A Study of the Safety, Tolerability, Pharmacokinetics, and Immunogenicity of BCX17725
Purpose
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.
Condition
- Netherton Syndrome
Eligibility
- Eligible Ages
- Between 12 Years and 65 Years
- Eligible Sex
- All
- Accepts Healthy Volunteers
- Yes
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
Study Design
- Phase
- Phase 1
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Sequential Assignment
- Intervention Model Description
- In Parts 1 and 2, participants will be randomized to BCX17725 or placebo under double-blind conditions. In Parts 3 and 4, participants will receive open-label BCX17725.
- Primary Purpose
- Treatment
- Masking
- Double (Participant, Investigator)
- Masking Description
- Parts 1 and 2 will be blinded; Parts 3 and 4 will be open label
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
|
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 |
|
Recruiting Locations
San Diego, California 92123
New Haven, Connecticut 06519
Chicago, Illinois 60611
Indianapolis, Indiana 46250
More Details
- NCT ID
- NCT06539507
- Status
- Recruiting
- Sponsor
- BioCryst Pharmaceuticals
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.