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
Sex
ALL
Min age
12 Years
Max age
65 Years
Healthy volunteers
Accepted
Key Inclusion Criteria:
* 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/m\^2, inclusive (Parts 1 and 2)
* Estimated glomerular filtration rate (eGFR) of ≥ 90 mL/min/1.73 m\^2 (Parts 1 and 2) or ≥ 60 mL/min/1.73 m\^2 (Part 3)
* Agree to follow the protocol contraception requirements from screening until 90 days after the last dose of study drug
* In the opinion of the investigator, expected to adequately comply with all required study procedures and restrictions for the duration of the study
Primary outcome measure(s)
Incidence of treatment-emergent adverse events (TEAEs) — From screening through EOS (ie, through Day 78 in Parts 1 and 3, and Day 106 in Part 2) Incidence of TEAEs as assessed by Common Terminology Criteria for Adverse Events (CTCAE) from screening through end-of-study (EOS) in each study part
Change from baseline in Ichthyosis Area and Severity Index (IASI) score at Week 12 (Part 4) — From baseline to Week 12 IASI measures the severity of erythema (IASI-E) and scaling (IASI-S); the maximum sub-scores for the IASI-E and IASI-S being 24, and the maximum total IASI score being 48. Higher scores indicate worse clinical outcome.
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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