Abstract: INFO21-TH
PRESERVE-C3Tx, a Phase 3b Pilot Study Design: Effect of Preemptive Pegcetacoplan Use on Recurrence of C3 Glomerulopathy or Primary Immune Complex-Membranoembranoproliferative Glomerulonephritis in Kidney Transplant Recipients
Session Information
- Informational Posters - 1
October 22, 2026 | Location: Exhibit Hall A, Convention Center
Abstract Time: 10:00 AM - 12:00 PM
Category: Transplantation
- No subcategory defined
Authors
- Haffner, Dieter, Medizinische Hochschule Hannover, Hannover, NDS, Germany
- Caravaca-Fontan, Fernando, Instituto de Investigacion Hospital 12 de Octubre, Madrid, Community of Madrid, Spain
- Le Quintrec, Moglie, Centre Hospitalier Universitaire de Lapeyronie, Montpellier, France
- Vivarelli, Marina, Laboratory of Nephrology and Clinical Trial Center, IRCCS Bambino Gesu Children's Hospital, Rome, Italy
- Cabral, Diogo Buarque C., Real Hospital Portugues de Beneficencia em Pernambuco, Recife, PE, Brazil
- Patel, Sheena, Swedish Orphan Biovitrum AB publ, Stockholm, Stockholm County, Sweden
- Szamosi, Johan, Swedish Orphan Biovitrum AB publ, Stockholm, Stockholm County, Sweden
- Sheerin, Neil S., Newcastle upon Tyne Hospitals National Health Service Foundation Trust, Newcastle upon Tyne, United Kingdom
Description
C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (pIC-MPGN) are rare diseases characterized by uncontrolled C3 activation and excessive glomerular deposition of C3 breakdown products, leading to kidney failure in up to 50% of patients within 10 years. Up to 89% of patients experience disease recurrence after kidney transplantation. There is a high unmet need for therapeutic strategies that improve long-term graft outcomes.
Pegcetacoplan, a targeted C3/C3b inhibitor, has demonstrated benefits in patients with native kidney or post-transplant recurrent C3G and pIC-MPGN. However, the role of pre-emptive pegcetacoplan on the development of recurrent disease has not been established.
This single-arm, open-label, multicenter Phase 3b pilot study will explore the efficacy and safety of pegcetacoplan on disease recurrence and graft outcomes after kidney transplantation.
Approximately 30 patients ≥12 years old with C3G or pIC-MPGN undergoing transplantation and with high risk of recurrence (as assessed by investigators) will be enrolled. Patients will initiate pegcetacoplan treatment at the time of kidney transplantation surgery. Twice weekly 1,080 mg pegcetacoplan treatment (or weight-based dosing in adolescents) will continue for up to 24 months post-transplant, and protocol biopsy analyses are scheduled at 6, 12, and 24 months.
The primary endpoint will be occurrence of clinically significant recurrent disease within 2 years after transplantation, requiring both histological evidence on kidney biopsy and clinical evidence (≥1 of: increase in proteinuria, decrease in estimated glomerular filtration rate [eGFR], or significant hematuria).
Secondary endpoints will include time to clinically significant recurrence, frequency and time to biopsy-proven recurrence, as well as safety of pegcetacoplan.
Exploratory endpoints will assess the impact of pegcetacoplan on disease activity markers (changes in proteinuria, eGFR, hematuria), renal biopsy features, graft outcomes (delayed graft function, graft rejection and failure events), complement biomarkers, as well as the impact of previous transplantations on the time to recurrence or graft failure.
PRESERVE-C3Tx is expected to start recruitment by the end of 2026 with data to be presented over the coming years.
Funding
- Swedish Orphan Biovitrum AB (Sobi)