RecruitingPhase 1

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

United States78 participantsStarted 2024-09-26

Plain-language summary

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.

Who can participate

Age range12 Years – 65 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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

What they're measuring

Incidence of treatment-emergent adverse events (TEAEs)

Timeframe: From screening through EOS (ie, through Day 78 in Parts 1 and 3, and Day 106 in Part 2)

Change from baseline in Ichthyosis Area and Severity Index (IASI) score at Week 12 (Part 4)

Timeframe: From baseline to Week 12

Trial details

NCT IDNCT06539507

SponsorBioCryst Pharmaceuticals

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2026-10

Contact for this trial

BioCryst Pharmaceuticals, Inc.

+1 919 859 1302 clinicaltrials@biocryst.com