Behavior and Learning Study for 6 to 21 Year Olds with Tuberous Sclerosis Complex (TSC)
What is the purpose of this study?
Cincinnati Children’s is conducting a research study, sometimes known as a clinical trial or clinical study, to determine if the study medication, RAD001 or Everolimus, can help improve how well people with TSC think, learn and behave as well as confirm the safety of this study medication.
Everolimus is approved by the Food and Drug Administration (FDA) for the treatment of individuals of all ages with subependymal giant cell astrocytoma’s (SEGA) and adults with acute myelogenous leukemia (AML).
Who can participate?
Children and adults diagnosed with TSC who:
- Are 6 to 21 years old
- Have an IQ of 60 or higher
- Have been on the same seizure medication(s), if applicable, for at least 6 months
What is involved?
You or your child will be in the research study for about 7 months and have 9 study visits and 3 phone visits. Participants will be randomly chosen to receive either the study medication (RAD001) or a placebo (a substance that looks like the study drug but contains no medication).
The following is a list of some of the procedures that will take place during the study:
- Review of medical history and current medications
- Physical exam and neurological exam
- Check of vital signs (blood pressure, heart rate, respiratory rate, body temperature)
- Neuropsychological evaluation (IQ and communication, socialization and other skills)
- Questionnaires on seizure severity and sleep
- Blood draws
- Urine sample
What are the benefits?
You or your child might not receive a direct medical benefit. However, the information gained from this study may benefit individuals with TSC in the future.
Will I get all the facts about the study?
You as a participant or as a parent interested in having your child participate will be given a consent form that thoroughly explains all of the details of the study. The form covers all of the procedures, the risks, the benefits, the pay, who to contact with questions or concerns and more. A member of the study staff will review the consent form with you and will be sure that all of your questions are answered. Study procedures will not begin until a parent or guardian has signed this form and, if of age, the child has given at minimum his/her verbal agreement.
What are the risks and discomforts of the study?
A detailed list of possible side effects will be provided to those participants, parents or guardians interested in knowing more about this study.
Do participants receive pay, compensation or reimbursement?
Participants do not receive any pay for their study involvement, however; the study drug is provided at no charge and there may be some travel reimbursement.
Who should I contact for more information?
Darcy Krueger, MD, PhD
Tuberous Sclerosis Clinic
Department of Neurology