CLINICAL TRIAL STILL RECRUITING: Testing the Effects of RAD001 on Cognition and Autism in Children (Age 6 to 21) Where: Boston Childrens Hospital and Cincinnati Childrens Hospital Principal Investigators: Mustafa Sahin, MD, PhD (Boston) and Darcy Krueger, MD, PhD (Cincinnati) Drs. Mustafa Sahin and Darcy Krueger initiated an investigator-sponsored clinical trial to identify possible neurocognitive benefits from treatment with RAD001 or placebo for a 6 month period. There may also be potential for improvements in seizure frequency, sleep and autistic behaviors. The trial is sponsored by the TS Alliance, Autism Speaks and Novartis Pharmaceuticals. RAD001, the experimental drug proposed in this trial, works as an mTOR pathway inhibitor. Tuberous Sclerosis Complex is caused by defects in the TSC1 and TSC2 genes that negatively control mTOR, a protein that acts as a central regulator of tumor cell division, blood vessel growth, cell metabolism and neuronal migration/wiring. Several mouse models of TSC have been generated to date and all of these mouse models display neurological symptoms, many of which can be reversed by treatment with mTOR inhibitors. While mouse models obviously do not replicate all the symptoms of the human disease, they provide strong scientific rationale for the proposed trial. RAD001, marketed as Afinitor, was approved in November 2010 by the FDA for treatment of SEGAs that cannot be treated with surgery in patients with TSC. Individuals diagnosed with TSC will be asked to participate in the study if they are between the ages of 6 and 21 years of age and have an IQ of greater than or equal to 60. Both males and females will be asked to participate. Additionally, to be eligible for study participation, individuals must have been on the same seizure medication(s), if applicable, for at least 6 months prior to enrollment. Individuals must also be able to participate in neuropsychological testing and meet certain medical criteria. If enrolled, participants will have a number of screening tests to help determine if they are eligible for participation in the clinical trial. If eligible for the treatment phase of the trial, participants will be asked to take either the study drug or a placebo, which is determined by chance. The study involves about 9 visits to the study center, 3 of which can be done locally, over a six month period, as well as follow-up calls with our research nurse and study coordinator. There is no fee to participate in this study, and there will be no financial compensation for participation in this study. The study drug will be provided at no charge during the course of the study. After all data has been analyzed, families will be informed of the overall results. Enrollment for this clinical trial is currently ongoing at both Boston Childrens Hospital and Cincinnati Children’s Hospital Medical Center and will close in June 2014. Interested out-of-state individuals are welcome to participate and some limited compensation may be available for travel costs. If you are interested in participating in this study, please contact Rachel Friedman at (617) 919-3499 or [email protected]. To participate at Cincinnati Childrens Hospital, please contact Maxwell Mays at (513) 636-8016 or [email protected].
Posted on: Fri, 07 Mar 2014 12:41:37 +0000
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