Testing a new treatment for severe alpha-1 antitrypsin deficiency

Plain-English translation of NCT07227207 on ClinicalTrials.gov ↗ · Source last updated September 2026 · Translation generated September 2026 · How we translate trials

Phase 1/2 — A combined trial that checks safety and dosing while also starting to look at whether the treatment works.

What is this trial?

This trial is testing a new medication called designed to help adults with severe alpha-1 antitrypsin deficiency, a genetic condition where your body doesn't make enough of a protective protein in your lungs and liver. The study will evaluate whether the medication is safe and whether it helps improve your health over time.

Alpha-1 antitrypsin deficiency is a serious genetic condition that causes progressive damage to the lungs and liver, and current treatment options are limited. This trial is exploring whether this new medication can effectively treat the underlying problem rather than just managing symptoms.

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 the PiZZ genetic type
you have lung disease (emphysema, bronchiectasis, or reduced lung function) and/or liver disease related to this condition
you have not smoked for at least 6 months and are willing to stay smoke-free throughout the study
you are either new to alpha-1 antitrypsin treatment or have stopped any previous related treatments at least 4 weeks before joining

You likely don't qualify if…

you have HIV or active hepatitis B or C infection
you have had recent severe lung infections requiring hospitalization or IV antibiotics within the past 6 months
you have received an organ transplant or are on a waiting list for one
you have had previous gene therapy using viral vectors or any treatment intended to permanently change your DNA
you have liver disease from causes other than alpha-1 antitrypsin deficiency, or you have signs of cirrhosis

What participation looks like

You will visit the study clinic multiple times for check-ups, blood tests, and breathing or liver function assessments. The study has three parts: the first tests increasing doses of the medication to find the safest dose, the second expands the study with more participants, and the third tests a repeat dose. Throughout your participation, the research team will monitor you closely to ensure your safety and to measure how well the medication works.

AI-generated summary from trial data · Sep 20, 2026 · Not medical advice

Trial locations(8 sites)

United States

Clinical Study Center, Jacksonville, Florida

Clinical Study Center, Boston, Massachusetts

Clinical Study Center, Rochester, Minnesota

Clinical Study Center, Charleston, South Carolina

Clinical Study Center, Seattle, Washington