New drug shows promise for fragile x teens in landmark trial

NCT ID NCT05163808

First seen Feb 04, 2026 · Last updated May 15, 2026 · Updated 17 times

Summary

This study tested an experimental drug called BPN14770 in 163 teenage boys with Fragile X syndrome. The goal was to see if the drug could improve thinking skills and daily life abilities compared to a placebo. The trial had two parts: first to check how the drug moves through the body, then a blinded comparison of the drug versus placebo over 13 weeks.

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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

Locations

  • Amnova Clinical Research

    Irvine, California, 92604, United States

  • Boston Children's Hospital

    Boston, Massachusetts, 02115, United States

  • Children's Hospital Colorado

    Aurora, Colorado, 80045, United States

  • Cincinatti Children's Hospital Medical Center

    Cincinnati, Ohio, 45229, United States

  • Clinic for Special Children

    Strasburg, Pennsylvania, 17579, United States

  • Emory University School of Medicine

    Atlanta, Georgia, 30322, United States

  • Greenwood Genetic Center

    Greenville, South Carolina, 29605, United States

  • Icahn School of Medicine at Mount Sinai Hospital

    New York, New York, 10029, United States

  • Kennedy Krieger Institute

    Baltimore, Maryland, 21205, United States

  • Rush University Medical Center

    Chicago, Illinois, 60612, United States

  • Suburban Research Associates

    Media, Pennsylvania, 19063, United States

  • Thompson Autism & Neurodevelopment Center - CHOC

    Orange, California, 92868, United States

  • U Mass

    Worcester, Massachusetts, 01655, United States

  • UC Davis

    Sacramento, California, 95817, United States

  • University of Kansas Medical Center

    Kansas City, Kansas, 66160, United States

  • University of Miami

    Miami, Florida, 33136, United States

  • University of Utah and Primary Childrens Hospital

    Salt Lake City, Utah, 84113, United States

Conditions

Explore the condition pages connected to this study.