Treatment of Fragile X Syndrome with a Neuroactive Steroid
Abstract
The protocol was approved by the UC Davis (UCD) IRB in September 2011 and approval for the renewal was obtained in July 2012. The protocol is being reviewed by the USAMRMC Office of Research Protections, Human Research Protection Office. We completed an initial site visit with Marinus in October 2011 to discuss drug manufacturing and the protocol. Dr Frank Kooy completed his Year 1 visit in February 2012. He presented on Towards targeted therapy in the fragile X syndrome using GABAergic drugs . At this meeting, the UCD team and Marinus team, along with Dr Kooy discussed the protocol. We created the electronic database and we are currently testing it. The DSMB was established. We have reviewed our subject database and identified approximately 60 potential subjects for screening. We had a site initiation visit on August 10, 2012 with Marinus. The anticipated drug delivery date to the UCD Investigational Drug Service is August 24, 2012. We continue to have bi-weekly conference calls with Marinus to operationalize the protocol and to prepare for the start of the study once HRPO approves the protocol. Dr Hagerman has also presented on targeted treatments at 5 different conferences during Year 1 of this project.
Document Details
- Document Type
- Technical Report
- Publication Date
- Aug 01, 2012
- Accession Number
- ADA580938
Entities
People
- Randi Hagerman
Organizations
- University of California