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

Nephrotic Syndrome is not a disease itself, but rather a group of signs and symptoms that result from damage in the part of the kidney that filters blood (glomeruli).

Common symptoms include:

• Foamy urine (called proteinuria) caused by protein “spilling” into the urine
• Severe swelling in parts of the body, most noticeably around the eyes, hands, feet, and abdomen (called edema)
• Weight gain due to a buildup of extra fluid
• Fatigue
• Loss of appetite
• Low levels of protein in the blood (hypoalbuminemia)
• Higher than normal fat and cholesterol levels in the blood (hyperlipidemia)

Nephrotic Syndrome can typically be diagnosed with a urine test.

Nephrotic Syndrome can be “primary” or “secondary” in nature.

Diseases that affect only the kidneys are called primary causes of Nephrotic Syndrome. Doctors often call these diseases “idiopathic,” which means that they arise from an unknown cause. Some of these diseases include:

• Minimal Change Disease (MCD) – most common in children
• Focal Segmental Glomerulosclerosis (FSGS)
• Membranous Nephropathy (MN) – most common in adults
• IgA Nephropathy (IgAN)

Secondary Nephrotic Syndrome is caused by an underlying, systemic condition like diabetes, lupus, HIV, and others.

Clinical studies are any studies that involve people. There are two main types: observational and interventional. In an observational trial, researchers track health outcomes over time in groups of participants to look for patterns that help us better understand a disease. In an interventional trial, participants receive specific interventions, which can include new treatments or behavorial changes (e.g., diet changes). To learn more about clinical research, please visit the NephCure website.