Navigation Links
New clinical trial to examine medication to treat social withdrawal in Fragile X and autism
Date:7/20/2011

(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
deb_song@rush.edu
312-942-0588
Rush University Medical Center
Source:Eurekalert

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:
*Name:
*Comment:
*Email:
(Date:5/26/2017)... ... ... After raising nearly $30,000 on Kickstarter , about three-times its original campaign ... crowdfunding price on Indiegogo . , “Along with creating an anti-stress gadget to ... fidget toy to the market that was made of superior quality and wouldn’t break ...
(Date:5/26/2017)... ... , ... Silver Birch of Hammond, a new assisted lifestyle community, has welcomed ... of land at 5620 Sohl Avenue in Hammond, serves older adults who need some ... and one-bedroom apartments. Each of the private apartments at Silver Birch features a kitchenette, ...
(Date:5/26/2017)... ... May 26, 2017 , ... Dr. Alex Rabinovich, a highly-skilled oral ... new, informational blog post on insurance options. If a Bay Area patient has to ... save time and money. Visiting an in-network provider for a second opinion can ensure ...
(Date:5/26/2017)... ... ... to the Cross”: the personal journey of Bob Massey and his faith in the Lord. ... and Margaret Massey. Bob Massey is small in stature but big in character. ... love for others is apparent in all of his life decisions. , “I’m in ...
(Date:5/26/2017)... ... ... What Happened in the Garden of Eden”: retells the stories of three Bible figures in ... published author, Penelope Colt, mother, trader, horse farmer, artist and a former GM journeyman. ... At six, they moved to Dayton, Ohio, where Penny graduated high school. At ...
Breaking Medicine News(10 mins):
(Date:5/26/2017)... 25, 2017  In response to the opioid epidemic ... Relief is working with Pfizer to make up to ... cost to community health centers, free and charitable clinics, ... "Pfizer has a long-standing commitment to improving ... patient safety through educational activities," said Caroline Roan ...
(Date:5/23/2017)... Calif. , May 23, 2017  Leaf ... most innovative medical devices for pressure ulcer prevention, ... the American Association of Critical Care Nurses, National ... May 22-25. The Leaf Patient Monitoring ... specifically for the hospital environment.  The system seamlessly ...
(Date:5/22/2017)... 22, 2017  As the specialty pharmacy industry ... to make the revolutionary shift from volume-based to ... ensuring positive patient outcomes and shaping the future ... focus away from clinical trials and toward data ... specialty drug therapy utilization in precise patient populations. ...
Breaking Medicine Technology: