This Plain Language Summary of Publication article (PLSP) from Future Rare Diseases describes the DISCOVERY study, a phase 2 clinical trial evaluating pegcetacoplan in patients with complement 3 glomerulopathy (C3G), a rare kidney disease caused by an overactive complement cascade that leads to C3 protein fragments accumulating in and damaging the kidneys. The study included participants aged 16+ with C3G who hadn’t responded to standard treatments, administering pegcetacoplan for 48 weeks alongside their existing medications. Researchers assessed the drug’s effectiveness by comparing pre-treatment and post-treatment measurements of proteinuria (protein in urine), kidney function, and blood C3 levels, while also monitoring for side effects. Success indicators included decreased proteinuria, stabilized kidney function, and increased blood C3 levels.

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This PLSP is based on an article called ‘Clinical Safety and Efficacy of Pegcetacoplan in a Phase 2 Study of Patients with C3 Glomerulopathy and Other Complement-Mediated Glomerular Diseases’ and was published in the Kidney International Reports.

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