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| ID | Type | Description | Link |
|---|---|---|---|
| U1111-1303-9510 | Other Identifier | WHO UTN | |
| 2025-521973-16 | EudraCT Number |
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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Part 1 - BCX17725 single dose | Experimental | Participants randomized to BCX17725 will receive BCX17725 as a single dose in sequential ascending dose cohorts |
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| Part 1 - placebo single dose | Experimental | Participants randomized to placebo will receive placebo as a single dose |
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| Part 2 - BCX17725 multiple doses | Experimental | Participants randomized to BCX17725 will receive BCX17725 as multiple doses in sequential ascending dose cohorts |
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| Part 2 - placebo multiple doses | Experimental | Participants randomized to placebo will receive placebo as multiple doses |
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| Part 3 - BCX17725 multiple doses | Experimental | Participants will receive BCX17725 as multiple doses |
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| Part 4 - BCX17725 multiple doses | Experimental |
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| BCX17725 | Drug | BCX17725 for injection |
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| Measure | Description | Time Frame |
|---|---|---|
| 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 |
| Measure | Description | Time Frame |
|---|---|---|
| Maximum observed serum concentration (Cmax) | Cmax of BCX17725 | Up to Day 64 (Part 1), Day 92 (Part 2), and Day 78 (Part 3) |
| Time to maximum observed serum concentration (Tmax) | Time to Cmax of BCX17725 |
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Key Inclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| BioCryst Pharmaceuticals, Inc. | Contact | +1 919 859 1302 | clinicaltrials@biocryst.com |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Stanford University School of Medicine | Not yet recruiting | Palo Alto | California | 94304 | United States |
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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.
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Parts 1 and 2 will be blinded; Parts 3 and 4 will be open label
Participants will receive BCX17725 as multiple doses |
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| Placebo | Drug | Placebo for injection |
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| 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) | AUC0-t of BCX17725 | Up to Day 64 (Part 1), Day 92 (Part 2), and Day 78 (Part 3) |
| Terminal elimination half-life (t1/2) | Terminal elimination half-life (t1/2) of BCX17725 | 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 | Incidence of ADAs to BCX17725 | Day 1 pre-dose and up to Day 64 (Part 1), Day 92 (Part 2), and Day 78 (Part 3) |
| Change from baseline in Investigator Global Assessment (IGA) score at Week 12 (Part 4) | IGA assesses the overall severity of a participant's NS skin disease based on a 5-point scale (0, clear; 1, almost clear; 2, mild; 3, moderate; and 4, severe). Higher scores indicate worse clinical outcome. | From baseline to Week 12 |
| Change from baseline in Worst Itch Numerical Rating Score (NRS) at Week 12 (Part 4) | Worst Itch Numerical Rating Scale (NRS) is a self-rated, single-item scale designed for assessing the worst itch in the past 7 days. The scale uses an 11-point NRS, scored from 0 (no itch) to 10 (worst possible itch). Higher scores indicate worse clinical outcome. | From baseline to Week 12 |
| Incidence of TEAEs (Part 4) | Incidence of TEAEs as assessed by CTCAE | From baseline through Week 20 |
| Serum concentrations of BCX17725 (Part 4) | Serum concentrations of BCX17725 | From baseline through Week 20 |
| Therapeutics Clinical Research | Recruiting | San Diego | California | 92123 | United States |
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| Yale Center for Clinical Investigation | Recruiting | New Haven | Connecticut | 06519 | United States |
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| Northwestern Dermatology CTU | Recruiting | Chicago | Illinois | 60611 | United States |
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| Dawes Fretzin Clinical Research Group, LLC | Recruiting | Indianapolis | Indiana | 46250 | United States |
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| Westmead Hospital - Department of Dermatology | Recruiting | Sydney | New South Wales | 2145 | Australia |
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| Nucleus Network | Completed | Brisbane | Queensland | 4006 | Australia |
| Veracity Clinical Research | Recruiting | Brisbane | Queensland | 4102 | Australia |
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| Hôpital Saint-Louis | Recruiting | Paris | 75010 | France |
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| Universitätsklinikum Heidelberg | Not yet recruiting | Heidelberg | 69120 | Germany |
| Maastricht Universitair Medisch Centrum (MUMC+) | Recruiting | Maastricht | 6229 HX | Netherlands |
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| Erasmus Universitair Medisch Centrum (EMC) | Recruiting | Rotterdam | 3015 GD | Netherlands |
| ID | Term |
|---|---|
| D056770 | Netherton Syndrome |
| ID | Term |
|---|---|
| D000015 | Abnormalities, Multiple |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D016113 | Ichthyosiform Erythroderma, Congenital |
| D007057 | Ichthyosis |
| D012868 | Skin Abnormalities |
| D012873 | Skin Diseases, Genetic |
| D030342 | Genetic Diseases, Inborn |
| D007232 | Infant, Newborn, Diseases |
| D007642 | Keratosis |
| D012871 | Skin Diseases |
| D017437 | Skin and Connective Tissue Diseases |
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