Evaluating the safety and effects of BCX17725 in patients with Netherton Syndrome
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
This study is testing a new drug called BCX17725 to see if it is safe and effective for people with Netherton Syndrome.
Quick facts
| Phase | Phase 1 |
|---|---|
| Study type | Interventional |
| Enrollment | 78 (estimated) |
| Ages | 12 Years to 65 Years |
| Sex | All |
| Sponsor | BioCryst Pharmaceuticals Industry-sponsored |
| Locations | 12 sites (Palo Alto, California and 11 other locations) |
| Trial ID | NCT06539507 on ClinicalTrials.gov |
What this trial studies
This is a first-in-human, Phase 1 study designed to assess the safety, tolerability, pharmacokinetics, and immunogenicity of BCX17725. The study consists of three parts: the first two parts involve healthy adult participants receiving single and multiple ascending doses of the drug in a randomized, placebo-controlled manner, while the third part focuses on participants with Netherton Syndrome receiving multiple doses in an open-label format. The goal is to gather comprehensive data on how the drug behaves in the body and its potential effects on the target condition.
Who should consider this trial
Good fit: Ideal candidates for this study are adults aged 18 to 65 with a confirmed diagnosis of Netherton Syndrome.
Not a fit: Patients who do not have Netherton Syndrome or are outside the specified age range will not benefit from this study.
Why it matters
Potential benefit: If successful, this study could provide a new treatment option for patients suffering from Netherton Syndrome.
How similar studies have performed: While this is a first-in-human study, similar approaches in pharmacokinetics and immunogenicity assessments have shown promise in other conditions.
Eligibility criteria
Show full inclusion / exclusion criteria
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
Where this trial is running
Palo Alto, California and 11 other locations
- Stanford University School of Medicine — Palo Alto, California, United States (Not_yet_recruiting)
- Therapeutics Clinical Research — San Diego, California, United States (Recruiting)
- Yale Center for Clinical Investigation — New Haven, Connecticut, United States (Recruiting)
- Northwestern Dermatology CTU — Chicago, Illinois, United States (Recruiting)
- Dawes Fretzin Clinical Research Group, LLC — Indianapolis, Indiana, United States (Recruiting)
- Westmead Hospital - Department of Dermatology — Sydney, New South Wales, Australia (Recruiting)
- Nucleus Network — Brisbane, Queensland, Australia (Active_not_recruiting)
- Veracity Clinical Research — Brisbane, Queensland, Australia (Recruiting)
- Hôpital Saint-Louis — Paris, France (Not_yet_recruiting)
- Universitätsklinikum Heidelberg — Heidelberg, Germany (Not_yet_recruiting)
- Maastricht Universitair Medisch Centrum (MUMC+) — Maastricht, Netherlands (Not_yet_recruiting)
- Erasmus Universitair Medisch Centrum (EMC) — Rotterdam, Netherlands (Recruiting)
Study contacts
- Study coordinator: BioCryst Pharmaceuticals, Inc.
- Email: clinicaltrials@biocryst.com
- Phone: +1 919 859 1302
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.