Rush University Medical Center Archives • Fragile X Research

Fragile X Clinical Trial of New PDE4D Inhibitor from Tetra

FRAXA Research Foundation 2018 Grants • 2019 Grants • 2020 Grants • Berry-Kravis, Elizabeth • Clinical • Has Results • Research Grant Results • Rush University Medical Center • Trials and Studies November 20, 2022January 26, 2023Alzheimer's, BPN14770, cyclic AMP, drug tolerance, FDA, FRAXA-DVI, NIH, PDE4, Pierce Family Fragile X Foundation, sound, Tetra Therapeutics

With a $200,043 grant from FRAXA Research Foundation, Dr. Elizabeth Berry-Kravis completed a successful Phase 2 clinical trial of a PDE4 inhibitor for adult men with Fragile X syndrome. This trial treated 30 males, 18-45 years of age with a new PDE4D allosteric inhibitor from Tetra Discovery Partners using a crossover design, so that everyone got active drug for part of the time and placebo for part of the time.

FX-Learn Clinical Trial for Children with Fragile X

FRAXA Research Foundation Berry-Kravis, Elizabeth • Rush University Medical Center • Trials and Studies November 9, 2021November 18, 2021AFQ056, mGluR5, NIH, Novartis

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

FRAXA Research Foundation 2021 Grants • 2022 Grants • Berry-Kravis, Elizabeth • Clinical • Current Research Grants • Drug Repurposing • Rush University Medical Center • Seminar Series • Trials and Studies October 19, 2021January 26, 2023drug repurposing, FRAXA-DVI, serotonin, sound

Can a combination of drugs make a meaningful difference for people with Fragile X? A new clinical trial is going to find out. 15-20 adult men with Fragile X will be included in this trial to test the effects of an available drug and a nutritional supplement taken together.

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

FRAXA Research Foundation 2021 Grants • 2022 Grants • Berry-Kravis, Elizabeth • Biomarkers • Current Research Grants • Richter, Joel • Rush University Medical Center • Shah, Sneha • University of Massachusetts September 2, 2021November 8, 2022mRNAs, Pierce Family Fragile X Foundation

The Pierce Family Fragile X Foundation helping to fund a joint grant to the University of Massachusetts Medical School and Rush University Medical Center in exploring biomarkers to measure whether a treatment is helping and determine how to optimize treatment for each individual.

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

FRAXA Research Foundation Berry-Kravis, Elizabeth • Rush University Medical Center July 10, 2018August 5, 2021Alzheimer's, autism, BPN14770, FDA, NIH, PDE4, seizure, sleep, Tetra Therapeutics

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.

New Fragile X Clinical Trial for Children Launching in June 2017

Theodore Coutilish Berry-Kravis, Elizabeth • Research Updates • Rush University Medical Center • Trials and Studies October 31, 2016February 25, 2019AFQ056, mGluR5, NIH, Novartis

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

FRAXA Research Foundation 2001-2005 Grants • 2006-2010 Grants • 2011 Grants • Berry-Kravis, Elizabeth • Biomarkers • Completed Research Grants • Rush University Medical Center February 22, 2011May 20, 2020lithium, mGluR5, NIH

With $281,824 in funding from FRAXA Research Foundation from 2002-2011, Dr. Berry-Kravis at the Rush University Medical Center attempted to validate a new automated video tracking system for quantifying physical activity as an outcome measure for Fragile X clinical trials.

Pilot Clinical Trial of Lithium in Fragile X Shows Promising Results

FRAXA Research Foundation 2001-2005 Grants • Berry-Kravis, Elizabeth • Clinical • Has Results • Research Grant Results • Rush University Medical Center • Trials and Studies November 14, 2010May 20, 2020lithium, minocycline, NIH, PAK (p21-activated kinase)

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.