Phase 2 Open-Label Study of Oral ManNAc Treatment for Adults with Primary Focal Segmental Glomerulosclerosis

What this Trial Is About

Focal segmental glomerulosclerosis FSGS is a kidney disease causing scarring in filtering parts of the kidneys, leading to protein loss in urine and worsening kidney function. This condition can progress to kidney failure, requiring dialysis or transplant. Current treatments often do not work well and have side effects, so better options are needed. This research studies a drug called ManNAc in adults with primary FSGS to evaluate its safety, tolerability, and effectiveness in reducing proteinuria. Participants will take ManNAc orally as a white powder dissolved in water, twice daily at a dose of 2,000 mg, for 12 weeks. The study is open-label and single-arm, involving 15 subjects. There will be 5 to 6 clinic visits over 14 weeks, including two overnight stays for 2 to 3 nights. During visits, physical exams, blood and urine tests, and questionnaires on health, sleep, and fatigue will be done. Some participants may have genetic testing and meet with a dietitian for nutrition advice while taking ManNAc. Participants will keep diaries to record doses and any side effects. The study team will call participants one week after the first dose and then every two weeks after clinic visits to monitor health. Researchers will measure proteinuria reduction primarily through urine proteincreatinine ratio and 24-hour urine collection. Other assessments include kidney function, symptom burden, and drug pharmacokinetics. The study aims to understand ManNAcs effects and safety over 12 weeks, with follow-up extending through the study duration.

An Open-Label Phase 2 Study of N-Acetyl-D-Mannosamine (ManNAc) in Subjects With Primary Focal Segmental Glomerulosclerosis