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
01
Male and female participants age ≥ 12 and ≤ 60 years at screening.
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03
Maximally recommended or tolerated dose of ACEI or ARB for at least 90 days. Stable doses of anti-proteinuric medications like MMF and Cortocosteroids.
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UPCR ≥ 1.0 g/g sampled from the first morning void urine sample at Day -75 and Day -15.
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Mandatory vaccination against Neisseria meningitidis and Streptococcus pneumoniae prior to the start of study treatment.
02
Diagnosis of C3G as confirmed by renal biopsy within 12 months prior to enrollment in adults and within 3 years in adolescents.
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​Reduced serum C3 at screening.
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06
Estimate GFR or measured GFR ≥ 30 ml/min/1.73m2