Phase III Study Assessing the Efficacy and Safety of Pegcetacoplan in Patients With C3 Glomerulopathy or Immune-Complex Membranoproliferative Glomerulonephritis
NCT ID: NCT05067127
Last Updated: 2026-01-29
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE3
124 participants
INTERVENTIONAL
2021-11-12
2025-01-14
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
TRIPLE
Study Groups
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Group 1: Pegcetacoplan administration
Subcutaneous infusion of 20mL (1080 mg), twice weekly (for adults or adolescents \>50kg), and the three other weight-based doses either of 10mL (540mg), 12mL (648mg), or 15mL (810mg)
Pegcetacoplan
Complement (C3) Inhibitor
Group 2: Placebo administration
Subcutaneous infusion of either 10mL, 12mL, 15mL, or 20mL, twice weekly
Placebo
Sterile solution of equal volume to active arm
Interventions
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Pegcetacoplan
Complement (C3) Inhibitor
Placebo
Sterile solution of equal volume to active arm
Eligibility Criteria
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Inclusion Criteria
2. A diagnosis of primary C3G or IC-MPGN (with or without previous renal transplant).
3. Evidence of active renal disease, based on one or more of the following:
1. In adults or adolescents with a baseline renal biopsy (either one collected during screening or a historic biopsy collected within 28 weeks prior to randomization), at least 2+ C3c staining on the baseline renal biopsy.
2. In adolescents not providing a baseline renal biopsy, at least one of the following:
* Plasma sC5b-9 level above the upper limit of normal during screening
* Serum C3 below the LLN during screening
* Presence of an active urine sediment during screening, as evidenced by hematuria with at least 5 red blood cells (RBCs) per high-power field (HPF) and/or red blood cell casts on local or central microscopic analysis of urine.
* Presence of C3 nephritic factor within 6 months of screening, based on central laboratory results or medical history.
4. No more than 50% global glomerulosclerosis or interstitial fibrosis on the baseline biopsy for adult participants or adolescent participants providing a baseline biopsy.
5. At least 1 g/day of proteinuria on a screening 24-hour urine collection and a uPCR of at least 1000 mg/g in at least 2 first-morning spot urine samples collected during screening.
6. eGFR ≥30 mL/min/1.73 m2 calculated by the Chronic Kidney Disease-Epidemiology Collaboration creatinine equation for adults or the Bedside Schwartz equation for adolescents.
7. Stable regimen for C3G/IC-MPGN treatment, as described below:
1. Angiotensin-converting enzyme inhibitor/, angiotensin receptor blocker, and/or sodium-glucose cotransporter-2 inhibitor therapy that is stable and optimized, in the opinion of the investigator, for at least 12 weeks prior to randomization
2. Stable doses of other medications that can affect proteinuria (eg, steroids, mycophenolate mofetil, and/or other allowed immunosuppressants that the participant is receiving for treatment of C3G or IC-MPGN) for at least 12 weeks prior to the baseline renal biopsy and randomization.
3. If a participant is on prednisone (or other systemic corticosteroid) for C3G or IC-MPGN treatment, the dosage is stable and no higher than 20 mg/day (or equivalent dosage of a corticosteroid other than prednisone) for at least 12 weeks prior to randomization.
8. Have received vaccinations against S pneumoniae, N meningitidis (types A, C, W, Y, and B), and H influenzae (type B) within 5 years prior to randomization or agree to receive vaccinations during screening.
Exclusion Criteria
2. C3G/IC-MPGN secondary to another condition (eg, infection, malignancy, monoclonal gammopathy, a systemic autoimmune disease such as systemic lupus erythematosus, chronic antibody-mediated rejection, or a medication), in the opinion of the investigator.
3. Current or prior diagnosis of human immunodeficiency virus (HIV), hepatitis B (HBV), or hepatitis C (HCV) infection or positive serology during screening that is indicative of infection with any of these viruses.
4. Body weight greater than 100 kg at screening.
5. Hypersensitivity to pegcetacoplan or to any of the excipients.
6. History of meningococcal disease.
7. Malignancy, except for the following:
1. Cured basal or squamous cell skin cancer
2. Curatively treated in situ disease
3. Malignancy-free and off treatment for ≥5 years
8. Severe infection (eg, requiring IV antibiotic therapy) within 14 days prior to the first dose of pegcetacoplan.
9. An absolute neutrophil count \<1000 cells/mm3 at screening.
10. Use of rituximab, belimumab, or any approved or investigational anticomplement therapy other than pegcetacoplan within 5 half-lives of that product prior to the screening period.
11. Female participants who are 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.
12. Presence or suspicion of severe infection during the screening period (including but not limited to recurrent or chronic infections) that, in the opinion of the investigator, may place the participant at unacceptable risk by study participation.
13. Known or suspected hereditary fructose intolerance.
12 Years
ALL
No
Sponsors
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Apellis Pharmaceuticals, Inc.
INDUSTRY
Responsible Party
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Locations
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University of Alabama at Birmingham
Birmingham, Alabama, United States
Academic Medical Research Institute
Los Angeles, California, United States
Keck School of Medicine, University of Southern California
Los Angeles, California, United States
Ronald Reagan UCLA Medical Center (01035)
Los Angeles, California, United States
UCI Center for Clinical Research
Orange, California, United States
UC Davis Medical Center (Transplant Research) (01016)
Sacramento, California, United States
Children's Hospital Colorado
Aurora, Colorado, United States
University of Florida
Gainesville, Florida, United States
Emory University School of Medicine
Atlanta, Georgia, United States
Fides Clinical Research, LLC (01042)
Atlanta, Georgia, United States
Institute for Public Health and Medicine Northwestern University Northwestern University (01041)
Chicago, Illinois, United States
NANIU Research Chicago (01040)
Oak Brook, Illinois, United States
Nephrology Associates of Northern IL and Inn (01043)
Fort Wayne, Indiana, United States
The University of Iowa
Iowa City, Iowa, United States
Boston Children's Hospital (01013)
Boston, Massachusetts, United States
Renal and Transplant Associates of New England, PC
Springfield, Massachusetts, United States
University of Michigan Medical Center
Ann Arbor, Michigan, United States
Children's Mercy Hospital
Kansas City, Missouri, United States
Hackensack Meridian Health
Hackensack, New Jersey, United States
Cohen Children Hospital
New Hyde Park, New York, United States
CUIMC - Columbia Nephrology
New York, New York, United States
The Ohio State University Medical Center
Columbus, Ohio, United States
Oregon Health & Science University (01038)
Portland, Oregon, United States
Northeast Clinical Research Center, LLC
Bethlehem, Pennsylvania, United States
MedResearch Inc
El Paso, Texas, United States
Texas Children's Hospital
Houston, Texas, United States
Hospital Universitario Austral
Buenos Aires, , Argentina
Hospital Privado-Universitario de Cordoba
Córdoba, , Argentina
Clinica Privada Velez Sarsfield
Córdoba, , Argentina
Canberra Hospital - Renal Clinical Trials & Research Unit
Garran, Australian Capital Territory, Australia
Princess Alexandra Hospital
Woolloongabba, Queensland, Australia
Monash University
Box Hill, , Australia
St. Vincents Melbourne
Fitzroy, , Australia
Princess Alexandra Hospital
Woolloongabba, , Australia
Medical University Hospital Innsbruck (43004)
Innsbruck, , Austria
Medizinische Universität Wien
Vienna, , Austria
Hopital Erasme HUB Service Pharmacie
Brussels, , Belgium
University Hospital Antwerp (32004)
Edegem, , Belgium
Catholic University of Leuven
Leuven, , Belgium
CHU Sart-Tilman
Liège, , Belgium
Clinical Trials CHU de Liège
Liège, , Belgium
Santa Casa de Misericordia de Belo Horizonte
Belo Horizonte, Minas Gerais, Brazil
Centro de Tratamento de Doencas Renais
Juiz de Fora, Minas Gerais, Brazil
Irmandade da Santa Casa de Misericordia de Porto Alegre
Porto Alegre, Rio Grande do Sul, Brazil
HC UNESP Botucatu
Botucatu, , Brazil
Hospital Universitario Walter Cantidio
Fortaleza, , Brazil
Irmandade da Santa Casa de Misericordia de Porto Alegre
Porto Alegre, , Brazil
Hospital de Clinicas de Porto Alegre
Porto Alegre, , Brazil
Real Hospital Portuguas de Beneficancia em Pernambuco
Recife, , Brazil
Hospital das Clinicas de Ribeirao Preto, Division of Nephrology
Ribeirão Preto, , Brazil
Nefrologia I-Dor
Rio de Janeiro, , Brazil
Ruschel Medicina E Pesquisa Clinica
Rio de Janeiro, , Brazil
Hospital de Base
São José do Rio Preto, , Brazil
UNIFESP - Hospital Sao Paulo
São Paulo, , Brazil
Instituto da Crianca-Hospital das Clinicas University of Sao Paulo
São Paulo, , Brazil
HCFMUSP-Hospital Clinicas da Faculdade Medicina da Universidade de São Paulo
São Paulo, , Brazil
Hospital for Sick Children (11003)
Toronto, Ontario, Canada
Hopital Maisonneuve-Rosemont
Montreal, Quebec, Canada
Institute for Clinical and Experimental Medicine
Prague, , Czechia
Faculty Hospital Kralovske Vinohrady (42002)
Prague, , Czechia
CHU de Bordeaux - Hopital Pellegrin
Bordeaux, , France
Hopital Henri-Mondor
Créteil, , France
Hospital Edouard Herriot, Hospices Civils de Lyon
Lyon, , France
CHU Montpellier, Hopital Lapeyronie
Montpellier, , France
Nantes University Hospital
Nantes, , France
Lille Regional University Hospital Center, Claude Huriez Hospital, Department of Nephrology
Paris, , France
Hopital Necker (33014)
Paris, , France
Hôpital Européen Georges-Pompidou
Paris, , France
CHU de Saint Etienne, Hospital Nord
Saint-Priest-en-Jarez, , France
University Hospital Strasbourg
Strasbourg, , France
Rangueil Hospital-University Hospital Center (CHU) of Toulouse
Toulouse, , France
Charite Universitatsmedizin (49007)
Berlin, , Germany
Universitatsklinikum Essen (AoR), Zentrum fur Kinder (49005)
Essen, , Germany
Medizinische Hochschule Hannover, Studienzentrum fur Nieren und Hochdruckerkrankungen
Hanover, , Germany
Universitatsmedizin Mainz
Mainz, , Germany
Universitatsklinikum Munster
Münster, , Germany
University Hospital Regensburg (49004)
Regensburg, , Germany
Rambam Health Care Campus
Haifa, , Israel
Institute of Pediatric Nephrology
Petah Tikva, , Israel
Policlinico di Bari
Bari, , Italy
Policlinico Sant Orsola-Malpighi
Bologna, , Italy
IRCCS Istituto Giannina Gaslini (39012)
Genova, , Italy
Universita degli Studi di Messina
Messina, , Italy
Istituto di Ricerche Farmacologiche Mario Negri IRCCS
Milan, , Italy
ASST Grande Ospedale Metropolitano Niguarda
Milan, , Italy
Azienda Ospedaliera Universitaria di Padova (39011)
Padua, , Italy
Instituti Clinici Scientifici Maugeri SPA-IRCCS
Pavia, , Italy
Ospedale Pediatrico Bambino Gesu
Rome, , Italy
Nagoya University Hospital (81003)
Nagoya, Aichi-ken, Japan
Aichi Children's Health and Medical Center
Ōbu, Aichi-ken, Japan
Gunma University Hospital (81006)
Maebashi, Gunma, Japan
NHO Kanazawa Medical Center
Kanazawa, Ishikawa-ken, Japan
Nagasaki University Hospital (81005)
Nagasaki, Nagasaki, Japan
Seirei Hamamatsu General Hospital (81004)
Hamamatsu, Shizuoka, Japan
Kitano Hospital
Osaka, , Japan
Kyorin University Hospital (81009)
Tokyo, , Japan
Emma Kinderziekenhuis, Amsterdam UMC
Amsterdam, , Netherlands
University Medical Center Groningen
Groningen, , Netherlands
Radboud University Medical Center
Nijmegen, , Netherlands
Samodzielny Publiczny Zaklad Opieki Zdrowotnej Uniwersytecki Szpital Kliniczny Nr 1 im. Norberta Barlickiego Uniwersytetu Medycznego w Lodzi
Lodz, , Poland
SPZOZ Centralny Szpital Kliniczny Uniwersytetu Medycznego w Lodzi
Lodz, , Poland
Keimyung University Dongsan Medical Center
Daegu, , South Korea
Yonsei University College of Medicine, Sinchon Severance Hospital
Seoul, , South Korea
Seoul National University Hospital (82005)
Seoul, , South Korea
Seoul National University Hospital
Soeul, , South Korea
Fundació Puigvert
Barcelona, , Spain
Hospital Universitari Vall D'Hebron
Barcelona, , Spain
Hospital Universitario Materno-Infantil Vall d' Hebron, Nefrologia Pediatrica
Barcelona, , Spain
Hospital Materno Infantil Sant Joan de Deu
Barcelona, , Spain
Hospital Universitario 12 de Octubre, Nephrology Department
Madrid, , Spain
Hospital Universitario Marques de Valdecilla
Santander, , Spain
University Hospital of Virgen del Rocio
Seville, , Spain
Hospital Universitario Dr Peset
Valencia, , Spain
Inselspital, Bern University Hospital
Bern, , Switzerland
CHUV Lausanne
Lausanne, , Switzerland
Universitatsspital Zurich
Zurich, , Switzerland
Gloucestershire Hospitals NHS Foundation Trust
Gloucester, , United Kingdom
University Hospitals of Leicester NHS trust (44003)
Leicester, , United Kingdom
Royal Free London NHS Foundation Trust (44015)
London, , United Kingdom
Evelina London Children Hospital (44016)
London, , United Kingdom
St George'Äôs University Hospitals NHS Foundation Trust (44014)
London, , United Kingdom
Imperial College Healthcare NHS Trust
London, , United Kingdom
Great Ormond Street Hospital Foundation Trust
London, , United Kingdom
Royal Manchester Children's Hospital
Manchester, , United Kingdom
Nottingham Children's Hospital
Nottingham, , United Kingdom
Countries
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References
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Fakhouri F, Bomback AS, Ariceta G, Delmas Y, Dixon BP, Gale DP, Greenbaum LA, Han SH, Isbel N, Le Quintrec M, Licht C, Mastrangelo A, Mizuno M, Neves de Holanda MI, Pickering MC, Remuzzi G, Van De Kar N, Vivarelli M, Walker PD, Wallace D, Zecher D, Francois C, Deschatelets P, Li L, Wang Z, Abad-Franch L, Kinnman N, Lopez-Lazaro L, Szamosi J, Nester CM; VALIANT Trial Investigators Group. Trial of Pegcetacoplan in C3 Glomerulopathy and Immune-Complex MPGN. N Engl J Med. 2025 Dec 4;393(22):2210-2220. doi: 10.1056/NEJMoa2501510.
Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Other Identifiers
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APL2-C3G-310
Identifier Type: -
Identifier Source: org_study_id
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