Joseph M. Sanzari Children’s Hospital NEJM-Published Research Explores Treatment for Focal Segmental Glomerulosclerosis

Phase 3 trial reduced proteinuria in patients facing condition without current treatment

Focal segmental glomerulosclerosis (FSGS) does not currently have an effective treatment, but recent research explored a possible option.

Dr. Kenneth Lieberman, chief of the Division of Pediatric Nephrology at Joseph M. Sanzari Children’s Hospital at Hackensack University Medical Center, was among the authors of a study published in The New England Journal of Medicine studying the potential for sparsentan to reduce proteinuria in patients with Focal Segmental Glomerulosclerosis (FSGS).

In a prior 8-week, phase 2 trial, sparsentan, a dual endothelin-angiotensin receptor antagonist, successfully reduced proteinuria in patients with FSGS. The recently published phase 3 trial enrolled pediatric and adult patients with FSGS who were randomly assigned to receive sparsentan or irbesartan (active control) for 108 weeks.

At 36 weeks, the percentage of patients with partial remission of proteinuria was 42.0% in the sparsentan group and 26.0% in the irbesartan group, a response that was sustained through 108 weeks. At 108 weeks, there were no significant between-group differences in the eGFR slope, despite a greater reduction in proteinuria with sparsentan than with irbesartan. The study was funded by Travere Therapeutics.

Safety was also evaluated, though safety and efficacy of longer-term treatment requires further study.

Learn more about innovative pediatric kidney treatments at Hackensack Meridian Children's Health.