New drug shows promise for Long-Term fragile x management
NCT ID NCT05367960
First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This 4-year study tests the long-term safety of the drug BPN14770 (zatolmilast) in 314 people with Fragile X syndrome who completed earlier trials. Participants include adult males and adolescents. The main goal is to see if the drug is safe over time, with secondary measures looking at cognitive function. This is an open-label study, so everyone receives the drug.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
BPN14770 (zatolmilast)
What this could lead to
If successful, this study could show that BPN14770 is safe to take long-term, potentially leading to a daily medication that helps manage Fragile X syndrome symptoms.
What could go wrong
This is an open-label extension study, meaning everyone knows they are getting the drug, which can bias results. It focuses on safety, not proof of effectiveness, and side effects or lack of benefit may still emerge.
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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Cincinnati 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
New York, New York, 10029, United States
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Kennedy Krieger Institute
Baltimore, Maryland, 21205, United States
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MIND Institute UC Davis Medical Center
Sacramento, California, 95817, 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, 92828, United States
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U Mass
Worcester, Massachusetts, 01655, 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 Children's Hospital
Salt Lake City, Utah, 84113, United States