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Abstract: TH-PO721

Compassionate Use of Pegcetacoplan in a Patient with C3 Glomerulopathy: A Case Report

Session Information

Category: Glomerular Diseases

  • 1402 Glomerular Diseases: Clinical, Outcomes, and Trials


  • Noor, Sahibzadi, Loma Linda University, Loma Linda, California, United States
  • Mathew, Roy O., VA Loma Linda Healthcare System, Loma Linda, California, United States
  • Abdi Pour, Amir, Loma Linda University, Loma Linda, California, United States
  • Norouzi, Sayna, Loma Linda University, Loma Linda, California, United States

Pegcetacoplan is a C3 complement inhibitor which is approved by the United States Food and Drug Administration for paroxysmal nocturnal hemoglobinuria and geographic atrophy secondary to macular degeneration. It is undergoing a phase III trial (Valiant trial, NCT05067127) which is assessing subcutaneous pegcetacoplan in patients with C3 glomerulopathy (C3G) or immune-complex mediated glomerulonephritis. Here, we discuss the case of a patient who did not qualify for the trial but was approved for compassionate use of pegcetacoplan.

Case Description

A 26-year-old female was diagnosed with biopsy proven C3G with negative genetic testing. Over the course of about one and a half year, her renal function had deteriorated from estimated glomerular filtration rate (eGFR) of 64 to 22 ml/min/m2 , with proteinuria progressing from 1.5 to 2.7 gm/day, while being on prednisone and mycophenolic acid (MFA). Due to the inability to tolerate MFA (MFA induced colitis on colon biopsy), she was transitioned to tacrolimus and prednisone. The patient kept being hospitalized frequently due to acute kidney injuries (AKI), with five admissions over four months. Patient was not a candidate for the Valiant trial due to eGFR < 30 ml/min/m2, 76% glomerular sclerosis (GS) and 60% interstitial fibrosis and tubular atrophy (IFTA) on the last biopsy. Therefore, a request for compassionate use of pegcetacoplan was submitted. It was approved after four months and started in September 2022. While being on pegcetacoplan for six months, the patient's renal function remained stable (eGFR 19-22 ml/min/m2, proteinuria 0.5-0.7 gm/day) and she did not require any hospitalizations. The medication was well tolerated without any serious adverse effects. In March 2023, the patient progressed to end stage renal disease (ESRD) and was started on hemodialysis.


There is currently no approved therapy for C3G. Our patient had frequent episodes of AKI, multiple hospitalizations and did not meet the criteria for Valiant trial due to her low GFR. We believe pegcetacoplan might have delayed her progression to ESRD, improved her quality of life by avoiding hospitalizations and allowed her time to get listed for transplant. Further studies are needed to evaluate the efficacy and safety of pegcetacoplan in advanced C3G.