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Abstract: INFO35

A Phase 3, Randomized, Multicenter Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Treatment of Complement 3 Glomerulopathy or Immune Complex Membranoproliferative Glomerulonephritis

Session Information

  • Informational Posters
    November 03, 2022 | Location: Exhibit Hall, Orange County Convention Center‚ West Building
    Abstract Time: 10:00 AM - 12:00 PM

Category: Glomerular Diseases

  • No subcategory defined

Authors

  • Li, Li, Apellis Pharmaceuticals Inc, Crestwood, Kentucky, United States
  • Pickering, Matthew C., Imperial College London, London, London, United Kingdom
  • Dixon, Bradley P., University of Colorado, Denver, Colorado, United States
  • Fakhouri, Fadi, Centre Hospitalier Universitaire Vaudois, Lausanne, Vaud, 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, Ospedale Pediatrico Bambino Gesu, Roma, Lazio, Italy
  • Kocinsky, Hetal S., Apellis Pharmaceuticals Inc, Crestwood, Kentucky, United States
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 of the efficacy and safety of pegcetacoplan in individuals with C3G or IC-MPGN.
Approximately 90 patients (age, ≥12 years; weight, 20-100 kg) diagnosed with C3G or IC-MPGN 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. 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 26 weeks. Assessments are first-morning uPCR every 4 weeks and renal biopsies at baseline/screening and weeks 26 and 52. The primary endpoint is proportion of participants with reduction in uPCR ≥50% relative to baseline at week 26. Secondary endpoints are proportion of participants with eGFR scores that are stable or improved from baseline; change in C3G histologic index activity score; and proportion of participants with decreased C3c staining on renal biopsy from baseline at week 26. Safety outcomes will also be monitored throughout the study. Participants may enter a subsequent 8-week follow-up period or long-term extension study.
C3G and IC-MPGN are rare, progressive renal diseases due to deposition in renal glomeruli. This study will evaluate the safety and efficacy of complement protein C3 inhibitor pegcetacoplan in treating C3G and IC-MPGN.

Funding

  • This study was funded by Apellis Pharmaceuticals, Inc. This abstract was developed under the direction of the authors with medical writing and editorial assistance provided by MedThink SciCom. Apellis Pharmaceuticals, Inc., and Sobi reviewed the poster. Funding for the medical writing and editorial assistance was provided by Apellis Pharmaceuticals, Inc.
Abstract: INFO35

A Phase 3, Randomized, Multicenter Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Treatment of Complement 3 Glomerulopathy or Immune Complex Membranoproliferative Glomerulonephritis

Session Information

  • Informational Posters
    November 04, 2022 | Location: Exhibit Hall, Orange County Convention Center‚ West Building
    Abstract Time: 10:00 AM - 12:00 PM

Category:

  • No subcategory defined

Authors

  • Li, Li, Apellis Pharmaceuticals Inc, Crestwood, Kentucky, United States
  • Pickering, Matthew C., Imperial College London, London, London, United Kingdom
  • Dixon, Bradley P., University of Colorado, Denver, Colorado, United States
  • Fakhouri, Fadi, Centre Hospitalier Universitaire Vaudois, Lausanne, Vaud, 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, Ospedale Pediatrico Bambino Gesu, Roma, Lazio, Italy
  • Kocinsky, Hetal S., Apellis Pharmaceuticals Inc, Crestwood, Kentucky, United States
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 of the efficacy and safety of pegcetacoplan in individuals with C3G or IC-MPGN.
Approximately 90 patients (age, ≥12 years; weight, 20-100 kg) diagnosed with C3G or IC-MPGN 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. 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 26 weeks. Assessments are first-morning uPCR every 4 weeks and renal biopsies at baseline/screening and weeks 26 and 52. The primary endpoint is proportion of participants with reduction in uPCR ≥50% relative to baseline at week 26. Secondary endpoints are proportion of participants with eGFR scores that are stable or improved from baseline; change in C3G histologic index activity score; and proportion of participants with decreased C3c staining on renal biopsy from baseline at week 26. Safety outcomes will also be monitored throughout the study. Participants may enter a subsequent 8-week follow-up period or long-term extension study.
C3G and IC-MPGN are rare, progressive renal diseases due to deposition in renal glomeruli. This study will evaluate the safety and efficacy of complement protein C3 inhibitor pegcetacoplan in treating C3G and IC-MPGN.

Abstract: INFO35

A Phase 3, Randomized, Multicenter Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Treatment of Complement 3 Glomerulopathy or Immune Complex Membranoproliferative Glomerulonephritis

Session Information

  • Informational Posters
    November 05, 2022 | Location: Exhibit Hall, Orange County Convention Center‚ West Building
    Abstract Time: 10:00 AM - 12:00 PM

Category:

  • No subcategory defined

Authors

  • Li, Li, Apellis Pharmaceuticals Inc, Crestwood, Kentucky, United States
  • Pickering, Matthew C., Imperial College London, London, London, United Kingdom
  • Dixon, Bradley P., University of Colorado, Denver, Colorado, United States
  • Fakhouri, Fadi, Centre Hospitalier Universitaire Vaudois, Lausanne, Vaud, 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, Ospedale Pediatrico Bambino Gesu, Roma, Lazio, Italy
  • Kocinsky, Hetal S., Apellis Pharmaceuticals Inc, Crestwood, Kentucky, United States
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 of the efficacy and safety of pegcetacoplan in individuals with C3G or IC-MPGN.
Approximately 90 patients (age, ≥12 years; weight, 20-100 kg) diagnosed with C3G or IC-MPGN 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. 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 26 weeks. Assessments are first-morning uPCR every 4 weeks and renal biopsies at baseline/screening and weeks 26 and 52. The primary endpoint is proportion of participants with reduction in uPCR ≥50% relative to baseline at week 26. Secondary endpoints are proportion of participants with eGFR scores that are stable or improved from baseline; change in C3G histologic index activity score; and proportion of participants with decreased C3c staining on renal biopsy from baseline at week 26. Safety outcomes will also be monitored throughout the study. Participants may enter a subsequent 8-week follow-up period or long-term extension study.
C3G and IC-MPGN are rare, progressive renal diseases due to deposition in renal glomeruli. This study will evaluate the safety and efficacy of complement protein C3 inhibitor pegcetacoplan in treating C3G and IC-MPGN.