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

NCT05809531 | Active Not Recruiting Phase 3 DMC FDA Drug

• 4 other identifiers: APL2-C3G-3142023-504625-39-00U1111-1290-78262022-002833-33
• Study Type: interventional
• Target: 100
• Locations: 15 countries
• Sites: 47

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).

Conditions

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

Outcome Measures

Primary Outcomes (1)

• 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

Study Arms (1)

Pegcetacoplan administered subcutaneously

EXPERIMENTAL

Pegcetacoplan administered subcutaneously twice weekly according to protocol defined dosing regimen

Drug: Pegcetacoplan

Interventions

Pegcetacoplan DRUG

Complement (C3) Inhibitor

Also known as: APL-2

Pegcetacoplan administered subcutaneously

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Apellis Pharmaceuticals, Inc.: lead

Study Sites (47)

Academic Medical Research Institute (01034)

Los Angeles, California, 90022, United States

Location

Children's Hospital Colorado (01037)

Aurora, Colorado, 80045, United States

Location

University of Florida, Department of Pediatric Nephrology (01010)

Gainsville, Florida, 32610, United States

Location

Emory Investigational Drug Service (01021)

Atlanta, Georgia, 30322, United States

Location

University of Iowa Hospital and Clinics (01030)

Iowa City, Iowa, 52242, United States

Location

University of Michigan Hospital (01008)

Ann Arbor, Michigan, 48109, United States

Location

Hackensack Meridian Health (01036)

Hackensack, New Jersey, 07601, United States

Location

Cohen Children Hospital, Pediatric Pharmacy (01022)

Hyde Park, New York, 11040, United States

Location

Columbia Nephrology (01039)

New York, New York, 10032, United States

Location

Oregon Health & Science Univ., Dept. Pediatrics (01038)

Portland, Oregon, 97239, United States

Location

Northeast Clinical Research Center LLC (01009)

Bethlehem, Pennsylvania, 18017, United States

Location

Texas Children's Hospital (01027)

Houston, Texas, 77030, United States

Location

Hospital Privado-Universitario de Cordoba (54004)

Córdoba, CPA X5016KEH, Argentina

Location

Clinica Privada Velez Sarsfield (54007)

Córdoba, X5000, Argentina

Location

Kidney Trials Unit, Princess Alexandra Hospital (61007)

Woolloongab, Queensland, 4102, Australia

Location

St. Vincent's Hospital Melbourne (61003)

Fitzroy, 3065, Australia

Location

Catholic University of Leuven (32001)

Leuven, B-3000, Belgium

Location

Santa Casa de Misericordia de Belo Horizonte (55017)

Belo Horizonte, Minas Gerais, 30150-221, Brazil

Location

Irmandade da Santa Casa Misericordia Porto Alegre (55016)

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

Location

HC UNESP Botucatu (55010)

Botucatu, 18618-687, Brazil

Location

Hospital de Clinicas de Porto Alegre (55018)

Porto Alegre, 90035-903, Brazil

Location

Real Hospital Portugues de Beneficencia (55015)

Recife, 52010-095, Brazil

Location

Ruschel Medicina E Pesquisa Clinica (55012)

Rio de Janeiro, 22270-060, Brazil

Location

Hospital De Base (55014)

São José do Rio Preto, 1500900-000, Brazil

Location

Hospital do Rim - Fundacao Oswaldo Ramos (55005)

São Paulo, 04038-002, Brazil

Location

Univ Sao Paulo, Av. Dr Eneas Carvalho de Aguiar, 155 (55007)

São Paulo, 05403-900, Brazil

Location

Institute for Clinical and Experimental Medicine (42001)

Prague, 14021, Czechia

Location

CHU de Bordeaux - Hopital Pellegrin (33010)

Bordeaux, 33076, France

Location

Institut de transplantation urologie-nephrologie (33011)

Nantes, 44093, France

Location

Schneider Childrens Medical Center (97204)

Petah Tikva, 4920235, Israel

Location

Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico (39004)

Milan, 20122, Italy

Location

Istituto di Ricerche Farmacologiche Mario Negri IRCCS (39003)

Ranica, 24020, Italy

Location

Ospesale Pediatrico bambino Gesu (39005)

Rome, 165, Italy

Location

Nagasaki University Hospital (81005)

Nagasaki, Nagasaki, 852-8501, Japan

Location

Seirei Hamamatsu General Hospital (81004)

Hamamatsu, Shizuoka, 430-8558, Japan

Location

AMC Apotheek Goederenontvangst (31002)

Amsterdam, 1105 AZ, Netherlands

Location

University Medical Center Groningen (31004)

Groningen de Brug, 9713 GZ, Netherlands

Location

Radboud University Medical Center (31003)

Nijmegen, 6500 HB, Netherlands

Location

Yonsei University College of Medicine, Sinchon Severance Hospital (82002)

Seoul, 3722, South Korea

Location

Seoul National University Hospital (82003 & 82005)

Soeul, 3080, South Korea

Location

Hosp. Universit. Materno-Infantil Vall d' Hebron (34006)

Barcelona, 8035, Spain

Location

Hospital Universitario 12 de Octubre (34008)

Madrid, 28041, Spain

Location

Hospital Universitario Dr Peset (34009)

Valencia, 46017, Spain

Location

CHUV Lausanne (41002)

Lausanne, CH-1011, Switzerland

Location

Imperial College Hammersmith Hospital (44013)

London, W12 0HS, United Kingdom

Location

Royal Manchester Children's Hospital (44011)

Manchester, M13 9WL, United Kingdom

Location

Nottingham University Hospitals NHS Trust (44012)

Nottingham, NG7 2UH, 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

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 30, 2023
• First Posted: April 12, 2023
• Study Start: May 29, 2023
• Primary Completion (Estimated): July 1, 2027
• Study Completion (Estimated): July 1, 2027
• Last Updated: April 2, 2026

Record last verified: 2026-03

Locations

AU (2); CH (1); IL (1); FR (2); ES (3); BE (1); IT (3); JP (2); US (12); CZ (1); NL (3); KR (2); AR (2); BR (9); GB (3)