* Safety and Efficacy of LNP023 in C3G patients (Adults)
Research type
Research Study
Full title
A multicenter, randomized, double-blind, parallel group, placebo-controlled study to evaluate the efficacy and safety of iptacopan (LNP023) in complement 3 glomerulopathy
IRAS ID
290279
Contact name
Edwin Wong
Contact email
Sponsor organisation
Novartis Pharma AG
Eudract number
2020-004589-21
Clinicaltrials.gov Identifier
Duration of Study in the UK
2 years, 1 months, 27 days
Research summary
C3 glomerulopathy (C3G) is an ultra-rare, severe disorder of excessive activation of the alternative complement pathway (AP) resulting in approximately 50% of patients progressing to kidney failure within 10 years of diagnosis. With no approved therapies, C3G carries one of the highest risks for irreversible renal failure of all primary glomerular diseases.
iptacopan (LNP023) is a drug that blocks key steps in the “alternative complement pathway”, which is known to be activated in the kidney of most patients with C3G, and as such may improve C3G.
The purpose of the study is to find out if LNP023 is safe; can reduce renal inflammation, proteinuria, and improve renal function in patients with C3G.
The study will run for approx. 2 years with 6 patients to be enrolled across 3 sites. Participants will receive either active drug or placebo for 6 months and then be provided with open label active drug for another 6 months, and will visit the study doctor 12 times over 12 months for assessments and monitoring of their condition as part of the study.
REC name
South Central - Hampshire B Research Ethics Committee
REC reference
22/SC/0321
Date of REC Opinion
3 Jun 2021
REC opinion
Further Information Favourable Opinion