Alcobra Ltd will be conducting a clinical trial of Metadoxine Extended Release (MDX) in adults and adolescents, 15 to 55 years old, with Fragile X syndrome. The planned Phase IIb MDX clinical trial will be a multi-center, randomized, placebo-controlled study, conducted primarily in the US. The study is supported by positive data collected from multiple earlier animal studies using metadoxine. Results from these studies demonstrated significant improvement in behavioral and cognitive outcomes in mice based on testing and performance of memory, learning, and social interaction.
There are few individuals in the US with Fragile X; as well as few treatment options; that is why the FDA has granted “Orphan Drug” designation. It is possible that participants may not benefit from participation in this trial. The drug is experimental and subjects will be informed of all safety concerns prior to participating in the trial.
Elizabeth M. Berry-Kravis, MD, PhD, Professor of Biochemistry, Neurological Sciences and Pediatrics at Rush University Medical Center in Chicago, Illinois and Principal Investigator in this study commented, “Patients with Fragile X syndrome currently have limited treatment options, with no FDA approved medications. The positive findings reported from the preclinical studies, together with information that is known on the mechanism of action of metadoxine, suggest that MDX may be helpful in treating cognitive symptoms in these patients. This Phase IIb study should provide us with important insights into the potential role of MDX in this condition.”
The trial will initiate in the second quarter of 2014 and will be conducted at approximately 10 centers throughout the continental US and one center in Israel. Specific information about center locations and the protocol can be found at Clintrials.gov at the following link clinicaltrials.gov/ct2/show/NCT02126995 as well as on the fragilex.org website.
For more specific information about the upcoming clinical trial, please contact:
Anthony Robinson, Director, Clinical Operations
ARobinson@alcobra-pharma.com