Trial of RAD001 and Neurocognition in Tuberous Sclerosis Complex (TSC)
Status:
Completed
Trial end date:
2014-12-01
Target enrollment:
Participant gender:
Summary
Tuberous Sclerosis Complex (TSC) is a multi-system disease, usually presenting with seizures,
mental retardation and autism, and exhibiting a high variability in clinical findings both
among and within families. Investigators are doing research in order to identify possible
neurocognitive benefits from treatment with RAD001 or placebo for a six month period. There
may also be potential for improvements in seizure frequency, sleep and autistic behaviors. We
hope this trial will lead to a better understanding of TSC and to new forms of treatment, to
benefit children and adults with TSC in the future.
Individuals diagnosed with TSC will be asked to participate in this 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. Individuals must also be able to participate in neuropsychological
testing and meet certain medical criteria. They will need to sign an informed consent. If
enrolled in the study, 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, they will be asked to take either the study drug or a placebo (pill with
no medicine), which is determined by chance.
The study involves about 9 visits, 3 of which can be done locally, over a six month period,
as well as follow-up calls with our research nurse. Study visits will vary in length.
Screening, three month and six month visits may last up to 8 hours, while all other visits
will be less than 2 hours. The study visits include blood draws, laboratory tests and
neuropsychological assessments. There is no fee to participate in this study. The study drug
will be provided at no charge during the study.
After all study data has been analyzed, families will be informed of the overall results.
Treatment on this study may or may not improve a child's learning skills (neurocognition).
Future patients may benefit from what is learned.