Familial nephrotic syndrome

This was a planned early-stage trial to test whether anti-CD19 CAR-T cells could safely help children with multi-drug resistant nephrotic syndrome, a serious kidney condition. The study was withdrawn before any patients were enrolled, so no data on safety or effectiveness were co…

This study tests the long-term safety and effectiveness of pegcetacoplan in 100 people with two rare kidney diseases, C3 glomerulopathy and IC-MPGN. Participants previously completed a one-year trial and saw benefit from the drug. The goal is to see if pegcetacoplan can keep redu…

This study tests whether a daily dose of alpha lipoic acid, an antioxidant supplement, can lower the risk of heart problems in 50 children who are on regular hemodialysis. Participants will either take the supplement or a placebo pill for a period, and researchers will monitor fo…

This program provides early access to pegcetacoplan for people aged 12 and older with C3 glomerulopathy or primary IC-MPGN, rare kidney diseases that can lead to kidney failure. Participants must have active disease with significant protein in their urine. The goal is to offer a …

This study tests whether dapagliflozin, a drug used in adults for diabetes and kidney protection, can reduce protein leakage in the urine of children with chronic kidney disease. Ten children aged 4 to 18 with persistent proteinuria despite standard therapy will take the drug, an…

This study gathers MRI, CT, and ultrasound images from 300 adults—some healthy, some with kidney or brain disease—to build a collection for future not-for-profit research. No new treatments are being tested; the goal is to make medical images available to scientists for advancing…