Abstract
Efficacy and Safety of Iptacopan in Patients with C3 Glomerulopathy: C3G Extension Trial Interim Results from the Phase 3 APPEAR-C3G Patients: FR-PO0838
Journal of the American Society of Nephrology, Vol.36(10S)
10/2025
DOI: 10.1681/ASN.202599z9n79j
Abstract
Background:
The Phase 3 APPEAR-C3G study evaluated the efficacy, safety, and tolerability of iptacopan vs placebo in C3G patients (pts).
Methods:
APPEAR-C3G (NCT04817618) was a multicenter, randomized, double-blind, placebo-controlled, pivotal Phase 3 study that included adult pts with biopsy-confirmed C3G. Upon completion of study treatment at 12m in APPEAR-C3G, participants had the option to continue open-label iptacopan treatment in the C3G extension study (NCT03955445). Here we present the results from the extension study with iptacopan treatment up to 24m.
Results:
Of 73 pts completing the 12m treatment, 66 pts entered the extension study. In the iptacopan arm, the geometric mean reduction in first morning void (FMV) UPCR at Month 12 (35.9%), was sustained up to Month 24 (34.2%) compared to baseline (Figure). In the placebo-iptacopan switch arm, the reduction at Month 12 (34.0%) was also sustained up to Month 24 (29.7%). The mean pre-treatment annualized eGFR slope (95%CI) in all pts (n=74) showed rapid decline with −7.22 mL/min/1.73m2/year (−10.10, −4.35). After initiation of iptacopan treatment, there was stabilization of annualized eGFR slope (95%CI) at −0.29 mL/min/1.73m2/year (−3.88, 3.31) assessed over 24 months. Iptacopan demonstrated a favorable safety profile with no new safety signals identified.
Conclusion:
Iptacopan demonstrated sustained reduction in proteinuria and stabilization of eGFR slope with treatment up to 24 months among APPEAR-C3G pts who rolled over to the long-term extension study. Iptacopan was well tolerated with a favorable safety profile in C3G pts.
Details
- Title: Subtitle
- Efficacy and Safety of Iptacopan in Patients with C3 Glomerulopathy: C3G Extension Trial Interim Results from the Phase 3 APPEAR-C3G Patients: FR-PO0838
- Creators
- Carla M. NesterRichard J. SmithDavid KavanaghMarina VivarelliGiuseppe RemuzziMing-Hui ZhaoEdwin Kwan Soon WongYaqin WangRubeen K. IsraniHari Varun KalluriInduja KrishnanJean GrisouardAndrew S. Bomback
- Resource Type
- Abstract
- Publication Details
- Journal of the American Society of Nephrology, Vol.36(10S)
- DOI
- 10.1681/ASN.202599z9n79j
- ISSN
- 1046-6673
- eISSN
- 1533-3450
- Publisher
- American Society of Nephrology
- Language
- English
- Date published
- 10/2025
- Academic Unit
- Roy J. Carver Department of Biomedical Engineering; Molecular Physiology and Biophysics; Anatomy and Cell Biology; Nephrology, Dialysis and Transplantation; Stead Family Department of Pediatrics; Iowa Neuroscience Institute; Otolaryngology; Internal Medicine
- Record Identifier
- 9985019028202771
Metrics
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