Abstract: TH-PO1262
Efficacy and Safety of Eculizumab in Patients with Anti-Glomerular Basement Membrane Disease: An Investigator-Initiated, Open-Label, Phase 2 Clinical Trial
Session Information
- Late-Breaking Research Posters
October 22, 2026 | Location: Exhibit Hall A, Convention Center
Abstract Time: 10:00 AM - 12:00 PM
Category: Glomerular Diseases
- 1402 Glomerular Diseases: Clinical, Outcomes, and Therapeutics
Authors
- Kuang, Huang, Peking University First Hospital, Beijing, China
- Jia, Xiaoyu, Peking University First Hospital, Beijing, China
- Cui, Zhao, Peking University First Hospital, Beijing, China
- Zhao, Minghui, Peking University First Hospital, Beijing, China
Background
Anti-glomerular basement membrane disease (GBM) is the most severe type of autoimmune kidney diseases, and is associated with the overactivation of the complement cascade. We aimed to evaluate the efficacy and safety of eculizumab, a complement C5 blocker that targets terminal pathways, in patients with anti-GBM disease.
Methods
An investigator-initiated, open-label, phase II clinical trial was conducted (NCT06513338). Eligible participants were aged at least 18 years diagnosed with anti-GBM disease at our center. Eculizumab was given at a dose of 900 mg for 4 consecutive weeks, followed by 1200 mg every other week for the subsequent 8 weeks. All patients received standard of care of the disease. The primary endpoint was end-stage kidney disease (ESKD) events at 6 months. In parallel, patients with anti-GBM disease diagnosed at the same center during the same period who did not receive eculizumab and were not enrolled in the trial were identified as a contemporaneous control cohort.
Results
Between March 2024 and April 2025, a total of 16 patients were enrolled to receive eculizumab treatment, along with 40 contemporaneous controls. ESKD events were 25.0% at 6 months following eculizumab treatment, compared to that of 52.5% in contemporaneous controls. Of 7 patients requiring initial dialysis, 3 (42.9%) achieved kidney function recovery, compared to that of 8.7% in contemporaneous controls. Adverse events were reported in 2 participants and others showed well tolerance to eculizumab administration during the trial.
Conclusion
Eculizumab treatment showed improvement in kidney survival and kidney function recovery in addition to standard of care. Eculizumab was well tolerated with an acceptable safety profile in patients with anti-GBM disease.