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

NCT06539507 · tracked via the Priya Life Science USA tracker

Sponsor

BioCryst Pharmaceuticals

Phase

Phase 1

Started

2024-09-26

Last updated

2026-08-27

Condition(s) studied

Netherton Syndrome

Investigational drug(s) / intervention(s)

BCX17725Placebo

BCX17725: BCX17725 for injection

Placebo: Placebo for injection

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

Sex

ALL

Min age

12 Years

Max age

65 Years

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

Primary outcome measure(s)

Incidence of treatment-emergent adverse events (TEAEs) — 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
Change from baseline in Ichthyosis Area and Severity Index (IASI) score at Week 12 (Part 4) — 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.

Trial sites (12)

Facility	City	Region	Status
Stanford University School of Medicine	Palo Alto	California	Recruiting
Therapeutics Clinical Research	San Diego	California	Recruiting
Yale Center for Clinical Investigation	New Haven	Connecticut	Recruiting
Northwestern Dermatology CTU	Chicago	Illinois	Recruiting
Dawes Fretzin Clinical Research Group, LLC	Indianapolis	Indiana	Recruiting
Westmead Hospital - Department of Dermatology	Sydney	New South Wales	Recruiting
Nucleus Network	Brisbane	Queensland	Completed
Veracity Clinical Research	Brisbane	Queensland	Recruiting
Hôpital Saint-Louis	Paris	France	Recruiting
Universitätsklinikum Heidelberg	Heidelberg	Germany	Not Yet Recruiting
Maastricht Universitair Medisch Centrum (MUMC+)	Maastricht	Netherlands	Recruiting
Erasmus Universitair Medisch Centrum (EMC)	Rotterdam	Netherlands	Recruiting

Other trials for the same condition

Safety, Tolerability and Efficacy of QRX003 Lotion in Subjects With Netherton Syndrome Active, not recruiting

Phase 2/3 Clinical Study of QRX003 Lotion in Subjects With Netherton Syndrome Recruiting

Clinical Study of QRX003 Lotion in Subjects With Netherton Syndrome Recruiting

Open Label, Safety and Efficacy Study of QRX003 Lotion in Subjects With Netherton Syndrome Recruiting

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06539507 on ClinicalTrials.gov ↗ ← All trials in the USA