Clinical Trial

A Study of KB408 for the Treatment of Alpha-1 Antitrypsin Deficiency

Study acronym: Serpentine-1
Recruiting Phase 1
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Record status
This record was last updated July 22, 2025 (before its estimated December 2025 completion). Its status may not reflect the trial's current state.
Summary
The Sponsor is developing KB408, a replication-defective, non-integrating herpes simplex virus type 1 (HSV-1)-derived vector engineered to deliver functional full-length human SERPINA1 to the airways of people with alpha-1 antitrypsin deficiency (AATD) via nebulization. This study is designed to evaluate safety and pharmacodynamics of KB408 in adults with AATD with a PI\*ZZ or PI\*ZNull genotype. Three planned dose levels of KB408 will be evaluated in single dose escalation cohorts. Repeat dosing will be evaluated at the mid dose level. Subjects taking intravenous AAT augmentation therapy are not required to wash out from IV AAT in the low and mid dose cohorts. In the repeat dose and the high dose cohorts, subjects must wash out from IV AAT for at least 10 days, as applicable.
Protocol Amendment History 6 amendments
This ClinicalTrials.gov record has been amended 6 times since 2023-09-15; most recent amendment 2025-07-17.
Status change: Not Yet Recruiting → Recruiting 2024-02-01
Trial Details
NCT Number NCT06049082
Lead Sponsor Krystal Biotech, Inc.
Conditions Alpha 1-Antitrypsin Deficiency
Enrollment 15 participants
Start Date 2024-02-15
Primary Completion 2025-12 (estimated)
Study Completion 2026-12 (estimated)
Updated on ClinicalTrials.gov 2025-07-22