Focal segmental glomerulosclerosis is a rare kidney condition in which small parts of the filtering units become scarred, leading to leakage of protein into the urine. This leakage, called proteinuria, can be measured by the amount of protein relative to creatinine in a urine sample. The study evaluates the investigational drug pegcetacoplan, which is given by a subcutaneous injection, meaning it is administered under the skin.
The purpose of the study is to determine whether twice‑weekly dosing of the drug can lower protein loss in the urine compared with placebo. Participants will first receive the active medication in an open‑label phase, followed by a randomized, double‑blind phase where some receive the drug and others receive the placebo, with regular monitoring of kidney function and safety.
Throughout the trial, participants will have periodic clinic visits where urine samples are collected to calculate the urine protein to creatinine ratio and the urine albumin to creatinine ratio, both of which indicate the level of protein leakage. Blood tests will also be performed to assess the estimated Glomerular Filtration Rate, a standard measure of how well the kidneys are filtering waste. The study continues over several months to observe changes in these measurements and to ensure the treatment is safe.
Germany 
Italy 
Portugal 
Slovakia 
Spain 
