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

Last updated: March 30, 2026
Sponsor: BioCryst Pharmaceuticals
Overall Status: Active - Recruiting

Phase

1

Condition

Ichthyosis

Treatment

BCX17725

Placebo

Clinical Study ID

NCT06539507
BCX17725-101
U1111-1303-9510
2025-521973-16
  • Ages 12-65
  • All Genders
  • Accepts Healthy Volunteers

Study 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.

Eligibility Criteria

Inclusion

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

Study Design

Total Participants: 78
Treatment Group(s): 2
Primary Treatment: BCX17725
Phase: 1
Study Start date:
September 26, 2024
Estimated Completion Date:
December 31, 2026

Study Description

Parts 1 and 2 are randomized, placebo-controlled, single-ascending-dose (SAD) and multiple-ascending-dose (MAD) study parts, respectively, in healthy participants. Part 3 will evaluate multiple dose administrations in participants with Netherton syndrome in an open-label design. Part 4 will evaluate multiple administrations of BCX17725 in participants with Netherton syndrome in an open-label study design over 12 weeks, with an 8-week post-treatment follow-up period.

Connect with a study center

  • Westmead Hospital - Department of Dermatology

    Sydney, New South Wales 2145
    Australia

    Active - Recruiting

  • Investigative site

    Sydney 2147714, New South Wales 2155400
    Australia

    Site Not Available

  • Investigative site

    Brisbane, Queensland
    Australia

    Site Not Available

  • Nucleus Network

    Brisbane, Queensland 4006
    Australia

    Site Not Available

  • Veracity Clinical Research

    Brisbane, Queensland 4102
    Australia

    Active - Recruiting

  • Investigative site

    Brisbane 2174003, Queensland 2152274 4102
    Australia

    Site Not Available

  • Hôpital Saint-Louis

    Paris, 75010
    France

    Site Not Available

  • Universitätsklinikum Heidelberg

    Heidelberg, 69120
    Germany

    Site Not Available

  • Maastricht Universitair Medisch Centrum (MUMC+)

    Maastricht, 6229 HX
    Netherlands

    Site Not Available

  • Erasmus Universitair Medisch Centrum (EMC)

    Rotterdam, 3015 GD
    Netherlands

    Active - Recruiting

  • Stanford University School of Medicine

    Palo Alto, California 94304
    United States

    Site Not Available

  • Therapeutics Clinical Research

    San Diego, California 92123
    United States

    Active - Recruiting

  • Yale Center for Clinical Investigation

    New Haven, Connecticut 06519
    United States

    Active - Recruiting

  • Investigative site

    New Haven 4839366, Connecticut 4831725 06519
    United States

    Site Not Available

  • Northwestern Dermatology CTU

    Chicago, Illinois 60611
    United States

    Active - Recruiting

  • Dawes Fretzin Clinical Research Group, LLC

    Indianapolis, Indiana 46250
    United States

    Active - Recruiting

  • Investigative site

    Indianapolis, Indiana 46250
    United States

    Site Not Available

  • Investigative site

    Indianapolis 4259418, Indiana 4921868 46250
    United States

    Site Not Available

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