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Recruiting NCT06539507

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

Phase I Interventional Netherton Syndrome

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: BCX17725, Placebo.
Who it may be relevant to
Registry conditions: Netherton Syndrome. Basic parameters: 12 years — 65 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United States, Australia, France, Germany, Netherlands
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official 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

Overview

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.

Detailed 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.

Interventions

  • Drug BCX17725
    BCX17725 for injection
  • Drug Placebo
    Placebo for injection

Primary outcome measures

  • 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)]
  • Change from baseline in Ichthyosis Area and Severity Index (IASI) score at Week 12 (Part 4) [Time frame: From baseline to Week 12]
Secondary outcome measures (9)
  • Maximum observed serum concentration (Cmax) [Time frame: Up to Day 64 (Part 1), Day 92 (Part 2), and Day 78 (Part 3)]
  • Time to maximum observed serum concentration (Tmax) [Time frame: Up to Day 64 (Part 1), Day 92 (Part 2), and Day 78 (Part 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)]
  • Terminal elimination half-life (t1/2) [Time frame: Up to Day 64 (Part 1), Day 92 (Part 2), and Day 78 (Part 3)]
  • 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)]
  • Change from baseline in Investigator Global Assessment (IGA) score at Week 12 (Part 4) [Time frame: From baseline to Week 12]
  • Change from baseline in Worst Itch Numerical Rating Score (NRS) at Week 12 (Part 4) [Time frame: From baseline to Week 12]
  • Incidence of TEAEs (Part 4) [Time frame: From baseline through Week 20]
  • Serum concentrations of BCX17725 (Part 4) [Time frame: From baseline through Week 20]

Eligibility criteria

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

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: Yes

Study design

Allocation
Randomized
Model
Sequential
Masking
Double blind
Primary purpose
Treatment

Study locations

United States · 5 centers
  • Stanford University School of Medicine — Palo Alto
  • Therapeutics Clinical Research — San Diego
  • Yale Center for Clinical Investigation — New Haven
  • Northwestern Dermatology CTU — Chicago
  • Dawes Fretzin Clinical Research Group, LLC — Indianapolis
Australia · 3 centers
  • Westmead Hospital - Department of Dermatology — Sydney
  • Nucleus Network — Brisbane
  • Veracity Clinical Research — Brisbane
Netherlands · 2 centers
  • Maastricht Universitair Medisch Centrum (MUMC+) — Maastricht
  • Erasmus Universitair Medisch Centrum (EMC) — Rotterdam
France · 1 center
  • Hôpital Saint-Louis — Paris
Germany · 1 center
  • Universitätsklinikum Heidelberg — Heidelberg

Identifiers

NCT: NCT06539507 · BCX17725-101 · U1111-1303-9510 · 2025-521973-16

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗