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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE1

Total Enrollment

78 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-09-26

Study Completion Date

2026-12-31

Brief Summary

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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.

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Detailed Description

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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

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Netherton Syndrome

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

SEQUENTIAL

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 Study Purpose

TREATMENT

Blinding Strategy

DOUBLE

Participants Investigators

Parts 1 and 2 will be blinded; Parts 3 and 4 will be open label

Study Groups

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Part 1 - BCX17725 single dose

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

Group Type EXPERIMENTAL

BCX17725

Intervention Type DRUG

BCX17725 for injection

Part 1 - placebo single dose

Participants randomized to placebo will receive placebo as a single dose

Group Type EXPERIMENTAL

Placebo

Intervention Type DRUG

Placebo for injection

Part 2 - BCX17725 multiple doses

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

Group Type EXPERIMENTAL

BCX17725

Intervention Type DRUG

BCX17725 for injection

Part 2 - placebo multiple doses

Participants randomized to placebo will receive placebo as multiple doses

Group Type EXPERIMENTAL

Placebo

Intervention Type DRUG

Placebo for injection

Part 3 - BCX17725 multiple doses

Participants will receive BCX17725 as multiple doses

Group Type EXPERIMENTAL

BCX17725

Intervention Type DRUG

BCX17725 for injection

Part 4 - BCX17725 multiple doses

Participants will receive BCX17725 as multiple doses

Group Type EXPERIMENTAL

BCX17725

Intervention Type DRUG

BCX17725 for injection

Interventions

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BCX17725

BCX17725 for injection

Intervention Type DRUG

Placebo

Placebo for injection

Intervention Type DRUG

Eligibility Criteria

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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

Minimum Eligible Age

12 Years

Maximum Eligible Age

65 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

Yes