Actively Recruiting
A Phase 1/1b Study Evaluating Safety, Tolerability, Pharmacokinetics, and Immunogenicity of BCX17725 in Healthy Adults and Participants With Netherton Syndrome
Led by BioCryst Pharmaceuticals · Updated on 2026-06-05
78
Participants Needed
12
Research Sites
8 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating BCX17725 in a first-in-human Phase 1/1b clinical trial to assess its safety, tolerability, pharmacokinetics, and immune response in healthy adults and participants with Netherton syndrome. The study includes four parts: Parts 1 and 2 focus on single and multiple ascending doses in healthy adults, Part 3 involves multiple doses in adults with Netherton syndrome, and Part 4 assesses the effectiveness, safety, and tolerability in adults and adolescents with Netherton syndrome over 12 weeks, followed by an 8-week post-treatment follow-up. Treatment involves randomized administration of BCX17725 or placebo in Parts 1 and 2, with single and multiple ascending doses respectively. Parts 3 and 4 use multiple doses of BCX17725 in participants with Netherton syndrome in open-label designs. Specifically, Part 4 includes intravenous and/or subcutaneous multiple doses over 12 weeks. Participants receive study drug or placebo according to the assigned group during each part. Participants will undergo monitoring for adverse events from screening through end of study, with assessments including skin severity scores such as the Ichthyosis Area and Severity Index and Investigator Global Assessment. Pharmacokinetics and immune response markers like anti-drug antibodies are measured up to specified days in each part. Part 4 includes follow-up assessments through Week 20. The total study duration varies per part, with detailed evaluations of safety, drug levels, and clinical skin outcomes.
CONDITIONS
Brief Title
A Study of the Safety, Tolerability, Pharmacokinetics, and Immunogenicity of BCX17725
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male or non-pregnant, non-lactating female aged 18 to 55 years (Parts 1 and 2), 18 to 65 years (Part 3), or 12 to 65 years (Part 4)
- Confirmed diagnosis of Netherton syndrome (Parts 3 and 4)
- Investigator Global Assessment (IGA) score of 3 or higher (Parts 3 and 4)
- Ichthyosis Area and Severity Index (IASI) score of 16 or higher (Part 4)
- Body mass index (BMI) between 18 and 30 kg/m² (Parts 1 and 2)
- Estimated glomerular filtration rate (eGFR) of at least 90 mL/min/1.73 m² (Parts 1 and 2) or at least 60 mL/min/1.73 m² (Part 3)
- Agree to follow contraception requirements from screening until 90 days after last dose
- Expected to comply with all study procedures and restrictions
You will not qualify if you...
History of severe allergic reactions to study medication Currently pregnant or breastfeeding Recent participation in another clinical trial within the last 30 days Presence of uncontrolled medical conditions that could affect safety
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 12 weeks depending on study part
Participants receive BCX17725 or placebo as single or multiple ascending doses depending on the study part, including multiple dose administrations in participants with Netherton syndrome.
Multiple visits depending on dosing schedule, including single-dose and multiple-dose administrations
Duration - Up to 8 weeks post-treatment
Participants are monitored for safety, tolerability, and pharmacokinetics after treatment ends, including an 8-week post-treatment follow-up period for participants with Netherton syndrome.
Multiple follow-up visits up to 8 weeks after treatment
Trial Site Locations
Total: 12 locations
1
Stanford University School of Medicine
Palo Alto, California, United States, 94304
Not Yet Recruiting
2
Therapeutics Clinical Research
San Diego, California, United States, 92123
Actively Recruiting
3
Yale Center for Clinical Investigation
New Haven, Connecticut, United States, 06519
Actively Recruiting
4
Northwestern Dermatology CTU
Chicago, Illinois, United States, 60611
Actively Recruiting
5
Dawes Fretzin Clinical Research Group, LLC
Indianapolis, Indiana, United States, 46250
Actively Recruiting
6
Westmead Hospital - Department of Dermatology
Sydney, New South Wales, Australia, 2145
Actively Recruiting
7
Nucleus Network
Brisbane, Queensland, Australia, 4006
Completed
8
Veracity Clinical Research
Brisbane, Queensland, Australia, 4102
Actively Recruiting
9
Hôpital Saint-Louis
Paris, France, 75010
Actively Recruiting
10
Universitätsklinikum Heidelberg
Heidelberg, Germany, 69120
Not Yet Recruiting
11
Maastricht Universitair Medisch Centrum (MUMC+)
Maastricht, Netherlands, 6229 HX
Not Yet Recruiting
12
Erasmus Universitair Medisch Centrum (EMC)
Rotterdam, Netherlands, 3015 GD
Actively Recruiting
Research Team
B
BioCryst Pharmaceuticals, Inc.
How is the study designed?
Study Type
INTERVENTIONAL
Masking
DOUBLE
Allocation
RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
6
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