Treatment of Fragile X Syndrome with a Neuroactive Steroid
Abstract
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.
Document Details
- Document Type
- Technical Report
- Publication Date
- Aug 01, 2013
- Accession Number
- ADA592346
Entities
People
- Randi Hagerman
Organizations
- California State University, Sacramento