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

NCT06049082 · Status: RECRUITING · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 15

Last updated 2025-07-22

No results posted yet for this study

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

Interventions

DRUG

KB408 (Nebulization)

Nebulized solution of KB408, a replication-defective HSV-1 vector expressing full length human SERPINA1

Sponsors & Collaborators

  • Krystal Biotech, Inc.

    lead INDUSTRY

Principal Investigators

  • David Sweet, MD, PhD · Director of Clinical Development

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Max Age
70 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-02-15
Primary Completion
2025-12-31
Completion
2026-12-31
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06049082 on ClinicalTrials.gov