Study Assessing the Safety and Efficacy of Pegcetacoplan in Post-Transplant Recurrence of C3G or IC-MPGN

NCT04572854 | Completed Phase 2 Results DMC FDA Drug

• 1 other identifier: APL2-C3G-204
• Study Type: interventional
• Target: 13
• Locations: 10 countries
• Sites: 23

Brief Summary

This is a Phase 2, multicenter, open-label, randomized, controlled study designed to evaluate the safety and efficacy of pegcetacoplan in patients who have post-transplant recurrence of C3G or IC-MPGN.

Conditions

C3G; IC-MPGN; Renal Transplant; Membranoproliferative Glomerulonephritis; Membranoproliferative Glomerulonephritis (MPGN); Immune Complex Membranoproliferative Glomerulonephritis (IC-MPGN); C3 Glomerulopathy; C3 Glomerulonephritis; Complement 3 Glomerulonephritis; Complement 3 Glomerulopathy; Complement 3 Glomerulopathy (C3G); Dense Deposit Disease (DDD)

Outcome Measures

Primary Outcomes (1)

• Percentage of Subjects With Reduction in C3c Staining on Renal Biopsy at Week 12

  C3c staining intensity is semi quantitatively graded on a scale of 0 to 3, in which negative, 1+, 2+ and 3+ staining corresponds to scores of 0 to 3, with 0 being absent and 3 being the highest intensity seen on immunofluorescence. Higher scores indicate worse outcome. Reduction in C3c staining was defined as decrease of at least 2 orders of magnitude of intensity from baseline. Baseline was defined as the most recent non-missing measurement prior to first administration of study drug for Group 1 subjects or randomization for Group 2 subjects.

  Baseline (Day 1) and Week 12

Secondary Outcomes (8)

• Percentage of Subjects With Reduction in C3c Staining on Renal Biopsy at Week 52

  Baseline (Day 1) and Week 52

• Number of Subjects With Shift of C3c Staining From Baseline to Weeks 12 and 52

  Baseline (Day 1), Week 12 and Week 52

• Percentage of Subjects With Stabilization or Improvement in Estimated Glomerular Filtration Rate (eGFR) at Week 52

  Baseline (Day 1) and Week 52

• Percentage of Subjects With Stabilization or Improvement of Serum Creatinine Concentration at Week 52

  Baseline (Day 1) and Week 52

• Change From Baseline in Estimated Glomerular Filtration Rate (eGFR) at Week 52

  Baseline (Day 1) and Week 52

Study Arms (2)

Group 1

EXPERIMENTAL

Pegcetacoplan treatment of 1080 mg (sub-cutaneous infusion) twice weekly will be given throughout the entire study.

Drug: Pegcetacoplan

Group 2

OTHER

No intervention given during the randomized controlled portion of the study (through week 12). After week 12, subjects will receive pegcetacoplan treatment.

Drug: Pegcetacoplan

Interventions

Pegcetacoplan DRUG

Complement (C3) Inhibitor

Group 1; Group 2

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• At least 18 years of age at screening
• Must have clinical and pathologic evidence of recurrent C3G or IC-MPGN
• Stable (not improving) or worsening disease, in the opinion of the investigator, in the 2 months preceding the first dose of pegcetacoplan
• eGFR ≥15 mL/min/1.73 m2, calculated by the Chronic Kidney Disease-Epidemiology Collaboration (CKD-EPI) creatinine equation for adults
• No more than 50% glomerulosclerosis or interstitial fibrosis on the screening renal allograft biopsy
• Stable regimen for recurrent C3G/IC-MPGN for at least 4 weeks prior to the screening renal allograft biopsy and from the time of the screening renal allograft biopsy until randomization
• Have received required vaccinations against N. meningitidis, S. pneumoniae, and H. influenzae (type B) or agree to receive vaccinations, if applicable vaccination records are not available. Vaccination is mandatory unless documented evidence exists that subjects are non-responders to vaccination.

You may not qualify if:

• Absolute neutrophil count \<1000 cells/mm3 during screening
• Previous treatment with pegcetacoplan
• Evidence of rejection on the screening renal allograft biopsy that requires treatment
• Diagnosis or history of HIV, hepatitis B, or hepatitis C infection or positive serology at screening indicative of infection with any of these viruses
• Weight more than 100 kg at screening
• Hypersensitivity to pegcetacoplan or any of the excipients
• History of meningococcal disease
• Malignancy, except for the following:
• Cured basal or squamous cell skin cancer
• Curatively treated in situ disease
• Malignancy free and off treatment for ≥5 years
• Significant renal disease in the renal allograft secondary to another condition (eg, infection, malignancy, monoclonal gammopathy, rejection, or a medication) that would, in the opinion of the investigator, confound interpretation of the study results
• Participation in any other investigational drug trial or exposure to other investigational agent, device, or procedure within 30 days or 5 half-lives from the last dose of the investigational agent (whichever is longer) prior to screening
• Known or suspected hereditary fructose intolerance.

Sponsors & Collaborators

• Apellis Pharmaceuticals, Inc.: lead

Study Sites (25)

Mayo Clinic Arizona

Phoenix, Arizona, 85054, United States

Location

Keck School of Medicine, University of Southern California

Los Angeles, California, 900033, United States

Location

Children's Hospital Colorado

Aurora, Colorado, 80045, United States

Location

Mayo Clinic

Rochester, Minnesota, 55905, United States

Location

Washington University, St.Louis

St Louis, Missouri, 63110, United States

Location

NYU Langone Health Transplant Insitute

New York, New York, 10016, United States

Location

CUIMC

New York, New York, 10032, United States

Location

Hospital de Alta Complejidad en Red El Cruce Dr. Nestor Carlos Kirchner

San Juan Bautista, Buenos Aires, B1888AAE, Argentina

Location

Hospital Universitario Fundacion Favaloro

Buenos Aires, C1093AAS, Argentina

Location

The Royal Melbourne Hospital

Parkville, Victoria, 3050, Australia

Location

Monash Medical Centre

Clayton, 3168, Australia

Location

Medical University of Vienna

Vienna, 1090, Austria

Location

Irmandade da Santa Casa de Misericordia de Porto Alegre

Porto Alegre, Rio Grande do Sul, 90020-090, Brazil

Location

UPECLIN - Unidade de Pesquisa Clínica da Faculdade de Medicina de Botucatu - FMB/UNESP

Botucatu, São Paulo, 18618-686, Brazil

Location

Santa Casa de Misericordia de Belo Horizonte

Belo Horizonte, CEP 30150-221, Brazil

Location

Clinical Research Center, Hospital das Clinicas da Faculdade de Medicina da Universidade de São Paulo

São Paulo, 05403-000, Brazil

Location

Lille Regional University Hospital Center, Claude Huriez Hospital, Department of Nephrology

Lille, 59000, France

Location

Hopital Edouard Herriot, Hospices Civils de Lyon

Lyon, 69437, France

Location

Center Hospitalier Universitaire de Montpellier

Montpellier, 34295 CEDEX 5, France

Location

Istituto di Ricerche Farmacologiche Mario Negri IRCCS

Ranica, 24020, Italy

Location

Radbound University Medical Center

Nijmegen, 6500 HB, Netherlands

Location

Hospital Universitario 12 de Octubre, Nephrology Department

Madrid, 28041, Spain

Location

CHUV

Lausanne, 1005, Switzerland

Location

Imperial College Healthcare NHS Trust

London, W12 0HS, United Kingdom

Location

Freeman Hospital

Newcastle upon Tyne, NE7 7DN, United Kingdom

Location

MeSH Terms

Conditions

Glomerulonephritis, Membranoproliferative

Interventions

pegcetacoplan

Condition Hierarchy (Ancestors)

Glomerulonephritis; Nephritis; Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Immune System Diseases

Results Point of Contact

• Title: Apellis Clinical Trial Information Line
• Organization: Apellis Pharmaceuticals, Inc

clinicaltrials@apellis.com

1-833-284-6361

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 29, 2020
• First Posted: October 1, 2020
• Study Start: September 7, 2021
• Primary Completion: February 27, 2023
• Study Completion: January 20, 2026
• Last Updated: April 21, 2026
• Results First Posted: February 7, 2025

Record last verified: 2026-03

Locations

IT (1); NL (1); AR (2); GB (2); FR (3); ES (1); AU (1); US (7); CH (1); BR (4)