Alternative Complement Pathway Inhibition With Iptacopan for the Treatment of C3 Glomerulopathy-Study Design of the APPEAR-C3G Trial.
C3G
LNP023
alternative pathway
clinical trials
complement system
factor B
iptacopan
Journal
Kidney international reports
ISSN: 2468-0249
Titre abrégé: Kidney Int Rep
Pays: United States
ID NLM: 101684752
Informations de publication
Date de publication:
Oct 2022
Oct 2022
Historique:
received:
23
03
2022
revised:
15
06
2022
accepted:
04
07
2022
entrez:
11
10
2022
pubmed:
12
10
2022
medline:
12
10
2022
Statut:
epublish
Résumé
Complement 3 glomerulopathy (C3G) is a rare kidney disease characterized by dysregulation of the alternative pathway (AP) of the complement system. About 50% of patients with C3G progress to kidney failure within 10 years of diagnosis. Currently, there are no approved therapeutic agents for C3G. Iptacopan is an oral, first-in-class, potent, and selective inhibitor of factor B, a key component of the AP. In a Phase II study, treatment with iptacopan was associated with a reduction in proteinuria and C3 deposit scores in C3G patients with native and transplanted kidneys, respectively. APPEAR-C3G (NCT04817618) is a randomized, double-blind, and placebo-controlled Phase III study to evaluate the efficacy and safety of iptacopan in C3G patients, enrolling 68 adults with biopsy-confirmed C3G, reduced C3 (<77 mg/dl), proteinuria ≥1.0 g/g, and estimated glomerular filtration rate (eGFR) ≥30 ml/min per 1.73 m This study aims to demonstrate the clinical benefits of AP inhibition with iptacopan in C3G.
Identifiants
pubmed: 36217526
doi: 10.1016/j.ekir.2022.07.004
pii: S2468-0249(22)01502-9
pmc: PMC9546729
doi:
Types de publication
Journal Article
Langues
eng
Pagination
2150-2159Informations de copyright
© 2022 International Society of Nephrology. Published by Elsevier Inc.
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