Netherton syndrome is a ultra-rare and very severe form of ichtyosis. It is a mongenetic disease. There is no specific treatment available for Netherton syndrome.~In this phase I/II trial a topical skin cream with the active substance SXR1096 will be tested in patients with Netherton syndrom. SXR1096 is a specific and potent protease inhibitor that can inhibit the proteases kallikrein 5, 7 and 14 - all recognised as up-regulated and causing the disease state in Netherton syndrome patients, as these patients have a gene defect in the gene spink5 encoding for the protease inhibitor LEKTI - that will normally regulate the activity of KLK5, 7 and 14 in the stratum corneum of the skin,~Patients will be treated for one month with active cream and placebo cream at different areas of the skin. The study will assess the safety and efficacy of the treatment in patients. There will also be pharmacokinetic, pharmacodynamic and biomarker studies conducted.
In this phase I/II trial a topical skin cream with the active substance SXR1096 will be tested in patients with Netherton syndroms, a rare inflammatory skin disease. SXR1096 is a specific and potent protease inhibitor that can inhibit the proteases kallikrein 5, 7 and 14 - all recognised as up-regulated and causing the disease state in Netherton syndrome patients. Patients will be treated for one month with active cream and placebo cream at different areas of the skin.