FX-Learn Clinical Trial for Children with Fragile X

FX-Learn Clinical Trial for Children with Fragile X

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.

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Purposeful and FRAXA Partnership Leads to Clinical Trial

Purposeful and FRAXA Partnership Leads to Clinical Trial

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 upcoming trial, which will test the effects of an available drug and a nutritional supplement taken together.

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

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

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.

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Fragile X Clinical Trial of New PDE4D Inhibitor from Tetra

Fragile X Clinical Trial of New PDE4D Inhibitor from Tetra

FRAXA Research Foundation has funded a clinical trial of an investigational new drug, led by Dr. Elizabeth Berry-Kravis at the Rush Fragile X Clinic in Chicago. This trial will treat 30 adult males with Fragile X syndrome with a PDE4D allosteric inhibitor from Tetra Discovery Partners using in a crossover design, so that everyone gets active drug for part of the time and placebo for part of the time.

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

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

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.

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Clinical Trials Outcome Measures

Clinical Trials Outcome Measures

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.

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Pilot Clinical Trial of Lithium in Fragile X Shows Promising Results

Pilot Clinical Trial of Lithium in Fragile X Shows Promising Results

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.

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