An Open-Label, Nonrandomized, Multicenter Extension Study to Evaluate the Long-term Safety and Efficacy of Pegcetacoplan in Participants With C3 Glomerulopathy or Immune-Complex Membranoproliferative Glomerulonephritis

Phase 3

Active, not recruiting

• Conditions: C3 Glomerulopathy; DDD; Membranoproliferative Glomerulonephritis; Complement 3 Glomerulopathy; Membranoproliferative Glomerulonephritis (MPGN); IC-MPGN; C3 Glomerulonephritis; Complement 3 Glomerulonephritis; Dense Deposit Disease; Immune Complex Membranoproliferative Glomerulonephritis (IC-MPGN)
• Interventions: Drug: Pegcetacoplan

• Registration Number: NCT05809531
• Lead Sponsor: Apellis Pharmaceuticals, Inc.

Brief Summary

This study is designed as a long-term extension to Study APL2-C3G-310, and is being conducted to establish the long-term safety and efficacy of pegcetacoplan in patients with C3 glomerulopathy (C3G) or immune-complex membranoproliferative glomerulonephritis (IC-MPGN).

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2023-03-30
• Study First Submitted QC: 2023-03-30
• Study First Posted: 2023-04-12
• Study Start: 2023-05-29
• Status Verified: 2025-03
• Last Update Submitted: 2025-03-27
• Last Update Posted: 2025-03-28
• Primary Completion: 2027-07
• Study Completion: 2027-07

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 100

Inclusion Criteria

• Completed participation in Study APL2-C3G-310 through the week 52 visit requirements
• Experienced clinical benefit from pegcetacoplan while participating in the previous trial, in the opinion of the investigator
• Must remain on a stable regimen for C3G or IC-MPGN treatment according to the requirements of Study APL2-C3G-310
• Received vaccinations against S pneumoniae, N meningitidis (types A, C, W, Y, and B), and H influenzae (type B) according to the requirements of Study APL2-C3G-310 and agree to receive any additional vaccinations recommended according to ACIP recommendations for adults or children with complement deficiencies and/or immunocompromising conditions or other similar local applicable guidelines
• Female participants of childbearing potential, defined as any woman who has experienced menarche and who is not permanently sterile or postmenopausal, must have a negative urine pregnancy test at visit 1 and must agree to use protocol-defined methods of contraception for the duration of the study through at least 90 days after receiving the last dose of pegcetacoplan
• Male participants must agree to use protocol-defined methods of contraception and agree to refrain from donating semen for the duration of the study through at least 90 days after receiving the last dose of pegcetacoplan
• Participants above the legal age of consent, in accordance with local regulations, must be willing and able to provide informed consent. The legally authorized representative of participants under the legal age of consent must be willing and able to provide informed consent; where appropriate, participants under the legal age of consent must also give their assent to participation in the study
• Willing and able to self-administer pegcetacoplan or have an identified caregiver who can perform the administration

Exclusion Criteria

• Female participants who are or are planning to become pregnant or who are currently breastfeeding and are unwilling to discontinue for the duration of the study and for at least 90 days after the final dose of study drug
• Inability or unwillingness to cooperate with the requirements of the protocol
• Any condition that, in the opinion of the investigator, creates an undue risk for the participant by participating in the study or is likely to confound interpretation of the study results
• Evidence of ongoing drug or alcohol abuse or dependence, in the opinion of the investigator

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Pegcetacoplan administered subcutaneously	Pegcetacoplan	Pegcetacoplan administered subcutaneously twice weekly according to protocol defined dosing regimen

Primary Outcome Measures

Name	Time	Method
Proportion of participants with a reduction in urine protein-to-creatinine ratio (uPCR) of at least 50% from the pretreatment value over time.	2.5 years

Trial Locations

Locations (47)

Academic Medical Research Institute (01034); 🇺🇸 Los Angeles, California, United States

Children's Hospital Colorado (01037); 🇺🇸 Aurora, Colorado, United States

University of Florida, Department of Pediatric Nephrology (01010); 🇺🇸 Gainesville, Florida, United States

Cohen Children Hospital, Pediatric Pharmacy (01022); 🇺🇸 Hyde Park, New York, United States

Oregon Health & Science Univ., Dept. Pediatrics (01038); 🇺🇸 Portland, Oregon, United States

Texas Children's Hospital (01027); 🇺🇸 Houston, Texas, United States

Hospital Privado-Universitario de Cordoba (54004); 🇦🇷 Cordoba, Argentina

Clinica Privada Velez Sarsfield (54007); 🇦🇷 Cordoba, Argentina

Kidney Trials Unit, Princess Alexandra Hospital (61007); 🇦🇺 Woolloongab, Queensland, Australia

Catholic University of Leuven (32001); 🇧🇪 Leuven, Belgium

Real Hospital Portugues de Beneficencia (55015); 🇧🇷 Recife, Brazil

Hospital De Base (55014); 🇧🇷 Sao Jose do Rio Preto, Brazil

Univ Sao Paulo, Av. Dr Eneas Carvalho de Aguiar, 155 (55007); 🇧🇷 Sao Paulo, Brazil

Institut de transplantation urologie-nephrologie (33011); 🇫🇷 Nantes, France

Schneider Childrens Medical Center (97204); 🇮🇱 Petah Tikva, Israel

Ospesale Pediatrico bambino Gesu (39005); 🇮🇹 Rome, Italy

Nagasaki University Hospital (81005); 🇯🇵 Nagasaki-shi, Nagasaki, Japan

Seirei Hamamatsu General Hospital (81004); 🇯🇵 Hamamatsu, Shizuoka, Japan

Seoul National University Hospital (82003 & 82005); 🇰🇷 Soeul, Korea, Republic of

AMC Apotheek Goederenontvangst (31002); 🇳🇱 Amsterdam, Netherlands

University Medical Center Groningen (31004); 🇳🇱 Groningen De Brug, Netherlands

Imperial College Hammersmith Hospital (44013); 🇬🇧 London, United Kingdom

Emory Investigational Drug Service (01021); 🇺🇸 Atlanta, Georgia, United States

University of Iowa Hospital and Clinics (01030); 🇺🇸 Iowa City, Iowa, United States

University of Michigan Hospital (01008); 🇺🇸 Ann Arbor, Michigan, United States

Santa Casa de Misericordia de Belo Horizonte (55017); 🇧🇷 Belo Horizonte, Minas Gerais, Brazil

HC UNESP Botucatu (55010); 🇧🇷 Botucatu, Brazil

Hackensack Meridian Health (01036); 🇺🇸 Hackensack, New Jersey, United States

Columbia Nephrology (01039); 🇺🇸 New York, New York, United States

Northeast Clinical Research Center LLC (01009); 🇺🇸 Bethlehem, Pennsylvania, United States

St. Vincent's Hospital Melbourne (61003); 🇦🇺 Fitzroy, Australia

Irmandade da Santa Casa Misericordia Porto Alegre (55016); 🇧🇷 Porto Alegre, RS, Brazil

Hospital de Clinicas de Porto Alegre (55018); 🇧🇷 Porto Alegre, Brazil

Ruschel Medicina E Pesquisa Clinica (55012); 🇧🇷 Rio De Janeiro, Brazil

Hospital do Rim - Fundacao Oswaldo Ramos (55005); 🇧🇷 São Paulo, Brazil

Institute for Clinical and Experimental Medicine (42001); 🇨🇿 Prague, Czechia

CHU de Bordeaux - Hopital Pellegrin (33010); 🇫🇷 Bordeaux, France

Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico (39004); 🇮🇹 Milano, Italy

Istituto di Ricerche Farmacologiche Mario Negri IRCCS (39003); 🇮🇹 Ranica, Italy

Yonsei University College of Medicine, Sinchon Severance Hospital (82002); 🇰🇷 Seoul, Korea, Republic of

Radboud University Medical Center (31003); 🇳🇱 Nijmegen, Netherlands

Hosp. Universit. Materno-Infantil Vall d' Hebron (34006); 🇪🇸 Barcelona, Spain

Hospital Universitario 12 de Octubre (34008); 🇪🇸 Madrid, Spain

Hospital Universitario Dr Peset (34009); 🇪🇸 Valencia, Spain

CHUV Lausanne (41002); 🇨🇭 Lausanne, Switzerland

Royal Manchester Children's Hospital (44011); 🇬🇧 Manchester, United Kingdom

Nottingham University Hospitals NHS Trust (44012); 🇬🇧 Nottingham, United Kingdom