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Recruiting Phase 1 Interventional

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

ClinicalTrials.gov ID: NCT06049082

Public ClinicalTrials.gov record NCT06049082. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.

ClinicalTrials.gov public records Last synced Sep 7, 2026, 8:34 AM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Official title

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

Brief summary

Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.

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.

Study identification

NCT ID
NCT06049082
Recruitment status
Recruiting
Study type
Interventional
Phase
Phase 1
Lead sponsor
Krystal Biotech, Inc.
Industry
Enrollment
15 participants

Conditions and interventions

Eligibility (public fields only)

Age range
18 Years to 70 Years
Sex
All
Healthy volunteers
Healthy volunteers not accepted

This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.

Study timeline

Start date
Feb 14, 2024
Primary completion
Nov 30, 2025
Completion
Nov 30, 2026
Last update posted
Jul 21, 2025

2024 – 2026

United States locations

U.S. sites
3
U.S. states
3
U.S. cities
3
Facility City State ZIP Site status
University of Florida, Gainesville Gainesville Florida 32610 Recruiting
Medical University of South Carolina Charleston South Carolina 29425 Recruiting
Renovatio Clinical The Woodlands Texas 77380 Recruiting

Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.

About this trial record page

What this page shows
Public field values for ClinicalTrials.gov record NCT06049082, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
What this page does not do
No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
Where the data comes from
Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
Last refresh
Last update posted Jul 21, 2025 · Synced Sep 7, 2026

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Open the official record

The complete protocol, eligibility criteria, and contact information for NCT06049082 live on ClinicalTrials.gov.

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