NCT06539507

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

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

• Status: Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 78 (estimated)
• Sponsor: BioCryst Pharmaceuticals · Industry
• Sex: All
• Age: 12 Years – 65 Years
• Healthy volunteers: Accepted

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.

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.

Conditions

• Netherton Syndrome

Interventions

Type	Name	Description
DRUG	BCX17725	BCX17725 for injection
DRUG	Placebo	Placebo for injection

Timeline

• Start date: 2024-09-26
• Primary completion: 2026-10-01
• Completion: 2026-12-01
• First posted: 2024-08-06
• Last updated: 2026-03-31

Locations

12 sites across 5 countries: United States, Australia, France, Germany, Netherlands

Regulatory

• FDA-regulated drug study