An Open-Label, Randomized, Controlled, Phase 2 Study to Evaluate the Safety and Efficacy of Pegcetacoplan in the Treatment of Post-Transplant Recurrence of C3G or IC-MPGN

Apellis Pharmaceuticals, Inc.50 sites in 11 countries13 target enrollmentSeptember 7, 2021

ConditionsC3G 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

InterventionsPegcetacoplan

DrugsPegcetacoplan

Overview

Phase: Phase 2
Intervention: Pegcetacoplan
Conditions: C3G
Sponsor: Apellis Pharmaceuticals, Inc.
Enrollment: 13
Locations: 50
Primary Endpoint: Percentage of Subjects With Reduction in C3c Staining on Renal Biopsy at Week 12
Status: Completed
Last Updated: 11 days ago

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

Brief Summary

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.

Registry

clinicaltrials.gov

Start Date

September 7, 2021

End Date

January 20, 2026

Last Updated

11 days ago

Study Type

Interventional

Study Design

Parallel

Sex

All

Investigators

Sponsor

Apellis Pharmaceuticals, Inc.

Responsible Party

Sponsor

Eligibility Criteria

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

Arms & Interventions

Group 1

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

Intervention: Pegcetacoplan

Group 2

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

Intervention: Pegcetacoplan

Outcomes

Primary Outcomes

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

Time Frame: Baseline (Day 1) and 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.

Secondary Outcomes

Percentage of Subjects With Reduction in C3c Staining on Renal Biopsy at Week 52(Baseline (Day 1) and Week 52)
Number of Subjects With Shift of C3c Staining From Baseline to Weeks 12 and 52(Baseline (Day 1), Week 12 and Week 52)
Percentage of Subjects With Stabilization or Improvement in Estimated Glomerular Filtration Rate (eGFR) at Week 52(Baseline (Day 1) and Week 52)
Percentage of Subjects With Stabilization or Improvement of Serum Creatinine Concentration at Week 52(Baseline (Day 1) and Week 52)
Change From Baseline in Estimated Glomerular Filtration Rate (eGFR) at Week 52(Baseline (Day 1) and Week 52)
Percentage Change From Baseline in Estimated Glomerular Filtration Rate (eGFR) at Week 52(Baseline (Day 1) and Week 52)
Change From Baseline in Serum Creatinine Concentration at Week 52(Baseline (Day 1) and Week 52)
Percentage Change From Baseline in Serum Creatinine Concentration at Week 52(Baseline (Day 1) and Week 52)