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

Phase 2Active Not RecruitingNCT04572854

Apellis Pharmaceuticals, Inc.13 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

C3G IC-MPGN Renal Transplant Complement 3 Glomerulopathy Complement 3 Glomerulopathy (C3G)Dense Deposit Disease (DDD)

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

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.

Locations (25)

Mayo Clinic Arizona

Phoenix, Arizona, United States

Keck School of Medicine, University of Southern California

Los Angeles, California, United States

Children's Hospital Colorado

Aurora, Colorado, United States

Mayo Clinic

Rochester, Minnesota, United States

Washington University, St.Louis

St Louis, Missouri, United States

NYU Langone Health Transplant Insitute

New York, New York, United States

CUIMC

New York, New York, United States

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

San Juan Bautista, Buenos Aires, Argentina

Hospital Universitario Fundacion Favaloro

Buenos Aires, Argentina

The Royal Melbourne Hospital

Parkville, Victoria, Australia

...and 15 more locations

Outcomes

Primary Outcomes

Percentage of Subjects With Reduction in C3c Staining on Renal Biopsy at Week 12

C3c staining intensity is semi quantitatively graded on a scale of 0 to 3, in which negative, 1+, 2+ and 3+ staining corresponds to scores of 0 to 3, with 0 being absent and 3 being the highest intensity seen on immunofluorescence. Higher scores indicate worse outcome. Reduction in C3c staining was defined as decrease of at least 2 orders of magnitude of intensity from baseline. Baseline was defined as the most recent non-missing measurement prior to first administration of study drug for Group 1 subjects or randomization for Group 2 subjects.

Time frame: Baseline (Day 1) and Week 12