Experimental drug aims to boost thinking skills in teens with fragile x
NCT ID NCT05163808
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study tested a drug called zatolmilast (BPN14770) in 163 male adolescents aged 9 to 18 with Fragile X syndrome. The trial had two parts: one to check how the drug moves through the body, and another where some teens got the drug and others got a placebo. Researchers measured changes in thinking skills and daily function after 13 weeks.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
zatolmilast (also called BPN14770)
What this could lead to
If it works, this could point toward a treatment that improves cognitive and daily functioning in adolescents with Fragile X syndrome.
What could go wrong
This is an early-to-mid-stage trial with a small number of participants, so results may not confirm benefit. The drug may cause side effects or fail to show meaningful improvement over placebo.
Disclaimer
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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.
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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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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Amnova Clinical Research
Irvine, California, 92604, United States
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Boston Children's Hospital
Boston, Massachusetts, 02115, United States
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Children's Hospital Colorado
Aurora, Colorado, 80045, United States
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Cincinatti Children's Hospital Medical Center
Cincinnati, Ohio, 45229, United States
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Clinic for Special Children
Strasburg, Pennsylvania, 17579, United States
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Emory University School of Medicine
Atlanta, Georgia, 30322, United States
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Greenwood Genetic Center
Greenville, South Carolina, 29605, United States
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Icahn School of Medicine at Mount Sinai Hospital
New York, New York, 10029, United States
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Kennedy Krieger Institute
Baltimore, Maryland, 21205, United States
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Rush University Medical Center
Chicago, Illinois, 60612, United States
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Suburban Research Associates
Media, Pennsylvania, 19063, United States
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Thompson Autism & Neurodevelopment Center - CHOC
Orange, California, 92868, United States
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U Mass
Worcester, Massachusetts, 01655, United States
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UC Davis
Sacramento, California, 95817, United States
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University of Kansas Medical Center
Kansas City, Kansas, 66160, United States
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University of Miami
Miami, Florida, 33136, United States
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University of Utah and Primary Childrens Hospital
Salt Lake City, Utah, 84113, United States