080210xf's Blog

L'X fragile sera vaincu | Fragile X will be conquered

Clinical trial for fragile X syndrome treatment

health.universityofcalifornia.edu |

UC Davis will participate in a nationwide trial targeting adolescents and young adults with fragile X syndrome.

The UC Davis MIND Institute is participating in a nationwide, multicenter clinical trial of an investigational medication that has shown clinically meaningful improvements in social functioning in adolescents and young adults with fragile X syndrome.

“This investigational drug has the potential to play a much-needed role in improving the core symptoms of fragile X syndrome and helping patients and families achieve an improved quality of life,” said Randi Hagerman, medical director of the UC Davis MIND Institute and the clinical trial lead investigator.

The medication is known as STX209, or Arbaclofen. It is an oral selective gamma-amino butyric acid type B (GABA-B) receptor agonist being developed by Seaside Therapeutics Inc. of Cambridge, Mass., which is sponsoring the trial.

The study will measure the efficacy, safety and tolerability of Arbaclofen for patients between 12 and 25 years of age with fragile X syndrome, who will receive the medication or a placebo followed by a withdrawal period. The UC Davis MIND Institute will enroll 12 participants.

Fragile X syndrome is the leading genetic cause of intellectual disability and the leading single-gene cause of autism. It is the result of the mutation of a single gene, the fragile X mental retardation 1 (FMR1) gene on the X chromosome.

The FMR1 gene produces a protein needed for normal brain development. Individuals with fragile X syndrome lack this protein and, as a result, the majority of affected individuals have significant intellectual disabilities and require lifetime care.

There currently are no Food and Drug Administration (FDA)-approved treatments for patients with fragile X syndrome.

For further information concerning the clinical trial, please contact Lindsey Partington at (916) 703-0471 or lindsey.partington@ucdmc.ucdavis.edu.

At the UC Davis MIND Institute, world-renowned scientists engage in research to find improved treatments as well as the causes and cures for autism, attention-deficit/hyperactivity disorder, fragile X syndrome, Tourette syndrome and other neurodevelopmental disorders. Advances in neuroscience, molecular biology, genetics, pharmacology and behavioral sciences are making inroads into a better understanding of brain function. The UC Davis MIND Institute draws from these and other disciplines to conduct collaborative, multidisciplinary research. For more information, visit mindinstitute.ucdavis.edu.

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