Abstract: INFO38
Phase 3, Randomized, Multicenter Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Treatment of C3G or IC-MPGN
Session Information
- Informational Posters
November 04, 2021 | Location: On-Demand, Virtual Only
Abstract Time: 10:00 AM - 12:00 PM
Category: Glomerular Diseases
- No subcategory defined
Authors
- Pickering, Matthew C., Imperial College London, London, London, United Kingdom
- Dixon, Bradley P., University of Colorado School of Medicine, Aurora, Colorado, United States
- Fakhouri, Fadi, Lausanne University Hospital and University of Lausanne, Lausanne, Switzerland
- Cook, H. Terence, Imperial College London, London, London, United Kingdom
- Kavanagh, David, National Renal Complement Therapeutics Centre, Newcastle upon Tyne, United Kingdom
- Remuzzi, Giuseppe, Istituto di Ricerche Farmacologiche Mario Negri, Milano, Lombardia, Italy
- Walker, Patrick D., Arkana Laboratories, Little Rock, Arkansas, United States
- Licht, Christoph, The Hospital for Sick Children, Toronto, Ontario, Canada
- Appel, Gerald B., Columbia University, New York, New York, United States
- Vivarelli, Marina, Division of Nephrology and Dialysis, Bambino Gesù Children’s Hospital, IRCCS, Rome, Italy
- Zhang, Zhiqun, Former Employee of Apellis Pharmaceuticals, Waltham, Massachusetts, United States
- Kocinsky, Hetal S., Apellis Pharmaceuticals, Waltham, Massachusetts, United States
Description
Objectives: Complement 3 glomerulopathy (C3G) and immune complex membranoproliferative glomerulonephritis (IC-MPGN) are rare diseases characterized by excessive deposition of C3 breakdown products in renal glomeruli leading to proteinuria and progressive renal disease. Pegcetacoplan is a targeted C3 investigational therapy for diseases related to complement overactivation. This is a phase 3, randomized, placebo-controlled, double-blind, multicenter study of the efficacy and safety of pegcetacoplan in individuals with C3G or IC-MPGN.
Methods: Approximately 90 patients (age, ≥12 years; weight, 20-100 kg) diagnosed with C3G or IC-MPGN, either as primary disease or posttransplant disease recurrence, will be recruited. Inclusion criteria include 2+ staining for C3c, global glomerulosclerosis <50%, urine protein-to-creatinine ratio (uPCR) ≥1000 mg/g, and estimated glomerular filtration rate (eGFR) >30 mL/min/1.73 m2. Exclusion criteria include previous pegcetacoplan exposure, C3G/IC-MPGN secondary to other conditions, and significant infection/malignancy. Patients will be randomized 1:1 to receive subcutaneous infusions of pegcetacoplan (1080 mg/20 mL) or matching volume of placebo twice weekly for 26 weeks (in addition to standard care). Thereafter, in the open-label period, all participants will receive pegcetacoplan twice weekly for another 26 weeks. Assessments include first-morning uPCR every 4 weeks and renal biopsies at baseline/screening and week 26. Primary endpoints include proportion of participants with reduction in uPCR ≥50% relative to baseline at week 26. Secondary endpoints include change in eGFR relative to baseline at weeks 26 and 52, proportion of participants with decreased C3c staining from baseline at week 26, and change in C3G histologic index activity score from baseline at week 26. Safety outcomes will also be monitored throughout the study. Participants may enter a subsequent long-term extension study or an 8-week follow-up period.
Results, Discussion, and Conclusion: This is a study design abstract.