First-in-Human trial tests inhaled gene therapy for rare lung disease

NCT ID NCT06633757

Summary

This early-stage study is testing an inhaled gene therapy called RCT1100 in adults with Primary Ciliary Dyskinesia (PCD) caused by specific DNAI1 gene mutations. The trial aims to see if the treatment improves mucus clearance from the lungs and is safe for participants. Researchers will measure how well the therapy works and monitor for any side effects in 12 adult volunteers.

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Contacts and locations

Locations

  • Copenhagen University Hospital - Rigshospitalet

    Copenhagen, 2100, Denmark

  • Münster University Hospital, Albert-Schweitzer-Campus 1

    Münster, North Rhine-Westphalia, 48149, Germany

  • UNC

    Chapel Hill, North Carolina, 27514, United States

Conditions

Explore the condition pages connected to this study.