Fragile X Clinical Trial of AZD7325 in Adults
Craig Erickson, MD
Principal Investigator
Cincinnati Children’s Hospital
Cincinnati, OH
2017-2021 Grant Funding: $51,000
Summary
With a $51,000 grant from FRAXA Research Foundation, Dr. Craig Erickson conducted a double-blind, placebo-controlled clinical trial of AZD7325 in adults ages 18-50 with Fragile X syndrome. The compound is an investigational new drug from AstraZeneca which boosts GABA receptors. Dr. Erickson was able to secure $60,000 in matching funds from Cincinnati Children’s Hospital to fully fund this trial.
The Science
Many studies have shown that the brain’s GABA system is impaired in Fragile X syndrome, and a number of medications that target GABA have shown promise for Fragile X. (See GABA and glutamate: the Yin and Yang of Fragile X.)
Dr. Erickson is testing an investigational new compound, AZD7325, which selectively boosts GABA(A). This compound is available for testing and development through AstraZeneca’s open innovation program. This initial trial will also use a number of innovative biomarkers which we hope will be useful outcome measures for future trials.
