New drug shows promise for Long-Term fragile x management

NCT ID NCT05367960

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

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.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Get updates

Get notified about this study

Our safety recommendation!

By submitting, you agree to our Terms of use

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 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

New York, New York, 10029, United States
Kennedy Krieger Institute

Baltimore, Maryland, 21205, United States
MIND Institute UC Davis Medical Center

Sacramento, California, 95817, 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, 92828, United States
U Mass

Worcester, Massachusetts, 01655, 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 Children's Hospital

Salt Lake City, Utah, 84113, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.