Testing a new way to give a protein replacement therapy by injection

Plain-English translation of NCT07555483 on ClinicalTrials.gov ↗ · Source last updated July 2026 · Translation generated June 2026 · How we translate trials

Phase 3 — Testing in thousands of people, comparing the treatment against what doctors currently use. This is the last big step before approval.

What is this trial?

This trial is testing Alpha1-Proteinase Inhibitor (a protein replacement therapy) given as a subcutaneous injection—meaning under the skin—compared to the standard way of receiving it through an IV (a vein). The study wants to see if the newer injection method works just as well and is safe and tolerable for people with Alpha-1 Antitrypsin Deficiency, a genetic condition that damages the lungs over time. Two different dose levels will be tested.

Currently, this medication is given through the vein, which requires regular hospital or clinic visits. The trial is exploring whether giving the treatment by injection under the skin instead could be just as effective, possibly more convenient, and easier for patients to manage at home.

Do you qualify?

You likely qualify if…

you have been diagnosed with Alpha-1 Antitrypsin Deficiency based on genetic testing
your blood levels of the protective protein are below a certain threshold (less than 11 μM)
your lung function shows moderate to severe airflow obstruction (roughly 25–80% of normal)
you are willing to stop any current Alpha-1 therapy and use only the study medication
you are able to give written informed consent and understand the study procedures

You likely don't qualify if…

you have had a moderate or severe lung disease flare-up in the past 4 weeks
you have had a lung or liver transplant, or are waiting for one
you have had lung surgery in the past year (except for a biopsy)
you have severe heart disease, advanced lung scarring, active cancer, or other serious medical conditions
you are pregnant, breastfeeding, or unwilling to use reliable birth control if you can become pregnant

What participation looks like

You would be randomly assigned to one of two treatment groups. For the first 8 weeks, you would receive the standard intravenous (IV) infusion at a clinic or hospital. Then, for the next 8 weeks, you would switch to receiving injections under your skin at home or a clinic. Throughout the study, you will have blood tests and breathing tests to measure how your body processes the medication and to check for any side effects. The study doctors will monitor you closely to ensure the treatment is working safely.

AI-generated summary from trial data · Jun 3, 2026 · Not medical advice

Testing a new way to give a protein replacement therapy by injection

What is this trial?

Do you qualify?

What participation looks like

Trial locations