Phase 11b Study Evaluating Safety and Effects of BCX17725 in Healthy Adults and People With Netherton Syndrome

What this Trial Is About

Researchers are evaluating BCX17725, a drug being studied for safety, tolerability, how it moves through the body, and immune response in healthy adults and people with Netherton syndrome. This first-in-human, Phase 11b study includes multiple parts to assess single and multiple doses in healthy volunteers and multiple doses in individuals with Netherton syndrome. The study also aims to evaluate the drugs effectiveness, safety, and tolerability over 12 weeks in adults and adolescents with this condition. The study has four parts Parts 1 and 2 are randomized, placebo-controlled phases testing single and multiple ascending doses in healthy participants. Part 3 uses an open-label design to evaluate multiple doses in participants with Netherton syndrome. Part 4 assesses multiple intravenous andor subcutaneous doses over 12 weeks in adults and adolescents with Netherton syndrome, followed by an 8-week post-treatment follow-up. Participants will undergo assessments including safety monitoring for adverse events, blood tests to measure drug levels and immune response, and evaluations of skin condition severity using specific scores like the Ichthyosis Area and Severity Index and Investigator Global Assessment. The study tracks these measurements from screening through follow-up, with some parts lasting up to 20 weeks. Participants are monitored closely to understand the drugs effects and tolerability during and after treatment.

A Study of the Safety, Tolerability, Pharmacokinetics, and Immunogenicity of BCX17725