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

NCT06539507 | Recruiting Phase 1 DMC FDA Drug

• 3 other identifiers: BCX17725-101U1111-1303-95102025-521973-16
• Study Type: interventional
• Target: 78
• Locations: 5 countries
• Sites: 12

Brief 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.

Conditions

Netherton Syndrome

Keywords

First-in-human; Single ascending dose; Multiple ascending dose; Pharmacokinetics; Placebo control; Netherton syndrome

Outcome Measures

Primary Outcomes (2)

• Incidence of treatment-emergent adverse events (TEAEs)

  Incidence of TEAEs as assessed by Common Terminology Criteria for Adverse Events (CTCAE) from screening through end-of-study (EOS) in each study part

  From screening through EOS (ie, through Day 78 in Parts 1 and 3, and Day 106 in Part 2)

• Change from baseline in Ichthyosis Area and Severity Index (IASI) score at Week 12 (Part 4)

  IASI measures the severity of erythema (IASI-E) and scaling (IASI-S); the maximum sub-scores for the IASI-E and IASI-S being 24, and the maximum total IASI score being 48. Higher scores indicate worse clinical outcome.

  From baseline to Week 12

Secondary Outcomes (9)

• Maximum observed serum concentration (Cmax)

  Up to Day 64 (Part 1), Day 92 (Part 2), and Day 78 (Part 3)

• Time to maximum observed serum concentration (Tmax)

  Up to Day 64 (Part 1), Day 92 (Part 2), and Day 78 (Part 3)

• Area under the serum concentration-time curve (AUC) from time 0 to the time of last measurable concentration (AUC0-t)

  Up to Day 64 (Part 1), Day 92 (Part 2), and Day 78 (Part 3)

• Terminal elimination half-life (t1/2)

  Up to Day 64 (Part 1), Day 92 (Part 2), and Day 78 (Part 3)

• Number of participants who are anti-drug antibody (ADA)-positive (baseline and post-baseline) and number of participants who have treatment-emergent ADAs

  Day 1 pre-dose and up to Day 64 (Part 1), Day 92 (Part 2), and Day 78 (Part 3)

Study Arms (6)

Part 1 - BCX17725 single dose

EXPERIMENTAL

Participants randomized to BCX17725 will receive BCX17725 as a single dose in sequential ascending dose cohorts

Drug: BCX17725

Part 1 - placebo single dose

EXPERIMENTAL

Participants randomized to placebo will receive placebo as a single dose

Drug: Placebo

Part 2 - BCX17725 multiple doses

EXPERIMENTAL

Participants randomized to BCX17725 will receive BCX17725 as multiple doses in sequential ascending dose cohorts

Drug: BCX17725

Part 2 - placebo multiple doses

EXPERIMENTAL

Participants randomized to placebo will receive placebo as multiple doses

Drug: Placebo

Part 3 - BCX17725 multiple doses

EXPERIMENTAL

Participants will receive BCX17725 as multiple doses

Drug: BCX17725

Part 4 - BCX17725 multiple doses

EXPERIMENTAL

Participants will receive BCX17725 as multiple doses

Drug: BCX17725

Interventions

BCX17725 DRUG

BCX17725 for injection

Part 1 - BCX17725 single dose; Part 2 - BCX17725 multiple doses; Part 3 - BCX17725 multiple doses; Part 4 - BCX17725 multiple doses

Placebo DRUG

Placebo for injection

Part 1 - placebo single dose; Part 2 - placebo multiple doses

Eligibility Criteria

• Age: 12 Years - 65 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

Sponsors & Collaborators

• BioCryst Pharmaceuticals: lead

Study Sites (12)

Stanford University School of Medicine

Palo Alto, California, 94304, United States

NOT YET RECRUITING

Therapeutics Clinical Research

San Diego, California, 92123, United States

RECRUITING

Yale Center for Clinical Investigation

New Haven, Connecticut, 06519, United States

RECRUITING

Northwestern Dermatology CTU

Chicago, Illinois, 60611, United States

RECRUITING

Dawes Fretzin Clinical Research Group, LLC

Indianapolis, Indiana, 46250, United States

RECRUITING

Westmead Hospital - Department of Dermatology

Sydney, New South Wales, 2145, Australia

RECRUITING

Nucleus Network

Brisbane, Queensland, 4006, Australia

ACTIVE NOT RECRUITING

Veracity Clinical Research

Brisbane, Queensland, 4102, Australia

RECRUITING

Hôpital Saint-Louis

Paris, 75010, France

NOT YET RECRUITING

Universitätsklinikum Heidelberg

Heidelberg, 69120, Germany

NOT YET RECRUITING

Maastricht Universitair Medisch Centrum (MUMC+)

Maastricht, 6229 HX, Netherlands

NOT YET RECRUITING

Erasmus Universitair Medisch Centrum (EMC)

Rotterdam, 3015 GD, Netherlands

RECRUITING

MeSH Terms

Conditions

Netherton Syndrome

Condition Hierarchy (Ancestors)

Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Ichthyosiform Erythroderma, Congenital; Ichthyosis; Skin Abnormalities; Skin Diseases, Genetic; Genetic Diseases, Inborn; Infant, Newborn, Diseases; Keratosis; Skin Diseases; Skin and Connective Tissue Diseases

Central Study Contacts

BioCryst Pharmaceuticals, Inc.

CONTACT

+1 919 859 1302; clinicaltrials@biocryst.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Masking Details: Parts 1 and 2 will be blinded; Parts 3 and 4 will be open label
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: 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.

Study Record Dates

• First Submitted: July 30, 2024
• First Posted: August 6, 2024
• Study Start: September 26, 2024
• Primary Completion (Estimated): October 1, 2026
• Study Completion (Estimated): December 1, 2026
• Last Updated: March 31, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will not share

Locations

US (5); FR (1); DE (1); NL (2); AU (3)