Behavior and Learning Study for 6 to 21 Year Olds with Tuberous Sclerosis Complex (TSC)
What is the purpose of this study?
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.
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
an IQ of 60 or higher
been on the same seizure medication(s), if applicable, for at least 6 months
What is involved?
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
- Physical exam and neurological
- 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?
or your child might not receive a direct medical benefit. However, the information gained from this
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.
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?
Tuberous Sclerosis Clinic
Children’s Hospital Medical Center
Krueger, MD, PhD
Tuberous Sclerosis Clinic
Department of Neurology