A PHASE 1/1B STUDY TO EVALUATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, AND IMMUNOGENICITY OF SINGLE AND MULTIPLE ASCENDING DOSES OF BCX17725 IN HEALTHY PARTICIPANTS AND MULTIPLE DOSES OF BCX17725 IN PARTICIPANTS WITH NETHERTON SYNDROME

Condition studied

Netherton Syndrome

Investigational medicinal product(s)

Eligibility

Primary endpoint

Effectiveness : Primary Effectiveness Endpoint: • Change from baseline in Ichthyosis Area and Severity Index (IASI) score at Week 12, Safety : Incidence of TEAEs through End-of-Study (EOS), PK : Concentration of BCX17725 in serum through EOS

Endpoint detail

Effectiveness : Key Secondary Effectiveness Endpoints: • Change from baseline in Investigator Global Assessment (IGA) score at Week 12 Change from baseline in the Worst Itch Numerical Rating Scale (NRS) score at Week 12