2 to 65 Year Olds With Tuberous Sclerosis Complex (TSC) and Facial Angiofibromas Needed for Research Study
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 appearance of facial angiofibromas, or elevated skin lesions, is reduced when a cream containing the investigational medication Rapamycin is rubbed onto them.
The study will also make sure Rapamycin is safe to use in people diagnosed with Tuberous Sclerosis Complex (TSC).
Who can participate?
Children, teens and adults 2 to 65 years old who have visible angiofibromas and are diagnosed with TSC may be eligible to participate.
Participants must not be on any Mammalian Target of Rapamycin (mTOR) inhibitors prior to being in this study. Examples of mTOR inhibitors are rapamycin, afinitor, sirolimus, everolimus and rapamune.
What is involved?
You or your child will be in the research study for about 7 months and have 7 study visits.
You or your child will be randomly selected to join 1 of 3 different study groups. One group will receive a low dose of the study drug, another group will receive a higher dose of the study drug and the third group will receive a cream that contains no medication. Neither you nor the study doctor will know what group you or your child will be in.
The following is a list of some of the procedures that will take place during the study:
- Medical and medication history
- Physical exam
- Photographs of the angiofibromas
- Quality of Life Questionnaire
- Blood draw
What are the benefits?
You or your child might not receive a direct medical benefit. However, the information gained from this study may help other people with TSC in the future.
Will I get all the facts about the study?
As someone interested in participating or as a parent interested in having your child participate, you 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.
Do participants receive pay, compensation or reimbursement?
Participants will not be compensated but will receive the study medication at no cost.
Who should I contact for more information?
Darcy Krueger, MD, PhD
Tuberous Sclerosis Clinic
Department of Neurology
Cincinnati Children’s Hospital Medical Center
3333 Burnet Ave.
Cincinnati, OH 45229-3039