ/SAR402671) in patients at risk of rapidly progressive autosomal dominant polycystic kidney disease (ADPKD)
IRAS ID
293267
Contact name
Catherine Anselmino
Contact email
Sponsor organisation
Sanofi-aventis Recherche & Développement
Eudract number
2020-004400-34
Clinicaltrials.gov Identifier
130913, IND number
Duration of Study in the UK
4 years, 5 months, 5 days
Research summary
Summary of Research
This is a open-label extension study enrolling adult patients at risk of rapidly progressive ADPKD (Autosomal dominant polycystic kidney disease) who have previously completed Stage 1 or Stage 2 of Study EFC15392.
ADPKD is a life-threatening genetic disease caused by mutations in PKD1 and PKD2 genes characterised 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.
Venglustat (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. These results support the hypothesis that GCS inhibition using venglustat could be a viable treatment in individuals with ADPKD.
The purpose of this study is to obtain long-term efficacy and safety data, up to 4 years, with venglustat administration. The primary objective is 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.
This study will be conducted globally and at one centre in the UK.
Summary of Results
Lay summary available at https://eur03.safelinks.protection.outlook.com/?url=https%3A%2F%2Fu2790089.ct.sendgrid.net%2Fls%2Fclick%3Fupn%3DXv3JSvJ-2B3M71ppf7N9agbfd6HtMZuEBXrupKun9DZUA4-2FnVVNehPn8N2Lx0Nc8M4HFHQ_E1aO2-2BZlVOSJJV-2FajQqskegTd6IRomHYTi-2Fbt8SH3YIRyZFiNEQlbvYZW72Xrp-2Bb-2B8GDYx1uaemQVS4uWgpUb6J5u3lHJ2HPA2D59dZS3QhmyjB1q4nBGlZcPHkCk15wZg-2FB9GQ9F0zZSQ4VbPXtxGPTD8-2B2YVOQW0iFqZZAmh5jikRJrkuDdP-2F9IXjhdGVolrdWa7cMG-2BQYBTkRrf4jyQ-3D-3D&data=05%7C01%7Capprovals%40hra.nhs.uk%7Cf529de6c20fb490b984708db0e79e767%7C8e1f0acad87d4f20939e36243d574267%7C0%7C0%7C638119688951589032%7CUnknown%7CTWFpbGZsb3d8eyJWIjoiMC4wLjAwMDAiLCJQIjoiV2luMzIiLCJBTiI6Ik1haWwiLCJXVCI6Mn0%3D%7C3000%7C%7C%7C&sdata=wYae00sGmP01jWHIXpScy0oN8%2FOn5CBrCU9t5XMBw8g%3D&reserved=0 & searching for LTS15823
REC name
North West - Greater Manchester Central Research Ethics Committee
REC reference
21/NW/0044
Date of REC Opinion
8 Apr 2021
REC opinion
Further Information Favourable Opinion