New hope for kids with Muscle-Wasting disease in major drug trial

NCT ID NCT07287982

Summary

This study is testing a new drug called ARGX-119 in children aged 5 to 18 with spinal muscular atrophy (SMA). The goal is to see if the drug is safe and if it can improve muscle strength and walking ability when given along with the child's current SMA treatments. For the first 24 weeks, some children will get the new drug and some will get a placebo, and then all children will have the option to receive the new drug for up to two more years.

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

  • Ann & Robert H. Lurie Children's Hospital of Chicago

    NOT_YET_RECRUITING

    Chicago, Illinois, 60611, United States

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

    Contact

  • Arkansas Children's Hospital

    NOT_YET_RECRUITING

    Little Rock, Arkansas, 72202, United States

    Contact

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

  • Boston Children's Hospital

    NOT_YET_RECRUITING

    Boston, Massachusetts, 02115, United States

    Contact

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

  • Children's Hospital Philadelphia - Neurology

    NOT_YET_RECRUITING

    Philadelphia, Pennsylvania, 19104, United States

    Contact

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

  • Childrens Hospital of The Kings Daughters

    NOT_YET_RECRUITING

    Norfolk, Virginia, 23507, United States

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

    Contact

  • Columbia University Herbert Irving Comprehensive Cancer Center

    NOT_YET_RECRUITING

    New York, New York, 10032, United States

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

    Contact

  • Connecticut Children's Medical Center

    NOT_YET_RECRUITING

    Hartford, Connecticut, 06106, United States

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

    Contact

  • Neurology Rare Disease Center

    RECRUITING

    Flower Mound, Texas, 75028, United States

    Contact

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

  • Rady Childrens Hospital

    NOT_YET_RECRUITING

    San Diego, California, 92123, United States

    Contact

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

  • Rare Disease Research FL LLC

    RECRUITING

    Kissimmee, Florida, 34746, United States

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

    Contact

  • Rare Disease Research NC, LLC

    RECRUITING

    Hillsborough, North Carolina, 27278, United States

    Contact

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

  • St. Jude Children's Research Hospital

    NOT_YET_RECRUITING

    Memphis, Tennessee, 38105, United States

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

    Contact

  • Stanford University Medical Center

    NOT_YET_RECRUITING

    Stanford, California, 94305, United States

    Contact

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

  • Texas Children's Hospital

    NOT_YET_RECRUITING

    Houston, Texas, 77030, United States

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

    Contact

  • The Curators of the University of Missouri on behalf of University of Missouri Health Care

    NOT_YET_RECRUITING

    Columbia, Missouri, 65212, United States

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

    Contact

  • The Johns Hopkins Hospital

    NOT_YET_RECRUITING

    Baltimore, Maryland, 21205, United States

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

    Contact

  • University of Iowa Stead Family Children's Hospital

    NOT_YET_RECRUITING

    Iowa City, Iowa, 52242, United States

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

    Contact

Conditions

Explore the condition pages connected to this study.