A Sequential Phase 2/3, Single-Arm, Open-Label Study in Adults Followed by a Randomized, Placebo-Controlled, Double-Blind, Multicenter Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Adults and Adolescents with Focal Segmental Glomerulosclerosis

Focal segmental glomerulosclerosis, often shortened to FSGS, is a serious kidney condition. Your kidneys contain millions of tiny filtering units that clean your blood. In FSGS, some of these filters become scarred and damaged. When this happens, proteins that your body needs slip through the filters and pass into your urine. Over time, this leakage can cause swelling in your legs and face, high blood pressure, and permanent kidney damage.

Pegcetacoplan is a medicine designed to calm down an overactive part of the body's immune system called the complement system. In some kidney diseases, this immune pathway accidentally attacks the kidneys and causes inflammation. By targeting this pathway, doctors hope pegcetacoplan can reduce inflammation, protect the filters, and stop proteins from leaking out.

This trial is divided into two sequential parts. In the initial phase, participants receive pegcetacoplan so researchers can evaluate its safety and early effects. In the next phase, participants are randomly assigned to receive either pegcetacoplan or a placebo (an inactive dummy treatment) without knowing which one they have received. This gold-standard approach helps scientists be completely certain whether any health improvements are due to the medicine itself.

Finding new treatments for FSGS is vital. Many patients currently have to rely on steroid medicines, which do not always work and can cause difficult side effects. If pegcetacoplan proves effective, it could offer a valuable new tool to help protect kidney function and delay or prevent the need for dialysis or kidney transplants.