IPTACOPAN
Excessive local activation and dysregulation of the alternative complement pathway in the glomeruli can cause excessive deposition of multiple complement components in the glomerulus. Iptacopan (LNP023) is a potent oral highly selective inhibitor of factor B of alternative complement pathway.
Enroll in this clinical study
How is Iptacopan being studied in C3GN?
In a phase 2 study, treatment with Iptacopan was associated with a statistically significant reduction in proteinuria and stabilization of eGFR in patients with C3G.
APPEAR-C3G is a randomized, double-blind, placebo-controlled pivotal Phase 3 study to evaluate the efficacy and safety of iptacopan in patients with native kidney C3G. 83 patients will be randomized 1:1 to receive either iptacopan 200 mg bid or placebo for 6 months, followed by open-label treatment with iptacopan 200 mg bid for all patients for 6 months. The primary objective is to demonstrate the superiority of iptacopan versus placebo on proteinuria reduction at 6 and 12 months.
Image from Smith et al, Kidney week 2021
Eligibility Criteria
Male and female participants age ≥ 12 and ≤ 60 years at screening.
Diagnosis of C3G as confirmed by renal biopsy within 12 months prior to enrollment in adults and within 3 years in adolescents.
Maximally recommended or tolerated dose of ACEI or ARB for at least 90 days. Stable doses of anti-proteinuric medications like MMF and Cortocosteroids.
Reduced serum C3 at screening.
UPCR ≥ 1.0 g/g sampled from the first morning void urine sample at Day -75 and Day -15.
Estimate GFR or measured GFR ≥ 30 ml/min/1.73m2
Mandatory vaccination against Neisseria meningitidis and Streptococcus pneumoniae prior to the start of study treatment.