Inhaled therapy aims to halt lung decline in rare genetic disease
NCT ID NCT04204252
First seen Nov 01, 2025 · Last updated May 16, 2026 · Updated 27 times
Summary
This study tests whether daily inhaled AAT (a protein) can slow lung function loss in people with Alpha-1 antitrypsin deficiency, a genetic condition that causes emphysema. About 220 adults with moderate to severe lung disease will receive either the drug or a placebo for 2 years, then all will receive the drug for 2 more years. The goal is to see if the treatment preserves breathing ability and lung density.
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Beaumont Hospital
RECRUITINGDublin, D09 YD60, Ireland
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Canisius Wilhelmina Hospital (CWZ)
RECRUITINGNijmegen, 6532SZ, Netherlands
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Leiden University Medical Center (LUMC)
RECRUITINGLeiden, ZA, 2333ZA, Netherlands
Contact Email: •••••@•••••
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Royal Infirmary of Edinburgh
RECRUITINGEdinburgh, EH16 4SA, United Kingdom
Contact Email: •••••@•••••
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Skåne University Hospital
RECRUITINGMalmö, SE-20502, Sweden
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Tays Central Hospital
RECRUITINGTampere, Finland
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact Phone: •••-•••-•••• Email: •••••@•••••
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University Hospital (UZ) Leuven
RECRUITINGLeuven, Belgium
Contact Phone: •••-•••-•••• Email: •••••@•••••
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University Hospital Southampton NHS Foundation Trust
RECRUITINGSouthampton, SO16 6YD, United Kingdom
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact Phone: •••-•••-•••• Email: •••••@•••••
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University Hospitals Birmingham NHS Foundation Trust
RECRUITINGBirmingham, B15 2GW, United Kingdom
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact Phone: •••-•••-•••• Email: •••••@•••••
Conditions
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