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

Apellis Pharmaceuticals

Status and phase: Active, not recruiting; Phase 2

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

Treatments: Drug: Pegcetacoplan

Study type: Interventional

Funder types: Industry

Identifiers

NCT04572854

APL2-C3G-204

Details and patient eligibility

About

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.

Enrollment: 13 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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13 participants in 2 patient groups

Group 1

Experimental group

Description:

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

Treatment:

Drug: Pegcetacoplan

Group 2

Other group

Description:

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

Treatment:

Drug: Pegcetacoplan