New drug shows promise for helping children with dwarfism grow

NCT ID NCT02724228

First seen Mar 17, 2026 · Last updated May 14, 2026 · Updated 8 times

Summary

This study looks at the long-term safety and effectiveness of a daily injection called BMN 111 in children with achondroplasia, a common form of dwarfism. About 30 children who already received BMN 111 for two years in a previous study will continue treatment. Researchers will track side effects and measure growth, body proportions, and final adult height.

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Contacts and locations

Locations

  • Ann and Robert H. Lurie Childrens Hospital of Chicago

    Chicago, Illinois, 60611, United States

  • Baylor College of Medicine

    Houston, Texas, 77030, United States

  • Children's Hospital & Research Center Oakland

    Oakland, California, 94609, United States

  • Guys & St. Thomas NHS Foundation Trust Evelina Hospital

    London, SE1 9RT, United Kingdom

  • Harbor - UCLA Medical Center

    Torrance, California, 90509, United States

  • Institut Necker

    Paris, 75015, France

  • Johns Hopkins McKusick - Institute of Genetic Medicine

    Baltimore, Maryland, 21287, United States

  • Murdoch Children's Research Institute

    Parkville, Victoria, 3052, Australia

  • Vanderbilt University

    Nashville, Tennessee, 37232-2578, United States

Conditions

Explore the condition pages connected to this study.