Recruiting
Phase 1

BCX17725

Sponsor:

BioCryst Pharmaceuticals

Code:

NCT06539507

Conditions

Netherton Syndrome

Eligibility Criteria

Sex: All

Age: 12 - 65

Healthy Volunteers: Accepted

Interventions

BCX17725

Placebo

Study Details

Brief 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.

Conditions

Netherton Syndrome

Study ID

NCT06539507

Start date

Sep 26, 2024

Status verified date

Jun, 2026

Completion date

Dec, 2026

Anticipated

Primary completion date

Oct, 2026

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 12 - 65

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

Study Design

Enrollment

78 participants

Anticipated

Allocation

Randomized

Intervention Model

Sequential

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Part 1 - BCX17725 single dose

Participants randomized to BCX17725 will receive BCX17725 as a single dose in sequential ascending dose cohorts

experimental: Part 1 - placebo single dose

Participants randomized to placebo will receive placebo as a single dose

experimental: Part 2 - BCX17725 multiple doses

Participants randomized to BCX17725 will receive BCX17725 as multiple doses in sequential ascending dose cohorts

experimental: Part 2 - placebo multiple doses

Participants randomized to placebo will receive placebo as multiple doses

experimental: Part 3 - BCX17725 multiple doses

Participants will receive BCX17725 as multiple doses

experimental: Part 4 - BCX17725 multiple doses

Participants will receive BCX17725 as multiple doses

Interventions

BCX17725

BCX17725 for injection

Placebo

Placebo for injection

Primary outcome measure

  • 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 ]

Central Contacts and Locations

Central contacts

BioCryst Pharmaceuticals, Inc.

+1 919 859 1302clinicaltrials@biocryst.com

Locations

Stanford University School of Medicine

Recruiting

Palo Alto, California, United States, 94304

Contacts

Chaw-Ning Lee

650-497-7230cnlee06@stanford.edu

Thomas Buschbacher

352-278-7603tbusch@stanford.edu

Therapeutics Clinical Research

Recruiting

San Diego, California, United States, 92123

Contacts

Hector Bailon

858-571-6800hbailon@therapeuticsresearch.com

Yale Center for Clinical Investigation

Recruiting

New Haven, Connecticut, United States, 06519

Contacts

Nicole Olszewski

203-785-5505nicole.olszewski@yale.edu

Northwestern Dermatology CTU

Recruiting

Chicago, Illinois, United States, 60611

Contacts

312-503-5944NUderm-research@northwestern.edu

Dawes Fretzin Clinical Research Group, LLC

Recruiting

Indianapolis, Indiana, United States, 46250

Contacts

Joannie Prusa

317-516-5030jprusa@ecommunity.com

More Information

Sponsor

BioCryst Pharmaceuticals

Last update posted

Aug 27, 2026

Last verified

Jun, 2026

Keywords

  • First-in-human
  • Single ascending dose
  • Multiple ascending dose
  • Pharmacokinetics
  • Placebo control
  • Netherton syndrome

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by BioCryst Pharmaceuticals on 2026-08-27.