A Sequential Phase 2/3 Study of Pegcetacoplan in Patients with Focal Segmental Glomerulosclerosis
Research type
Research Study
Full title
A sequential Phase 2/3, single-arm, open-label study in adults followed by a randomized, placebo-controlled, double-blind, multicenter study to evaluate the efficacy and safety of pegcetacoplan in adults and adolescents with focal segmental glomerulosclerosis
IRAS ID
1013588
Contact name
Vincent Hohn
Contact email
Sponsor organisation
Apellis Pharmaceuticals, Inc.
Clinicaltrials.gov Identifier
Research summary
Focal segmental glomerulosclerosis (FSGS) is a rare kidney disease that causes scarring of parts of the kidneys, leading to protein leakage into the urine and progressive loss of kidney function. Many patients progress to kidney failure despite current treatments, highlighting an unmet medical need.
This study will evaluate whether pegcetacoplan, a medicine that targets the complement system (part of the immune system involved in kidney damage), is effective and safe in reducing proteinuria and slowing kidney function decline in patients with FSGS.
This is a sequential Phase 2/3 clinical trial. In Phase 2, approximately 15 adult participants will receive pegcetacoplan in addition to standard of care in an open-label setting to assess initial safety and efficacy. In Phase 3, approximately 250 participants (adults and, where permitted, adolescents aged 12–17 years) will be randomised to receive either pegcetacoplan or placebo in addition to standard of care in a double-blind, multicentre study.
Eligible participants include individuals with primary, genetic, or undetermined FSGS, significant proteinuria, and reduced kidney function, who are on stable therapy.
Participation will last up to approximately 120 weeks and includes an initial screening period (up to 8 weeks), a treatment period (104 weeks), and a follow-up period (8 weeks). Participants will receive twice-weekly subcutaneous infusions and attend regular clinic visits for blood and urine tests, kidney function assessments, and safety monitoring.
Potential benefits include improved disease control; however, participants may not directly benefit. Risks include infections and hypersensitivity reactions, which will be closely monitored.
The study will be conducted at multiple hospital-based nephrology centres. It is sponsored and funded by Apellis Pharmaceuticals, Inc.
REC name
Wales REC 2
REC reference
26/WA/0124
Date of REC Opinion
15 Jul 2026
REC opinion
Further Information Favourable Opinion