This study involves people with Netherton Syndrome, a rare genetic skin condition that causes red, scaly, and inflamed skin along with severe itching. The condition is caused by changes in a specific gene called SPINK5, which leads to problems with the skin’s protective barrier. People with this condition often experience ongoing skin inflammation and discomfort that can significantly affect their daily lives.
The treatment being tested is called BCX17725, which is an experimental medication given as an injection either into a vein, under the skin, or into a muscle. This medication is designed to work by blocking a specific protein that is overactive in people with Netherton Syndrome and contributes to the skin problems. The purpose of the study is to evaluate how safe and well-tolerated BCX17725 is when given to people with Netherton Syndrome, to measure how the body processes the medication, and to see if it has the potential to improve the skin condition.
During the study, participants will receive BCX17725 for 12 weeks. The study will include both adults and adolescents between the ages of 12 and 65 years who have been diagnosed with Netherton Syndrome. Throughout the study, doctors will monitor participants for any side effects and will assess changes in their skin condition using various scoring systems that measure the severity of scaling, redness, and itching. Blood samples will be taken to measure the amount of medication in the body over time, and skin samples may be collected using strips. Participants will need to attend regular study visits and complete questionnaires about their symptoms.
France 
Germany 
The Netherlands 
