Clinical Trials

SOBI and Apellis Pharmaceuticals have announced the results from the VALIANT trial, which highlight that systemic pegcetacoplan treatment could benefit patients with C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN).

The VALIANT trial is a double-blind, randomised, multi-centre and placebo-controlled study that aimed to evaluate the efficacy and safety of pegcetacoplan in 124 patients over 12 years old who suffer from C3G or primary IC-MGPN.

Patients who were treated with pegcetacoplan achieved a key measure of kidney function: stabilisation of estimated glomerular function (eGFR). This stabilisation happened in the span of six months, with a difference of 6.3mL/min/1.73m2 (95% CI 0.5, 12.1; nominal p value = 0.03) compared to a placebo.

At week 26, patients who were treated with pegcetacoplan, in addition to a standard-of-care therapy, showed a statistically significant and clinically meaningful 68.1% (p<0.0001) proteinuria reduction (log-transformed ratio of urine protein-to-creatinine ratio).

The reduction of proteinuria was obser ved as early as week four, and continued through the six-month treatment period. Proteinuria reduction was consistent across broad patient subgroups including adolescent and adult patients, including those with primary IC-MPGN and C3G, in addition to patients with native and post-transplant kidneys.

Carla Nester, principle investigator, director of paediatric nephrology and professor of internal medicine and paediatrics at University of Iowa Stead Family Children’s Hospital, US, stated: “Pegcetacoplan is the only treatment to achieve substantial and clinically meaningful effects across all key markers of disease: proteinuria, eGFR stabilisation and C3c staining."