Treatment of Fragile X Syndrome with a Neuroactive Steroid
Annual rept. 15 Jul 2012-14 Jul 2013
CALIFORNIA STATE UNIV SACRAMENTO
Pagination or Media Count:
We renewed the UC Davis UCD IRB approval for the protocol and we are in the process of renewing it with USAMRMC HRPO. We received drug and placebo in August 2012 and began enrollment in November 2012. Thus far, eleven subjects have been enrolled with seven completed. No serious adverse events have occurred. Three subjects were enrolled at the beginning of Year 3 and four more subjects scheduled for screening between August and October 2013. The electronic database is completed and data-entry is done regularly. The first DSMB meeting is scheduled in September 2013. We are planning to adjust recruitment to 3-4 subjects per month for Years 3 and 4 of the project to account for the late start in enrollment. If we can demonstrate efficacy of ganaxolone in our outcome measures this will have a major impact on the treatment of fragile X syndrome and move us closer to a cure for these children.
- Anatomy and Physiology
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