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@article{Wong:2023:10.1016/j.ekir.2023.09.017,
author = {Wong, E and Nester, C and Cavero, T and Karras, A and Le, Quintrec M and Lightstone, L and Eisenberger, U and Soler, MJ and Kavanagh, D and Daina, E and Praga, M and Medjeral-Thomas, NR and Gaeckler, A and Garcia-Carro, C and Biondani, A and Chaperon, F and Kulmatycki, K and Milojevic, J and Webb, NJA and Nidamarthy, PK and Junge, G and Remuzzi, G},
doi = {10.1016/j.ekir.2023.09.017},
journal = {Kidney International Reports},
pages = {2754--2764},
title = {Efficacy and safety of iptacopan in patients with C3 glomerulopathy},
url = {http://dx.doi.org/10.1016/j.ekir.2023.09.017},
volume = {8},
year = {2023}
}
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TY  - JOUR
AB  - IntroductionComplement 3 glomerulopathy (C3G) is a rare inflammatory kidney disease mediated by dysregulation of the alternative complement pathway. No targeted therapy exists for this aggressive glomerulonephritis. Efficacy, safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) (measured by complement biomarkers) of iptacopan were assessed in patients with C3G.MethodsIn this phase 2, multicenter, open-label, single-arm, nonrandomized study, adults with biopsy-proven, native kidney C3G (native cohort) and kidney transplant recipients with C3G recurrence (recurrent kidney transplant [KT] cohort) received iptacopan twice daily (bid) for 84 days (days 1–21: 10–100 mg; days 22–84: 200 mg). The primary end point was the urine protein-to-creatinine ratio (UPCR; native cohort) and the change in the C3 deposit score of kidney biopsy (recurrent KT cohort). The complement pathway measures included Wieslab assay, soluble C5b9, and serum C3 levels.ResultsA total of 27 patients (16 native cohort and 11 recurrent KT cohort) were enrolled and all completed the study. In the native cohort, UPCR levels decreased by 45% from baseline to week 12 (P = 0.0003). In the recurrent KT cohort, the median C3 deposit score decreased by 2.50 (scale: 0–12) on day 84 versus baseline (P = 0.03). Serum C3 levels were normalized in most patients; complement hyperactivity observed pretreatment was reduced. Severe adverse events (AEs) included post-biopsy hematuria and hyperkalemia. No deaths occurred during the study.ConclusionIptacopan resulted in statistically significant and clinically important reductions in UPCR and normalization of serum C3 levels in the native cohort and reduced C3 deposit scores in the recurrent KT cohort with favorable safety and tolerability. (ClinicalTrials.gov identifier: NCT03832114).
AU  - Wong,E
AU  - Nester,C
AU  - Cavero,T
AU  - Karras,A
AU  - Le,Quintrec M
AU  - Lightstone,L
AU  - Eisenberger,U
AU  - Soler,MJ
AU  - Kavanagh,D
AU  - Daina,E
AU  - Praga,M
AU  - Medjeral-Thomas,NR
AU  - Gaeckler,A
AU  - Garcia-Carro,C
AU  - Biondani,A
AU  - Chaperon,F
AU  - Kulmatycki,K
AU  - Milojevic,J
AU  - Webb,NJA
AU  - Nidamarthy,PK
AU  - Junge,G
AU  - Remuzzi,G
DO  - 10.1016/j.ekir.2023.09.017
EP  - 2764
PY  - 2023///
SN  - 2468-0249
SP  - 2754
TI  - Efficacy and safety of iptacopan in patients with C3 glomerulopathy
T2  - Kidney International Reports
UR  - http://dx.doi.org/10.1016/j.ekir.2023.09.017
UR  - https://www.sciencedirect.com/science/article/pii/S2468024923015103?via%3Dihub
VL  - 8
ER  -
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