Experimental drug aims to boost brain function in fragile x syndrome

NCT ID NCT05358886

Completed Disease control Sponsor: Tetra Discovery Partners Source: ClinicalTrials.gov ↗

First seen Jul 10, 2026 · Last updated Jul 10, 2026

Summary

This study tests an experimental drug called BPN14770 (zatolmilast) in men aged 18 to 45 with Fragile X syndrome, a genetic condition that causes intellectual disability and behavioral challenges. The trial compares the drug against a placebo to see if it improves cognitive abilities, such as vocabulary and reading, as well as daily functioning and language skills. Participants take the drug twice daily for several months, and researchers measure changes using standard cognitive tests and caregiver reports.

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 could become the first approved treatment to improve cognitive and daily functioning in adults with Fragile X syndrome.
What could go wrong: This is a phase 3 trial, but previous experimental treatments for Fragile X have failed. The drug may not show significant benefit over placebo, and side effects are possible.

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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
Cincinnati Childrens 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
UC Davis Health System

Sacramento, California, 95817, United States
University of Kansas Medical Center

Kansas City, Kansas, 66160, United States
University of Massachusetts Medical School

Worcester, Massachusetts, 01655, 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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