First human trial tests potential new treatment for rare muscle disease

NCT ID NCT05027269

Summary

This study tested the safety and effects of a new drug called AOC 1001 in adults with myotonic dystrophy type 1, a genetic muscle-wasting disease. Thirty-nine participants received either the drug or a placebo through an IV to see how their bodies handled it and if it affected the disease. The main goal was to check for side effects and understand 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.

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for DM1 are added.

Our safety recommendation!

By submitting, you agree to our Terms of use

Contacts and locations

Locations

  • Kansas University Medical Center

    Kansas City, Kansas, 66205, United States

  • Ohio State University

    Columbus, Ohio, 43221, United States

  • Stanford University

    Palo Alto, California, 94304, United States

  • University of California Los Angeles

    Los Angeles, California, 90095, United States

  • University of Colorado

    Denver, Colorado, 80045, United States

  • University of Florida

    Gainesville, Florida, 32608, United States

  • University of Rochester Medical Center

    Rochester, New York, 14642, United States

  • Virginia Commonwealth University

    Richmond, Virginia, 23298, United States

Conditions

Explore the condition pages connected to this study.