Pilot Clinical Trial of Lithium in Fragile X

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 2008: Open-label treatment trial of lithium to target the underlying defect in fragile X syndrome
Elizabeth Berry-Kravis, MD, PhD, Fragile X researcher
$65,000 Grant
Elizabeth Berry-Kravis, MD, PhD
Principal Investigator
Rush University Medical Center
2005 FRAXA Research Grant

by Elizabeth Berry-Kravis, 2/26/2011

Fifteen subjects with Fragile X will be treated with lithium to get pilot data to justify a larger trial of lithium. The lithium treatment will be added on to other medications the individuals enrolled are already taking, and treatment will be for at least two months and up to a year if the lithium is helpful. Tests will be given at the beginning of the study, after two months of treatment, and after a year of treatment for those who are treated a full year. Side effects will be monitored closely to make sure that lithium is safe in the Fragile X population. We will use a battery of behavioral and thinking tests which we are validating specifically for use in medication trials in Fragile X. We will try new physiological tests to measure over-stimulation and eye aversion, a special blood test that may serve as a biomarker for improvement in the cellular defect in Fragile X, and some new tests of associative learning. These new tests, in combination with some of the tests that worked best in our previous Ampakine study, should allow us to know if lithium is improving some of the behaviors and learning deficits seen in Fragile X syndrome. If the new tests are able to measure a medication response in this study, we can then use them for other future studies.

Results of the pilot trial can be found here.

A 2014 review of this study by Zhonghua Liu and Carolyn Beebe Smith  from the National Institute of Mental Health can be found here.

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