First-in-Human gene therapy trial targets inherited heart weakness

NCT ID NCT07218887

Summary

This early-stage study is testing a new, one-time gene therapy called ALXN2350 for adults with a specific genetic form of heart muscle disease (BAG3 mutation). The main goals are to find a safe dose and see if the treatment helps control the disease. It will involve a small group of participants who will receive a single infusion and be monitored closely for safety and heart function.

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.

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for BAG3 MUTATION ASSOCIATED DILATED CARDIOMYOPATHY are added.

Our safety recommendation!

By submitting, you agree to our Terms of use

Contacts and locations

Study contacts

  • Contact

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

Locations

  • Research Site

    RECRUITING

    Birmingham, Alabama, 35233, United States

  • Research Site

    RECRUITING

    Boston, Massachusetts, 02115, United States

  • Research Site

    RECRUITING

    Cincinnati, Ohio, 45219, United States

  • Research Site

    RECRUITING

    Portland, Oregon, 97239-3098, United States

  • Research Site

    RECRUITING

    Houston, Texas, 77030, United States

  • Research Site

    RECRUITING

    Barcelona, 08035, Spain

  • Research Site

    RECRUITING

    Majadahonda, 28222, Spain

Conditions

Explore the condition pages connected to this study.