This study investigates the effects of a medication called BI 764198 in people with Focal Segmental Glomerulosclerosis, which is a rare kidney disease that damages the small filtering units in the kidneys. The study specifically looks at individuals with primary focal segmental glomerulosclerosis or those with a version of the disease caused by TRPC6 gene mutations, which are changes in the genetic code that affect how the disease behaves. Some participants may also be taking a calcineurin inhibitor, which is a type of medicine used to manage certain immune-related conditions.
The purpose of the study is to determine if BI 764198 is more effective than a placebo at reducing the amount of protein leaked into the urine. During the study, participants will take either the active medication or a placebo in the form of an oral film-coated tablet. The treatment period lasts for 104 weeks, and the process is double-blind, meaning neither the participants nor the researchers know which treatment is being administered during the trial.
Who Can Join the Study?
You must be a male or female aged 12 years or older on the day you sign the consent forms.
Your weight must be at least 40 kg (about 88 pounds).
Your Body Mass Index (BMI), which is a measure of body fat based on your height and weight, must be 40 or less.
You must have a confirmed diagnosis of focal segmental glomerulosclerosis (FSGS), which is a rare kidney disease, through either:
A biopsy (a procedure where a small piece of kidney tissue is removed to be examined under a microscope).
A genetic test showing a specific change, called a gain-of-function mutation, in the TRPC6 gene.
Your urinary protein-to-creatinine ratio (UPCR), which is a test that measures the amount of protein in your urine compared to a waste product called creatinine, must be 1500 mg/g or higher.
Your estimated glomerular filtration rate (eGFR), which is a calculation used to measure how well your kidneys are filtering waste from your blood, must be 25 mL/min/1.73 m2 or higher.
Who Cannot Join the Study?
You have a known genetic condition or a syndrome that causes focal segmental glomerulosclerosis (a type of kidney disease), unless it is specifically caused by a TRPC6 gain-of-function mutation (a specific type of change in a gene that makes it more active than normal).
There is evidence from a doctor or a histologic examination (a study of tissue samples under a microscope) that your kidney disease was caused by other factors, such as damage from toxins or other medical conditions.
Your kidney disease is classified as FSGS-UC, which means the cause of the disease is currently unknown, and this was diagnosed before your first visit to the study.
You have a history of receiving an organ transplant (an operation where a healthy organ is placed into your body to replace a damaged one) or if you are planning to have an organ transplant while participating in this study.
You have used intravenous immunosuppressive agents (medicines given directly into a vein to lower the body’s immune system response) such as cyclophosphamide, rituximab, or obinutuzumab within the last 6 months before your first study visit.
BI 764198 is an experimental medicine taken by mouth that works by inhibiting a specific protein in the body called TRPC6. This treatment is being studied to see if it can help reduce the amount of protein lost in the urine for people with certain types of kidney diseases known as focal segmental glomerulosclerosis.
Focal segmental glomerulosclerosis – This condition affects the small filtering units of the kidneys known as glomeruli. It is characterized by scarring in certain parts of these filters. As the disease progresses, the damage to the kidney structures can lead to increased amounts of protein being released into the urine. This process often results from damage to the specialized cells that help filter blood. Over time, the scarring can spread and affect the overall ability of the kidneys to function properly.
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