Phase 2
N=13
Study Assessing the Safety and Efficacy of Pegcetacoplan in Post-Transplant Recurrence of C3G or IC-MPGN
C3G · IC-MPGN · Renal Transplant · Complement 3 Glomerulopathy · Complement 3 Glomerulopathy (C3G)
Bottom Line
View on ClinicalTrials.gov: NCT04572854 ↗Enrolled (actual)
13
Serious AEs
38.5%
Results posted
Feb 2025
Primary outcome: Primary: Percentage of Subjects With Reduction in C3c Staining on Renal Biopsy at Week 12 — 50.0; 33.3 Percentage of participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Pegcetacoplan (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Apellis Pharmaceuticals, Inc.
- Primary completion
- Feb 2023
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percentage of Subjects With Reduction in C3c Staining on Renal Biopsy at Week 12 |
50.0; 33.3 | — |
| SECONDARY Percentage of Subjects With Reduction in C3c Staining on Renal Biopsy at Week 52 |
50.0; 66.7 | — |
| SECONDARY Number of Subjects With Shift of C3c Staining From Baseline to Weeks 12 and 52 |
3; 0; 1; 0; 1; 1 | — |
| SECONDARY Percentage of Subjects With Stabilization or Improvement in Estimated Glomerular Filtration Rate (eGFR) at Week 52 |
70.0; 66.7 | — |
| SECONDARY Percentage of Subjects With Stabilization or Improvement of Serum Creatinine Concentration at Week 52 |
70.0; 66.7 | — |
| SECONDARY Change From Baseline in Estimated Glomerular Filtration Rate (eGFR) at Week 52 |
10.1; -10.3 | — |
| SECONDARY Percentage Change From Baseline in Estimated Glomerular Filtration Rate (eGFR) at Week 52 |
29.65; -16.67 | — |
| SECONDARY Change From Baseline in Serum Creatinine Concentration at Week 52 |
-0.044; 0.267 | — |
| SECONDARY Percentage Change From Baseline in Serum Creatinine Concentration at Week 52 |
3.198; 17.778 | — |
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.
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
- 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.
Data sourced from ClinicalTrials.gov (NCT04572854). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.