NCT06049082

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

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

• Status: Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 15 (estimated)
• Sponsor: Krystal Biotech, Inc. · Industry
• Sex: All
• Age: 18 Years – 70 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Alpha 1-Antitrypsin Deficiency

Interventions

Type	Name	Description
DRUG	KB408 (Nebulization)	Nebulized solution of KB408, a replication-defective HSV-1 vector expressing full length human SERPINA1

Timeline

• Start date: 2024-02-15
• Primary completion: 2025-12-01
• Completion: 2026-12-01
• First posted: 2023-09-21
• Last updated: 2025-07-22

Locations

3 sites across 1 country: United States

Regulatory

• FDA-regulated drug study