Needed: 2 to 65 Year Olds with Tuberous Sclerosis Complex (TSC) and Uncontrolled Partial-Onset Epilepsy
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 evaluate the safety of the drug everolimus (Afinitor®) and find out if it has helpful effects in people who have tuberous sclerosis complex (TSC) and refractory, or uncontrolled, partial-onset seizures.
These are seizures that involve some type of body movement and are not adequately responding to treatment with epilepsy drugs.
Who can participate?
Children, teens and adults 2 to 65 years old who have TSC and:
- Have a diagnosis of refractory partial-onset epilepsy or seizures
- Are taking no more than 3 stable, anti-epileptic drugs
What is involved?
You or your child will have between 11 and 16 study visits for this research study. Participants will be randomly chosen to receive either the study medication (everolimus) 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)
- ECG (measures the electrical activity of the heart)
- Neuropsychological evaluation (IQ and communication, socialization and other skills)
- Questionnaires on seizure history and developmental milestones
- Completion of seizure diary
- Blood draws
- Urine sample
You or your child may also be asked to participate in a sub-study designed to evaluate how the everolimus treatment is working in TSC patients. This method uses diffusion tensor imaging (DTI) tractography (a kind of 3D MRI) accompanied by high frequency oscillations (HFO) EEG analysis. Participation is completely optional and will not affect your participation in the main study. The sub-study consists of two MRI sessions and two 1-hour EEG sessions.
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 and epilepsy 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?
Tuberous Sclerosis Clinic
Cincinnati Children’s Hospital Medical Center
3333 Burnet Ave.
Cincinnati, OH 45229-3039
David Franz, MD
Tuberous Sclerosis Clinic
Department of Neurology