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

ConditionNetherton Syndrome

Trial statusRecruiting

Trial phasePhase 1

Trial typeInterventional

Biological sexAll

Age12-65

SponsorBioCryst Pharmaceuticals

About this trial

This is a first-in-human, Phase 1/1b, 4-part study that includes the evaluation of safety, tolerability, pharmacokinetics (PK), and immunogenicity of BCX17725 when administered via single and multiple doses in healthy adult participants (Parts 1 and 2), and multiple doses in adult participants with Netherton syndrome (Part 3). In Part 4, the effectiveness, safety, and tolerability of BCX17725 when administered via multiple IV and/or SC doses through 12 weeks will be evaluated in adult and adolescent participants with Netherton syndrome.

Eligibility criteria

Qualifiers

Male or non-pregnant, non-lactating female aged 18 to 55 years, inclusive (Parts 1 and 2), 18 to 65 years, inclusive (Part 3), or 12 to 65 years, inclusive (Part 4)

Confirmed diagnosis of Netherton syndrome (Parts 3 and 4)

IGA score of ≥ 3 (Parts 3 and 4) and IASI score of ≥ 16 (Part 4)

BMI between 18 and 30 kg/m², inclusive (Parts 1 and 2)