Testing an inhaled therapy for alpha-1 antitrypsin deficiency

Plain-English translation of NCT06049082 on ClinicalTrials.gov ↗ · Source last updated July 2025 · Translation generated June 2026 · How we translate trials

Phase 1 — Testing in a small group (usually 20–80 people) to find a safe dose and watch for side effects.

What is this trial?

This is an early-stage study testing KB408, an inhaled medication designed to deliver a healthy gene directly into the lungs of people with alpha-1 antitrypsin deficiency (AATD). The medication uses a modified, harmless virus to help the lungs produce a protein that is missing or not working properly in people with this genetic condition. Researchers want to see if the treatment is safe and whether it can help improve how well the lungs work.

Alpha-1 antitrypsin deficiency is a genetic disease where the lungs gradually lose function because they lack a protective protein. Currently, some patients receive intravenous infusions of this protein, but this medication aims to offer a new option by delivering the gene directly to the lungs through inhalation, potentially making treatment easier and more effective.

Do you qualify?

You likely qualify if…

you are between 18 and 70 years old
you have been diagnosed with alpha-1 antitrypsin deficiency with specific genetic types (PI*ZZ or PI*ZNull)
your oxygen levels are at least 92% while breathing normal air at rest
you are in stable overall health aside from your lung condition
you are willing to follow a stable treatment plan and stay on it during the study

You likely don't qualify if…

you have had a lung infection or flare-up in the past six weeks
your lung function is very severely reduced (less than 40% predicted)
you have smoked cigarettes, used tobacco, or vaped within the past six months
you have significant liver disease or problems with liver function
you have an active oral herpes infection or have had one in the past month

What participation looks like

You would visit the study clinic to receive the inhaled medication through a nebulizer (a device that turns liquid medicine into a mist you breathe in). Depending on which group you join, you may receive a single dose or multiple doses spread over time. Throughout the study, you will have blood tests, breathing tests, and check-ins to monitor your safety and how your lungs respond to the treatment. The study team will closely watch for any side effects.

AI-generated summary from trial data · Jun 15, 2026 · Not medical advice

Trial locations(3 sites)

United States

University of Florida, Gainesville, Gainesville, Florida

Medical University of South Carolina, Charleston, South Carolina

Renovatio Clinical, The Woodlands, Texas