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

NCT ID: NCT04572854

Last Updated: 2025-03-30

Study Results

Results available

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Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE2

Total Enrollment

13 participants

Study Classification

INTERVENTIONAL

Study Start Date

2021-09-07

Study Completion Date

2026-01-31

Brief Summary

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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.

Detailed Description

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Conditions

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

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Group 1

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

Group Type EXPERIMENTAL

Pegcetacoplan

Intervention Type DRUG

Complement (C3) Inhibitor

Group 2

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

Group Type OTHER

Pegcetacoplan

Intervention Type DRUG

Complement (C3) Inhibitor

Interventions

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Pegcetacoplan

Complement (C3) Inhibitor

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* 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.

Exclusion Criteria

* 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.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Apellis Pharmaceuticals, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Mayo Clinic Arizona

Phoenix, Arizona, United States

Site Status

Keck School of Medicine, University of Southern California

Los Angeles, California, United States

Site Status

Children's Hospital Colorado

Aurora, Colorado, United States

Site Status

Mayo Clinic

Rochester, Minnesota, United States

Site Status

Washington University, St.Louis

St Louis, Missouri, United States

Site Status

NYU Langone Health Transplant Insitute

New York, New York, United States

Site Status

CUIMC

New York, New York, United States

Site Status

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

San Juan Bautista, Buenos Aires, Argentina

Site Status

Hospital Universitario Fundacion Favaloro

Buenos Aires, , Argentina

Site Status

The Royal Melbourne Hospital

Parkville, Victoria, Australia

Site Status

Monash Medical Centre

Clayton, , Australia

Site Status

Medical University of Vienna

Vienna, , Austria

Site Status

Irmandade da Santa Casa de Misericordia de Porto Alegre

Porto Alegre, Rio Grande do Sul, Brazil

Site Status

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

Botucatu, São Paulo, Brazil

Site Status

Santa Casa de Misericordia de Belo Horizonte

Belo Horizonte, , Brazil

Site Status

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

São Paulo, , Brazil

Site Status

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

Lille, , France

Site Status

Hopital Edouard Herriot, Hospices Civils de Lyon

Lyon, , France

Site Status

Center Hospitalier Universitaire de Montpellier

Montpellier, , France

Site Status

Istituto di Ricerche Farmacologiche Mario Negri IRCCS

Ranica, , Italy

Site Status

Radbound University Medical Center

Nijmegen, , Netherlands

Site Status

Hospital Universitario 12 de Octubre, Nephrology Department

Madrid, , Spain

Site Status

CHUV

Lausanne, , Switzerland

Site Status

Imperial College Healthcare NHS Trust

London, , United Kingdom

Site Status

Freeman Hospital

Newcastle upon Tyne, , United Kingdom

Site Status

Countries

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United States Argentina Australia Austria Brazil France Italy Netherlands Spain Switzerland United Kingdom

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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APL2-C3G-204

Identifier Type: -

Identifier Source: org_study_id

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