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NCT Number: NCT06539507

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

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.

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Key information

Conditions

Netherton Syndrome Abnormalities, Multiple Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Ichthyosiform Erythroderma, Congenital Ichthyosis Infant, Newborn, Diseases Keratosis Skin Abnormalities Skin Diseases Skin Diseases, Genetic Skin and Connective Tissue Diseases

Age range

12 year–65 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

Westmead Hospital - Department of Dermatology, Sydney, New South Wales, Australia

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About this study

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.

Who can participate

Healthy volunteers accepted: Yes

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

BCX17725

Drug

BCX17725 for injection

Placebo

Drug

Placebo for injection

Primary outcomes

  1. Incidence of treatment-emergent adverse events (TEAEs)

    Time frame: 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

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

    Time frame: 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.

Secondary outcomes

  1. Maximum observed serum concentration (Cmax)

    Time frame: Up to Day 64 (Part 1), Day 92 (Part 2), and Day 78 (Part 3)

    Cmax of BCX17725

  2. Time to maximum observed serum concentration (Tmax)

    Time frame: Up to Day 64 (Part 1), Day 92 (Part 2), and Day 78 (Part 3)

    Time to Cmax of BCX17725

  3. Area under the serum concentration-time curve (AUC) from time 0 to the time of last measurable concentration (AUC0-t)

    Time frame: Up to Day 64 (Part 1), Day 92 (Part 2), and Day 78 (Part 3)

    AUC0-t of BCX17725

  4. Terminal elimination half-life (t1/2)

    Time frame: Up to Day 64 (Part 1), Day 92 (Part 2), and Day 78 (Part 3)

    Terminal elimination half-life (t1/2) of BCX17725

  5. Number of participants who are anti-drug antibody (ADA)-positive (baseline and post-baseline) and number of participants who have treatment-emergent ADAs

    Time frame: Day 1 pre-dose and up to Day 64 (Part 1), Day 92 (Part 2), and Day 78 (Part 3)

    Incidence of ADAs to BCX17725

  6. Change from baseline in Investigator Global Assessment (IGA) score at Week 12 (Part 4)

    Time frame: From baseline to Week 12

    IGA assesses the overall severity of a participant's NS skin disease based on a 5-point scale (0, clear; 1, almost clear; 2, mild; 3, moderate; and 4, severe). Higher scores indicate worse clinical outcome.

  7. Change from baseline in Worst Itch Numerical Rating Score (NRS) at Week 12 (Part 4)

    Time frame: From baseline to Week 12

    Worst Itch Numerical Rating Scale (NRS) is a self-rated, single-item scale designed for assessing the worst itch in the past 7 days. The scale uses an 11-point NRS, scored from 0 (no itch) to 10 (worst possible itch). Higher scores indicate worse clinical outcome.

  8. Incidence of TEAEs (Part 4)

    Time frame: From baseline through Week 20

    Incidence of TEAEs as assessed by CTCAE

  9. Serum concentrations of BCX17725 (Part 4)

    Time frame: From baseline through Week 20

    Serum concentrations of BCX17725

Study contacts

Contact information is provided by the study sponsor or research team.

BioCryst Pharmaceuticals, Inc.

CONTACT

[email protected]

+1 919 859 1302

Sponsors and collaborators

Lead sponsor

BioCryst Pharmaceuticals

Industry

Registry information

Official study title

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

Important dates

Study start
2024
Primary completion
2026
Study completion
2026
First posted
Aug 6, 2024
Registry last updated
Jun 16, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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