Phase III Study Assessing the Efficacy and Safety of Pegcetacoplan in Patients With C3 Glomerulopathy or Immune-Complex Membranoproliferative Glomerulonephritis (VALIANT)

A Phase 3, Randomized, Placebo-Controlled, Double-Blinded, Multicenter Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With C3 Glomerulopathy or Immune-Complex Membranoproliferative Glomerulonephritis

This is a Phase 3 study to assess the efficacy and safety of twice-weekly subcutaneous (SC) doses of pegcetacoplan compared to placebo in patients with C3 glomerulopathy (C3G) or immune-complex membranoproliferative glomerulonephritis (IC-MPGN) on the basis of a reduction in proteinuria.

Study Overview

• Status: Completed
• 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)
• Intervention / Treatment: Drug: Pegcetacoplan; Other: Placebo

• Study Type: Interventional
• Enrollment (Actual): 124
• Phase: Phase 3

Contacts and Locations

Study Locations

• Argentina
  • Buenos Aires, Argentina, 1629
    • Hospital Universitario Austral
  • Córdoba, Argentina, CPA X5016KEH
    • Hospital Privado-Universitario de Cordoba
  • Córdoba, Argentina, X5000
    • Clinica Privada Velez Sarsfield
• Australia
  • Box Hill, Australia, VIC 3128
    • Monash University
  • Fitzroy, Australia, VIC 3065
    • St. Vincents Melbourne
  • Woolloongabba, Australia, QLD 4102
    • Princess Alexandra Hospital
  • Australian Capital Territory
    • Garran, Australian Capital Territory, Australia, 2605
      • Canberra Hospital - Renal Clinical Trials & Research Unit
  • Queensland
    • Woolloongabba, Queensland, Australia, QLD 4102
      • Princess Alexandra Hospital
• Austria
  • Innsbruck, Austria, 6020
    • Medical University Hospital Innsbruck (43004)
  • Vienna, Austria, A-1090
    • Medizinische Universität Wien
• Belgium
  • Brussels, Belgium, 1070
    • Hopital Erasme HUB Service Pharmacie
  • Edegem, Belgium, 2650
    • University Hospital Antwerp (32004)
  • Leuven, Belgium, B-3000
    • Catholic University of Leuven
  • Liège, Belgium, B-4000
    • CHU Sart-Tilman
  • Liège, Belgium, B-4000
    • Clinical Trials CHU de Liège
• Brazil
  • Botucatu, Brazil, 18618-687
    • HC UNESP Botucatu
  • Fortaleza, Brazil, 60430-372
    • Hospital Universitario Walter Cantidio
  • Porto Alegre, Brazil, 90035-903
    • Hospital de Clínicas de Porto Alegre
  • Porto Alegre, Brazil, 90020-090
    • Irmandade Da Santa Casa de Misericórdia de Porto Alegre
  • Recife, Brazil, 52010-095
    • Real Hospital Portuguas de Beneficancia em Pernambuco
  • Ribeirão Preto, Brazil, 14110-000
    • Hospital das Clinicas de Ribeirao Preto, Division of Nephrology
  • Rio de Janeiro, Brazil, 22270-060
    • Ruschel Medicina e Pesquisa Clínica
  • Rio de Janeiro, Brazil, 22211-225
    • Nefrologia I-Dor
  • São José do Rio Preto, Brazil, 150900-000
    • Hospital de Base
  • São Paulo, Brazil, 04038-002
    • UNIFESP - Hospital Sao Paulo
  • São Paulo, Brazil, 05403-000
    • Instituto da Crianca-Hospital das Clinicas University of Sao Paulo
  • São Paulo, Brazil, 05403-900
    • HCFMUSP-Hospital Clinicas da Faculdade Medicina da Universidade de São Paulo
  • Minas Gerais
    • Belo Horizonte, Minas Gerais, Brazil, 30150-221
      • Santa Casa de Misericordia de Belo Horizonte
    • Juiz de Fora, Minas Gerais, Brazil, 36025-340
      • Centro de Tratamento de Doencas Renais
  • Rio Grande do Sul
    • Porto Alegre, Rio Grande do Sul, Brazil, 90020-090
      • Irmandade Da Santa Casa de Misericórdia de Porto Alegre
• Canada
  • Ontario
    • Toronto, Ontario, Canada, M5G 1X8
      • Hospital for Sick Children (11003)
  • Quebec
    • Montreal, Quebec, Canada, QC H1T2M4
      • Hôpital Maisonneuve-Rosemont
• Czechia
  • Prague, Czechia, 140 21
    • Institute for Clinical and Experimental Medicine
  • Prague, Czechia
    • Faculty Hospital Kralovske Vinohrady (42002)
• France
  • Bordeaux, France, 33076
    • CHU de Bordeaux - Hôpital Pellegrin
  • Créteil, France, 94010
    • Hôpital Henri-Mondor
  • Lyon, France, 69437
    • Hospital Edouard Herriot, Hospices Civils de Lyon
  • Montpellier, France, 34295
    • CHU Montpellier, Hopital Lapeyronie
  • Nantes, France, 44093
    • Nantes University Hospital
  • Paris, France, 75015
    • Hôpital Européen Georges-Pompidou
  • Paris, France, 59037
    • Lille Regional University Hospital Center, Claude Huriez Hospital, Department of Nephrology
  • Paris, France, 75015
    • Hopital Necker (33014)
  • Saint-Priest-en-Jarez, France, 42055
    • CHU de Saint Etienne, Hospital Nord
  • Strasbourg, France, 67091
    • University Hospital Strasbourg
  • Toulouse, France, 31059
    • Rangueil Hospital-University Hospital Center (CHU) of Toulouse
• Germany
  • Berlin, Germany, 10117
    • Charite Universitatsmedizin (49007)
  • Essen, Germany, D-45147
    • Universitatsklinikum Essen (AoR), Zentrum fur Kinder (49005)
  • Hanover, Germany, 30625
    • Medizinische Hochschule Hannover, Studienzentrum fur Nieren und Hochdruckerkrankungen
  • Mainz, Germany, 55131
    • Universitätsmedizin Mainz
  • Münster, Germany, 48149
    • Universitätsklinikum Münster
  • Regensburg, Germany, 93053
    • University Hospital Regensburg (49004)
• Israel
  • Haifa, Israel, 3109601
    • Rambam Health Care Campus
  • Petah Tikva, Israel, 4920235
    • Institute of Pediatric Nephrology
• Italy
  • Bari, Italy, 70123
    • Policlinico di Bari
  • Bologna, Italy, 40138
    • Policlinico Sant Orsola-Malpighi
  • Genova, Italy, 16147
    • IRCCS Istituto Giannina Gaslini (39012)
  • Messina, Italy, 98125
    • Università degli Studi di Messina
  • Milan, Italy, 20162
    • ASST Grande Ospedale Metropolitano Niguarda
  • Milan, Italy, 20156
    • Istituto di Ricerche Farmacologiche Mario Negri IRCCS
  • Padua, Italy, 35128
    • Azienda Ospedaliera Universitaria di Padova (39011)
  • Pavia, Italy, 27100
    • Instituti Clinici Scientifici Maugeri SPA-IRCCS
  • Rome, Italy, 00165
    • Ospedale Pediatrico Bambino Gesù
• Japan
  • Osaka, Japan, 530-8480
    • Kitano Hospital
  • Tokyo, Japan, 181-8611
    • Kyorin University Hospital (81009)
  • Aichi-ken
    • Nagoya, Aichi-ken, Japan, 466-8560
      • Nagoya University Hospital (81003)
    • Ōbu, Aichi-ken, Japan, 474-8710
      • Aichi Children's Health and Medical Center
  • Gunma
    • Maebashi, Gunma, Japan, 371-8511
      • Gunma University Hospital (81006)
  • Ishikawa-ken
    • Kanazawa, Ishikawa-ken, Japan, 9208650
      • NHO Kanazawa Medical Center
  • Nagasaki
    • Nagasaki, Nagasaki, Japan, 852-8501
      • Nagasaki University Hospital (81005)
  • Shizuoka
    • Hamamatsu, Shizuoka, Japan, 430-8558
      • Seirei Hamamatsu General Hospital (81004)
• Netherlands
  • Amsterdam, Netherlands, 1105 AZ
    • Emma Kinderziekenhuis, Amsterdam UMC
  • Groningen, Netherlands, 9713 GZ
    • University Medical Center Groningen
  • Nijmegen, Netherlands, 6500 HB
    • Radboud University Medical Center
• Poland
  • Lodz, Poland, 92-213
    • SPZOZ Centralny Szpital Kliniczny Uniwersytetu Medycznego w Lodzi
  • Lodz, Poland, 90-153
    • Samodzielny Publiczny Zaklad Opieki Zdrowotnej Uniwersytecki Szpital Kliniczny Nr 1 im. Norberta Barlickiego Uniwersytetu Medycznego w Lodzi
• South Korea
  • Daegu, South Korea, 42601
    • Keimyung University Dongsan Medical Center
  • Seoul, South Korea, 03722
    • Yonsei University College of Medicine, Sinchon Severance Hospital
  • Seoul, South Korea, 3080
    • Seoul National University Hospital (82005)
  • Soeul, South Korea, 03080
    • Seoul National University Hospital
• Spain
  • Barcelona, Spain, 08035
    • Hospital Universitari Vall d'Hebron
  • Barcelona, Spain, 08025
    • Fundacio Puigvert
  • Barcelona, Spain, 08035
    • Hospital Universitario Materno-Infantil Vall d' Hebron, Nefrologia Pediatrica
  • Barcelona, Spain, 08950
    • Hospital Materno Infantil Sant Joan de Deu
  • Madrid, Spain, 28041
    • Hospital Universitario 12 de Octubre, Nephrology Department
  • Santander, Spain, 39008
    • Hospital Universitario Marqués de Valdecilla
  • Seville, Spain, 41013
    • University Hospital of Virgen del Rocio
  • Valencia, Spain, 46017
    • Hospital Universitario Dr Peset
• Switzerland
  • Bern, Switzerland, CH-3010
    • Inselspital, Bern University Hospital
  • Lausanne, Switzerland, CH-1011
    • CHUV Lausanne
  • Zurich, Switzerland, CH-8091
    • UniversitätsSpital Zürich
• United Kingdom
  • Gloucester, United Kingdom, GL1 3NN
    • Gloucestershire Hospitals NHS Foundation Trust
  • Leicester, United Kingdom, LE1 5WW
    • University Hospitals of Leicester NHS trust (44003)
  • London, United Kingdom, W12 0HS
    • Imperial College Healthcare NHS Trust
  • London, United Kingdom, NW3 2QG
    • Royal Free London NHS Foundation Trust (44015)
  • London, United Kingdom, SE1 7EH
    • Evelina London Children Hospital (44016)
  • London, United Kingdom, SW17 0QT
    • St George'Äôs University Hospitals NHS Foundation Trust (44014)
  • London, United Kingdom, WC1N 3JH
    • Great Ormond Street Hospital Foundation Trust
  • Manchester, United Kingdom, M13 9WL
    • Royal Manchester Children's Hospital
  • Nottingham, United Kingdom, NG7 2UH
    • Nottingham Children's Hospital
• United States
  • Alabama
    • Birmingham, Alabama, United States, 35294
      • University of Alabama at Birmingham
  • California
    • Los Angeles, California, United States, 90022
      • Academic Medical Research Institute
    • Los Angeles, California, United States, 90033
      • Keck School of Medicine, University of Southern California
    • Los Angeles, California, United States, 90095
      • Ronald Reagan UCLA Medical Center (01035)
    • Orange, California, United States, 92868
      • UCI Center for Clinical Research
    • Sacramento, California, United States, 95817
      • UC Davis Medical Center (Transplant Research) (01016)
  • Colorado
    • Aurora, Colorado, United States, 80045
      • Children's Hospital Colorado
  • Florida
    • Gainesville, Florida, United States, 32610
      • University of Florida
  • Georgia
    • Atlanta, Georgia, United States, 30322
      • Emory University School of Medicine
    • Atlanta, Georgia, United States, 30342
      • Fides Clinical Research, LLC (01042)
  • Illinois
    • Chicago, Illinois, United States, 60611
      • Institute for Public Health and Medicine Northwestern University Northwestern University (01041)
    • Oak Brook, Illinois, United States, 60523
      • NANIU Research Chicago (01040)
  • Indiana
    • Fort Wayne, Indiana, United States, 46804
      • Nephrology Associates of Northern IL and Inn (01043)
  • Iowa
    • Iowa City, Iowa, United States, 52242
      • The University of Iowa
  • Massachusetts
    • Boston, Massachusetts, United States, 02115
      • Boston Children's Hospital (01013)
    • Springfield, Massachusetts, United States, 01107
      • Renal and Transplant Associates of New England, PC
  • Michigan
    • Ann Arbor, Michigan, United States, 48109
      • University of Michigan Medical Center
  • Missouri
    • Kansas City, Missouri, United States, 64102
      • Children's Mercy Hospital
  • New Jersey
    • Hackensack, New Jersey, United States, 07601
      • Hackensack Meridian Health
  • New York
    • New Hyde Park, New York, United States, 11040
      • Cohen Children Hospital
    • New York, New York, United States, 10032
      • CUIMC - Columbia Nephrology
  • Ohio
    • Columbus, Ohio, United States, 43210
      • The Ohio State University Medical Center
  • Oregon
    • Portland, Oregon, United States, 97239
      • Oregon Health & Science University (01038)
  • Pennsylvania
    • Bethlehem, Pennsylvania, United States, 18017
      • Northeast Clinical Research Center, LLC
  • Texas
    • El Paso, Texas, United States, 79902
      • Medresearch Inc
    • Houston, Texas, United States, 77030
      • Texas Children's Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 12 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Aged at least 18 years; where approved, adolescents (aged 12-17 years) weighing at least 30 kg may also be enrolled.
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:

1. Previous exposure to pegcetacoplan.
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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Triple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: 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)	Drug: Pegcetacoplan; Complement (C3) Inhibitor
Placebo Comparator: Group 2: Placebo administration; Subcutaneous infusion of either 10mL, 12mL, 15mL, or 20mL, twice weekly	Other: Placebo; Sterile solution of equal volume to active arm

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Randomized Controlled Period: Change From Baseline in Log-Transformed Urine Protein-to-Creatinine Ratio (uPCR) at Week 26	Baseline uPCR value was calculated as the average of the uPCR measurements from at least 6 of the 9 first-morning spot urine (FMU) samples collected between the start of screening and Day 1, inclusive. The uPCR values used to calculate baseline included those from the samples collected on Day -2, Day -1, and before dosing on Day 1. In situations where less than 6 samples or more than 9 samples were collected, the average of all collected samples was used for baseline derivation. The difference between treatment groups using a composite contrast of equal-weighted average over Weeks 24, 25, and 26 was estimated.	Baseline (Day -70 to Day 1) to Week 26

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Randomized Controlled Period: Percentage of Subjects Who Achieved the Composite Renal Endpoint at Week 26	Subject who achieved a composite renal endpoint was defined as: (1) a stable or improved estimated glomerular filtration rate (eGFR) compared to baseline (<=15% reduction in eGFR), and (2) a >=50% reduction in uPCR compared to baseline. Percentages are rounded off to the hundredth decimal place.	Week 26
Randomized Controlled Period: Percentage of Subjects With a Reduction of At Least 50% From Baseline in Urine Protein-to-Creatinine Ratio at Week 26	Baseline uPCR value was calculated as the average of the uPCR measurements from at least 6 of the 9 FMU samples collected between the start of screening and Day 1, inclusive. The uPCR values used to calculate baseline included those from the samples collected on Day -2, Day -1, and before dosing on Day 1. In situations where less than 6 samples or more than 9 samples were collected, the average of all collected samples was used for baseline derivation. Percentages are rounded off to the hundredth decimal place.	Baseline (Day -70 to Day 1) and Week 26
Randomized Controlled Period: Change From Baseline in the C3 Glomerulopathy (C3G) Histologic Index Activity Score at Week 26	The C3G histologic index used to assess disease activity and chronicity in C3G. The C3G total activity score ranges from 0 (worse) to 21 (best). Higher scores indicate better outcome. Baseline was defined as the most recent non-missing measurement prior to taking the first dose of study drug.	Baseline (Day 1) and Week 26
Randomized Controlled Period: Percentage of Subjects Who Showed Decrease in C3c Staining on Renal Biopsy From Baseline at Week 26	Subject who showed decrease in C3c staining was defined as decrease of at least 2 orders of magnitude of intensity from baseline. Percentages are rounded off to the hundredth decimal place.	Baseline (Day 1) and Week 26
Randomized Controlled Period: Change From Baseline in Estimated Glomerular Filtration Rate at Week 26	Serum samples were collected to determine the eGFR, calculated by using chronic kidney disease-epidemiology collaboration (CKD-EPI) creatinine equation for adults and the Bedside Schwartz equation for adolescents. Baseline eGFR value was calculated using the last non-missing assessment prior to first dose of study drug.	Baseline (Day 1) and Week 26
Randomized Controlled Period: Percentage of Subjects Who Achieved Proteinuria <1 Gram (g)/Day at Week 24	Urine samples were collected to determine the proteinuria. Percentage of subjects who achieved proteinuria <1 g/day was assessed by 24-hour urine protein. Percentages are rounded off to the hundredth decimal place.	Week 24
Randomized Controlled Period: Percentage of Subjects With Normalization of Serum Albumin Levels at Week 26	Baseline serum albumin value was calculated as the average of up to 2 serum albumin measurements preceding and including Day 1. Week 26 serum albumin values was calculated as the average of up to 2 serum albumin measurements preceding and including Week 26, no earlier than Week 20 measurement. Percentages are rounded off to the hundredth decimal place.	Week 26
Randomized Controlled Period: Percentage of Subjects With Serum C3 Levels Above the Lower Limit of Normal at Week 26	Baseline serum C3 value was calculated as the average of up to 2 serum C3 measurements preceding and including Day 1. Week 26 serum C3 value was calculated as the average of up to 2 serum C3 measurements preceding and including Week 26, no earlier than Week 20 measurement. Percentages are rounded off to the hundredth decimal place.	Week 26
Randomized Controlled Period: Change From Baseline in the Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) Score at Week 26	The FACIT-Fatigue scale was a 13-item Likert scaled instrument that was self-administered by subjects. Subjects were presented with 13 statements and asked to indicate their responses as it applied to the past 7 days. The 5 possible responses were "not at all" (0), "a little bit" (1), "somewhat" (2), "quite a bit" (3) and "very much" (4). With 13 statements the total score has a range of 0 (worse health-related quality of life) to 52 (best health-related quality of life). Higher scores indicate better quality of life. Baseline was defined as the most recent non-missing measurement prior to taking the first dose of study drug.	Baseline (Day 1) and Week 26
Randomized Controlled Period: Change From Baseline in the Kidney Disease Quality of Life (KDQOL) Score at Week 26	The KDQOL score was constructed as the KDQOL-36 Summary Score (KSS) by averaging the 24 items from Burden of Kidney Disease, Symptoms and Problems of Kidney Disease, and Effects of Kidney Disease on scale ranging from 0 (worse health-related quality of life) to 100 (best health-related quality of life). Higher scores indicate better quality of life. Baseline was defined as the most recent non-missing measurement prior to taking the first dose of study drug.	Baseline (Day 1) and Week 26

Collaborators and Investigators

• Sponsor: Apellis Pharmaceuticals, Inc.

Publications and helpful links

General Publications

• 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.

Helpful Links

• Study Website

Study record dates

Study Major Dates

• Study Start (Actual): November 12, 2021
• Primary Completion (Actual): June 26, 2024
• Study Completion (Actual): January 14, 2025

Study Registration Dates

• First Submitted: September 23, 2021
• First Submitted That Met QC Criteria: September 23, 2021
• First Posted (Actual): October 5, 2021

Study Record Updates

• Last Update Posted (Actual): January 29, 2026
• Last Update Submitted That Met QC Criteria: January 23, 2026
• Last Verified: August 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Urogenital Diseases; Male Urogenital Diseases; Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Immune System Diseases; Glomerulonephritis; Nephritis; Glomerulonephritis, Membranoproliferative; pegcetacoplan
• Other Study ID Numbers: APL2-C3G-310

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No