Experimental drug aims to boost thinking skills in teens with fragile x

NCT ID NCT05163808

Completed Symptom relief Sponsor: Tetra Discovery Partners Source: ClinicalTrials.gov ↗

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.

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Conditions

The condition(s) this trial relates to.

fragile X syndrome

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

FRAGILE X SYNDROME

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

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