Feb. 19, 2009 Autism Speaks has announced initial results reported for the first industry-sponsored trial of the Autism Clinical Trials Network (ACTN), the Study of Fluoxetine in Autism (SOFIA). These results showed that fluoxetine was not effective for reducing repetitive behaviors in children and adolescents with Autistic Disorder as compared to placebo (non-medicinal sugar pill). The study was carried out in collaboration with study sponsor, Neuropharm Group Plc., a specialty pharmaceutical company focused on neurodevelopmental disorders.
The study evaluated the efficacy and safety of Neuropharm's new low-dose form of fluoxetine, known as NPL-2008, which is designed specifically for the treatment of Autistic Disorders in children and adolescents. NPL-2008 uses the Zydis® melt-in-the-mouth formula, intended to be an easier form of the medication for children. The study showed that repetitive behaviors were reduced when children were given either NPL-2008 or a placebo, but no differences between groups were found in terms of the level of reduction of repetitive behaviors. Thus, it was concluded that NPL-2008 was not more efficacious than the placebo. The new formulation was generally well tolerated by patients and no serious adverse events were reported.
Geraldine Dawson, Ph.D., Autism Speaks' Chief Science Officer remarked, “The development of effective drugs that are easily taken by individuals with autism is an important goal. While it is disappointing that NPL-2008 was not found to be effective for reducing repetitive behaviors, this study does highlight the importance of high quality, rigorously controlled clinical trials. Studies like this help build the evidence-base and offer parents guidance in making more informed decisions about their child's clinical care.”
The Autism Speaks ACTN was launched in 2005 to address the need for more thoroughly tested biological treatments for autism. The ACTN provides a platform to rigorously test the safety and efficacy of existing compounds, most of which do not have specific indications or specific dosing instructions for their use in autism. These treatments are prescribed by medical practitioners for “off-label” use.
Fluoxetine is one of the most widely prescribed central nervous system (CNS) drugs for disorders such as anxiety, depression, and obsessive-compulsive disorder. It has been prescribed “off-label” by physicians to alleviate repetitive behavioral symptoms in autism. Smaller studies in children and adults showed promising results, but there had not been a large scale study of this type to systematically confirm the efficacy of the drug for reducing these behaviors in autism. Currently, there is only one treatment with a specific FDA-approved indication for autism, but it does not target the core symptoms of autism.
The SOFIA study is the largest trial ever conducted in patients with Autistic Disorder. The study was carried out across 19 ACTN medical centers and clinical sites throughout the US. A total of 158 patients between the ages of 5 and 17 were enrolled into the SOFIA study in which patients received either NPL-2008 or placebo during a 14 week treatment period. The full analysis of the primary and secondary data from the study is ongoing.
Peter Bell, Executive Vice President for Programs and Services commented, “Finding safe and effective medications that treat the core symptoms of autism is one of the greatest challenges facing our community. One of our primary goals at Autism Speaks is to help identify effective treatments that lead to improved quality of life for all those affected by autism.”
Robert Mansfield, Neuropharm's CEO, stated “These initial results are both unexpected and disappointing, and we await the results of the full analysis which is currently ongoing. We would like to thank Autism Speaks' Autism Clinical Trials Network who facilitated the timely completion of this study and to thank the teams at the sites involved in SOFIA.”
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