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
Location: See all (12) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 1
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.
Participation Requirements• 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
Stanford University School of Medicine
NOT_YET_RECRUITING
Palo Alto
Therapeutics Clinical Research
RECRUITING
San Diego
Yale Center for Clinical Investigation
RECRUITING
New Haven
Northwestern Dermatology CTU
RECRUITING
Chicago
Dawes Fretzin Clinical Research Group, LLC
RECRUITING
Indianapolis
Nucleus Network
ACTIVE_NOT_RECRUITING
Brisbane
Veracity Clinical Research
RECRUITING
Brisbane
Westmead Hospital - Department of Dermatology
RECRUITING
Sydney
Hôpital Saint-Louis
NOT_YET_RECRUITING
Paris
Universitätsklinikum Heidelberg
NOT_YET_RECRUITING
Heidelberg
Maastricht Universitair Medisch Centrum (MUMC+)
NOT_YET_RECRUITING
Maastricht
Erasmus Universitair Medisch Centrum (EMC)
RECRUITING
Rotterdam
Primary
BioCryst Pharmaceuticals, Inc.
clinicaltrials@biocryst.com
+1 919 859 1302
Start Date: 2024-09-26
Estimated Completion Date: 2026-12
Target number of participants: 78
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
Leads: BioCryst Pharmaceuticals