Rush University Medical Center Archives • FRAXA Research Foundation

Fragile X Clinical Trial of New PDE4D Inhibitor from Tetra

November 20, 2022

A $200K FRAXA grant enabled a successful Phase 2 trial of a PDE4D inhibitor for adult men with Fragile X, showing strong cognitive gains without side effects or tolerance.

FX-Learn Clinical Trial for Children with Fragile X

November 9, 2021

Thirteen centers across the US enrolled children with Fragile X in a large-scale clinical trial of Novartis AFQ056. Dr. Elizabeth Berry-Kravis and colleagues aim to show that this targeted treatment — an mGluR5 blocker for Fragile X which failed in previous adult human trials — can be better evaluated by studying effects on learning in young children.

Purposeful and FRAXA Partnership Leads to Clinical Trial

October 19, 2021

AI and FRAXA-DVI identified a drug + supplement combo that reversed all Fragile X symptoms in mice. A clinical trial tested this in adults with Fragile X.

Alternative Splicing in White Blood Cells: A Biomarker for Fragile X Syndrome

September 2, 2021

This team found 1,600 blood-based Fragile X biomarkers that vary by individual—opening the door to personalized treatment and better ways to measure progress.

Tetra Discovery Partners Initiates Phase 2 Trial of BPN14770 in Fragile X Syndrome

July 10, 2018

This 2-Period Crossover Study of BPN14770 is accepting adults males with Fragile X syndrome at Rush University Medical Center in Chicago. Principal Investigator of the study is Elizabeth Berry-Kravis, MD, PhD.
A selective inhibitor of the phosphodiesterase type-4D (PDE4D), BPN14770 has shown the ability to improve the quality of connections between neurons and to improve multiple behavioral outcomes in the Fragile X mouse model.

Elizabeth Berry-Kravis, MD, PhD, Fragile X researcher

New Fragile X Clinical Trial for Children Launching in June 2017

October 31, 2016

Rush University Medical Center Professor Elizabeth M. Berry-Kravis, MD, PhD, has launched and is recruiting participants for a large-scale clinical trial to study effects of AFQ056, an mGluR5 blocker, on learning in young children.

Clinical Trials Outcome Measures

February 22, 2011

In Fragile X participants, low-dose lithium showed functional benefits and supported development of reliable biomarkers and behavioral assessment tools.

Pilot Clinical Trial of Lithium in Fragile X Shows Promising Results

November 14, 2010

With a $65,000 grant from FRAXA Research Foundation in 2005, Dr. Berry-Kravis at the Rush University Medical Center conducted a pilot clinical trial of lithium in 15 patients with Fragile X syndrome. Results published.