New hope for boys with Muscle-Wasting disease: experimental drug enters advanced testing

NCT ID NCT05540860

Summary

This study is testing an oral medication called EDG-5506 (sevasemten) in children with Duchenne muscular dystrophy (DMD), a serious muscle-wasting condition. Researchers want to see if the drug is safe, how it moves through the body, and if it can reduce signs of muscle damage. The trial has two parts: first, some children get the drug and some get a placebo for 12 weeks, then all children can receive the drug in a long-term extension phase for up to four years.

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Contacts and locations

Locations

  • Arkansas Children's Hospital

    Little Rock, Arkansas, 72202, United States

  • Children's Hospital Colorado

    Aurora, Colorado, 80045, United States

  • Cincinnati Children's Hospital

    Cincinnati, Ohio, 45229, United States

  • Cook Children's Medical Center

    Fort Worth, Texas, 76104, United States

  • Kennedy Krieger Institute

    Baltimore, Maryland, 21205, United States

  • Nationwide Children's Hospital

    Columbus, Ohio, 43205, United States

  • Rare Disease Research

    Atlanta, Georgia, 30329, United States

  • UC Davis Medical Center

    Sacramento, California, 95817, United States

  • UCLA Medical Center

    Los Angeles, California, 90095, United States

  • University of Florida

    Gainesville, Florida, 32610, United States

  • University of Iowa

    Iowa City, Iowa, 52242, United States

  • University of Kansas Medical Center

    Kansas City, Kansas, 66160, United States

  • University of Massachusetts Memorial Medical Center

    Worcester, Massachusetts, 01605, United States

  • Washington University School of Medicine

    St Louis, Missouri, 63110, United States

Conditions

Explore the condition pages connected to this study.