Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT06539507 | A Study of the Safety, Tolerability, Pharmacokinetics, and Immunogenicity of BCX17725 | PHASE1 | RECRUITING | 78 | — | — | Sep 26, 2024 | Dec 1, 2026 | Jun 5, 2026 | 12 | United States, Australia +3 |
Incidence of TEAEs as assessed by Common Terminology Criteria for Adverse Events (CTCAE) from screening through end-of-study (EOS) in each study part
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.
| Arm | Type | Description |
|---|---|---|
| Part 1 - BCX17725 single dose | EXPERIMENTAL | Participants randomized to BCX17725 will receive BCX17725 as a single dose in sequential ascending dose cohorts |
| Part 1 - placebo single dose | EXPERIMENTAL | Participants randomized to placebo will receive placebo as a single dose |
| Part 2 - BCX17725 multiple doses | EXPERIMENTAL | Participants randomized to BCX17725 will receive BCX17725 as multiple doses in sequential ascending dose cohorts |
| Part 2 - placebo multiple doses | EXPERIMENTAL | Participants randomized to placebo will receive placebo as multiple doses |
| Part 3 - BCX17725 multiple doses | EXPERIMENTAL | Participants will receive BCX17725 as multiple doses |
| Part 4 - BCX17725 multiple doses | EXPERIMENTAL | Participants will receive BCX17725 as multiple doses |
| Name | Type | Description |
|---|---|---|
| BCX17725 | DRUG | BCX17725 for injection |
| Placebo | DRUG | Placebo for injection |
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 scor...