Treatment of Fragile X Syndrome with a Neuroactive Steroid.

摘要

We renewed the protocol with the UC Davis (UCD) IRB and the USAMRMC HRPO. This past year (Jul 15, 2013 to Jul 14, 2014), twenty-five subjects were enrolled, bringing total enrollment to thirty-six. Twenty-six have completed and there have been seven early terminations and two screen failures. Data-entry is done regularly. No serious adverse events have occurred. The first DSMB meeting took place in September 2013, and the trial was allowed to proceed. We plan to increase recruitment to 4 subjects per month for Year 4 of the project to meet the enrollment goal of sixty. 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.