First pill for genetic lung & liver disease enters human testing

NCT ID NCT06738017

Summary

This is a very early study to check if a new oral drug called BMN 349 is safe for people with Alpha-1 Antitrypsin Deficiency, a genetic condition that can damage the lungs and liver. A small group of 12 adults with specific genetic types of the disease will take a single dose of either the drug or a placebo (a dummy pill). Researchers will closely monitor participants for any side effects and measure how the drug moves through the body.

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

  • Medpace Clinical Pharmacology Unit

    RECRUITING

    Cincinnati, Ohio, 45227, United States

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

  • NHS Lothian

    RECRUITING

    Edinburgh, UK, United Kingdom

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

  • Nottingham University Hospitals

    NOT_YET_RECRUITING

    Nottingham, UK, United Kingdom

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

  • Royal Free London NHS Foundation Trust

    RECRUITING

    London, UK, United Kingdom

    Contact Email: •••••@•••••

  • Saint Louis University

    RECRUITING

    St Louis, Missouri, 63110, United States

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

  • The Medical University of South Carolina

    RECRUITING

    Charleston, South Carolina, 29425, United States

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

  • University Hospital Southampton NHS Foundation Trust

    RECRUITING

    Southampton, UK, United Kingdom

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

  • University of California, San Diego

    RECRUITING

    San Diego, California, 92037, United States

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

Conditions

Explore the condition pages connected to this study.