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Inhaled therapy aims to halt lung decline in rare genetic disease

NCT ID NCT04204252

First seen Nov 01, 2025 · Last updated May 21, 2026 · Updated 28 times

Summary

This study tests whether daily inhaled AAT (a protein) can slow lung function loss in adults with Alpha-1 antitrypsin deficiency and moderate-to-severe lung disease. About 220 participants will receive either the drug or a placebo for two years, then all will receive the drug for two more years. The main goal is to see if treatment preserves breathing ability and lung density.

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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

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • Beaumont Hospital

    RECRUITING

    Dublin, D09 YD60, Ireland

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • Canisius Wilhelmina Hospital (CWZ)

    RECRUITING

    Nijmegen, 6532SZ, Netherlands

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • Leiden University Medical Center (LUMC)

    RECRUITING

    Leiden, ZA, 2333ZA, Netherlands

    Contact Email: •••••@•••••

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • Royal Infirmary of Edinburgh

    RECRUITING

    Edinburgh, EH16 4SA, United Kingdom

    Contact Email: •••••@•••••

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • Skåne University Hospital

    RECRUITING

    Malmö, SE-20502, Sweden

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • Tays Central Hospital

    RECRUITING

    Tampere, Finland

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • University Hospital (UZ) Leuven

    RECRUITING

    Leuven, Belgium

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • University Hospital Southampton NHS Foundation Trust

    RECRUITING

    Southampton, SO16 6YD, United Kingdom

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact Phone: •••-•••-•••• Email: •••••@•••••

  • University Hospitals Birmingham NHS Foundation Trust

    RECRUITING

    Birmingham, B15 2GW, United Kingdom

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact Phone: •••-•••-•••• Email: •••••@•••••

Conditions

Explore the condition pages connected to this study.