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Abstract: INFO12-SA

Phase 3, Randomized, Multicenter Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Treatment of C3 Glomerulopathy (C3G) or Immune Complete Membranoproliferative Glomerulonephritis (IC-MPGN)

Session Information

  • Informational Posters - III
    November 04, 2023 | Location: Exhibit Hall, Pennsylvania Convention Center
    Abstract Time: 10:00 AM - 12:00 PM

Category: Glomerular Diseases

  • No subcategory defined

Authors

  • Dixon, Bradley P., University of Colorado Anschutz Medical Campus School of Medicine, Aurora, Colorado, United States
  • Pickering, Matthew C., Imperial College London, London, London, United Kingdom
  • Fakhouri, Fadi, Universite de Lausanne, Lausanne, Vaud, Switzerland
  • Cook, H. Terence, University of Colorado Anschutz Medical Campus School of Medicine, Aurora, Colorado, United States
  • 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, Bambino Gesù Pediatric Hospital, Rome, Italy
  • Li, Li, Apellis Pharmaceuticals Inc, Waltham, Massachusetts, United States

Group or Team Name

  • on behalf of the Valiant study investigators
Description

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 C3 investigational therapy for diseases related to complement overactivation. This is a phase 3, randomized, placebo-controlled, double-blind, multicenter study on efficacy and safety of pegcetacoplan in patients with C3G or IC-MPGN. Approximately 80 to 100 patients (aged ≥12 years; weight 30–100 kg) diagnosed with C3G or IC-MPGN as primary disease or post-transplant 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. Patients will be randomized 1:1 (double blind) to receive subcutaneous infusions of pegcetacoplan (1080 mg/20 mL) or placebo (matching volume) twice weekly for 26 weeks, in addition to standard care. Thereafter, in the open-label period, all patients will receive pegcetacoplan twice weekly for 26 weeks. Assessments are first-morning uPCR every 4 weeks and renal biopsies at baseline/screening and Week 26 (Week 52 optional). The primary endpoint is the log-transformed ratio of uPCR at Week 26 relative to baseline. Key secondary endpoints measured as changes from baseline at Week 26 are proportion of patients who achieve the composite renal endpoint (≤15% eGFR and ≥50% uPCR reductions), proportion of patients with ≥50% uPCR reduction, change in eGFR, change in C3G histologic index activity score, and proportion of patients with decreased C3c staining on renal biopsy. Safety will be monitored throughout. Patients may enter a subsequent 8-week follow-up period or long-term extension study. In conclusion, this study will evaluate the safety and efficacy of the C3 inhibitor pegcetacoplan in treating patients with the rare, progressive renal diseases of C3G and IC-MPGN.

Funding

  • Apellis Pharmaceuticals, Inc.