Navigation Links
New clinical trial to examine medication to treat social withdrawal in Fragile X and autism

(CHICAGO) Children and adults with social withdrawal due to Fragile X syndrome, the most common cause of inherited intellectual disability and the most common known single gene cause of autism, may benefit from an experimental drug under study by pediatric neurologists at Rush Children's Hospital at Rush University Medical Center.

Rush is the only site in Illinois and one of 21 hospitals in the U.S. participating in the trial for Fragile X.

Fragile X syndrome is a neurodevelopmental disorder characterized by impaired social function, cognition and speech, as well as attention deficits and anxiety.

People with Fragile X, autism or autism spectrum disorders often display social impairment including social withdrawal and anxiety and have difficulty communicating and interacting with others. Although there are behavioral and psychological interventions, there are no approved medications for the treatment of social or communication difficulties in Fragile X, autism and autism spectrum disorders.

"The condition can be severely debilitating and this medication has the potential to play a much needed role in improving the core symptoms of fragile X syndrome and helping patients and their families achieve an improved quality of life," said Dr. Elizabeth Berry-Kravis, pediatric neurologist at Rush and principal investigator of the study.

The study is sponsored by Seaside Therapeutics, Inc, and will test the efficacy, safety and tolerability of the drug called STX209 (arbaclofen).

Racemic baclofen (mixture of arbaclofen and esbaclofen) is approved by the FDA to treat spasticity and stiff muscles due to cerebral palsy or other forms of brain or spinal cord injury, but arbaclofen, the more active form of baclofen, is not FDA approved.

"There is some evidence that the medication may help with social behaviors in people with developmental disabilities," said Berry-Kravis, who is a professor of pediatrics, neurology and biochemistry at Rush University.

Participants in the randomized, double-blind, placebo controlled phase III trial will be randomized to receive either the study drug, STX209, or a placebo. The clinical trial will include screening, treatment, withdrawal of medication, and a follow-up period. Subjects who complete the study may be eligible to enroll in a subsequent open-label study in which all subjects are treated with STX209.

STX209 has been studied in a previous small placebo-controlled trial in children and adults with fragile X syndrome and showed evidence of benefit for social withdrawal.

"Previous research has found that from one-quarter to one-half of people with fragile X have autism spectrum disorders," said Berry-Kravis.

"This trial is exciting, because it represents the culmination of 20 years work in fragile X research since discovery of the fragile X gene in 1991," said Kravis. "We're not expecting this to cure fragile X or autism, but it's a very important step in the development of new treatments."


Contact: Deb Song
Rush University Medical Center

Related medicine news :

1. PACS improves radiologists use of clinical decision support systems
2. Wake Forest Baptist conducts clinical study for insomnia using new technology
3. Damon Runyon Cancer Research Foundation awards $3.45 million to 9 top young clinical investigators
4. Neuralstem updates clinical trial progress
5. Universal flu vaccine clinical trials show promise
6. Pioneering stem cell bandage receives approval for clinical trial
7. NIH grant for the move toward clinical trials targeting the lysosomal storage disease MPSIIIB
8. Clinical trials for new Alzheimers disease treatment to be awarded European Commission €6m funding
9. Dubai to host largest clinical bone meeting in the Middle East and Africa region
10. Top 5 list helps primary care doctors make wiser clinical decisions
11. Researchers examine procedure utilization trends in patients with clinically localized renal masses
Post Your Comments:
(Date:10/13/2015)... ... ... In an age where the cost of energy is rising, Susquehanna Health’s ... 2009. The Williamsport Regional Medical Center (WRMC) campus has grown 50 percent since 2010, ... , According to Facilities Management Administrative Director Dieter Reichmann, this is the result of ...
(Date:10/13/2015)... ... 2015 , ... Dr. Poneh Ghasri, dentist in West Hollywood , comments ... research, which was conducted at the Dental Institute at King's College London in the ... during pregnancy and future dental health in the child. For years, researchers have long ...
(Date:10/13/2015)... New York, NY (PRWEB) , ... October 13, 2015 , ... ... Android®. The app helps individuals find others to train with, participate in local fitness ... the App Store and Google Play. , “As high medical costs drive us to ...
(Date:10/13/2015)... ... 13, 2015 , ... Leading Internet marketing product review website ... Academy" training course . Singal's new program, in which he reveals his strategies ... the Internet are weighing in with reviews. , "Considering the fairly high number ...
(Date:10/13/2015)... ... October 13, 2015 , ... Altec Products, ... Title Sponsorship of Synergy 2015. The annual WennSoft KEY2ACT user conference will take ... and will unite customers, partners, WennSoft team members and sponsors to facilitate the ...
Breaking Medicine News(10 mins):
(Date:10/13/2015)... -- Data Science Automation (DSA), a system integration and automation engineering ... in the United Kingdom (UK) as ... . The decision to open the new office comes ... customers in the medical device industry throughout the UK. ... have had tremendous success over the last several years in ...
(Date:10/13/2015)... 13, 2015  SRI International has been awarded ... over five years by the National Institute of ... National Institutes of Health, for the development of ... effects of radiation exposure. Under this multi-year contract, ... International will provide services, facilities, expertise and capabilities ...
(Date:10/13/2015)... 2015  Nanomedical Diagnostics, a biotech company developing ... diagnostics, announces the completion of a Series A ... Ventures. --> ... monitoring and diagnostic platforms that empower individuals to ... will enable the company to commercially release AGILE ...
Breaking Medicine Technology: