Can an inhaled protein save lungs in Alpha-1? new trial aims to find out
NCT ID NCT04204252
First seen Jun 26, 2026 · Last updated Aug 13, 2026 · Updated 2 times
Summary
This study tests whether inhaling alpha-1 antitrypsin (AAT) daily can slow lung function loss in people with Alpha-1 deficiency and moderate-to-severe lung disease. 220 adults will receive either the drug or a placebo for two years, then all will receive the drug for two more years. Researchers will measure lung function, lung density on CT scans, and quality of life.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- Alpha-1 antitrypsin (AAT) for inhalation
- What this could lead to
- If it works, this could provide a new inhaled treatment to slow lung function decline in people with Alpha-1 antitrypsin deficiency.
- What could go wrong
- This is a Phase 3 trial, but results are not yet known. The drug may not slow lung decline better than placebo, and daily inhalation requires commitment.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for ALPHA 1-ANTITRYPSIN DEFICIENCY are added.
By submitting, you agree to our Terms of use
Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
-
Beaumont Hospital
Dublin, D09 YD60, Ireland
-
Canisius Wilhelmina Hospital (CWZ)
Nijmegen, 6532SZ, Netherlands
-
Leiden University Medical Center (LUMC)
Leiden, ZA, 2333ZA, Netherlands
-
Royal Infirmary of Edinburgh
Edinburgh, EH16 4SA, United Kingdom
-
Skåne University Hospital
Malmö, SE-20502, Sweden
-
Tays Central Hospital
Tampere, Finland
-
University Hospital (UZ) Leuven
Leuven, Belgium
-
University Hospital Southampton NHS Foundation Trust
Southampton, SO16 6YD, United Kingdom
-
University Hospitals Birmingham NHS Foundation Trust
Birmingham, B15 2GW, United Kingdom
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- AI model could predict who needs a lung transplant for rare emphysema
- Could a simple shot replace IV drips for Alpha-1 patients?
- Hidden liver harm: study tracks silent damage in genetic disorder
- Promising liver drug trial halted early: what it means for patients
- Gene-Editing shot aims to fix lung and liver damage in rare disease
- New drug AIR-001 enters first human tests for rare lung condition