Why are we doing this research?
Cincinnati Children’s is conducting a research study, sometimes known as a clinical trial or clinical study, to learn more about the safety, tolerability and effectiveness of an investigational drug for the treatment of Tourette syndrome (TS). We also want to see how the body absorbs, processes, distributes and gets rid of the drug.
An investigational drug is one that is not yet approved by the United States Food and Drug Administration (FDA)
Who can participate?
Children and teens, 6 to 17 years old, who have been diagnosed with TS, may be eligible for participation.
Your child CANNOT participate in the study if he or she has/is any of the following:
- Taking medication for TS or for other medical conditions and have not been using a stable dose for at least 30 days
- Pregnant or breastfeeding
- Of childbearing potential and unwilling to remain abstinent or use acceptable methods of contraception while in the research study
- Unable to produce negative drug and alcohol tests at Screening and Baseline Visits
- A diagnosed history of drug or alcohol dependence within 3 months of the Baseline Visit
- A hematologic malignancy or solid tumor diagnosed within 3 years of the Screening Visit, with the exception of localized skin cancer or carcinoma of the cervix
- A positive HIV, hepatitis B, and/or hepatitis C test result at the Screening Visit
- Have used any investigational drugs within 30 days before the Screening Visit
- Have donated blood or lost greater than 550 mL of blood within 30 days before the Baseline Visit
- Have ingested food containing poppy seeds 7 days before the Screening and Baseline Visits
- Have an allergy, hypersensitivity or intolerance to tetrabenazine
What will happen in the study?
If your child qualifies and you decide to participate, you will come to Cincinnati Children’s for 6 study visits over the next 11 weeks.
These are the tests and procedures that will happen during 1 or more of the study visits:
- Review of medical history –questions about general health, mental health and any tobacco and/or alcohol use
- Review of medication history –questions about any medications, vitamins and/or supplements taken in the past 30 days
- Physical exam
- Vital signs - blood pressure, heart rate, breathing rate, temperature, weight and height
- Electrocardiogram (ECG) – a test that measures the electrical activity of the heart
- Blood sample will be collected for lab tests (6 in total)
- Urine sample will be collected for lab tests
- Alcohol breath test
- Movements and tics evaluation –to determine the severity of tics (this will be video recorded and uploaded to a secure server for review)
- Questions about TS related symptoms - urge for tics, how your child is feeling, and if he or she has thoughts about self-harm
Before any study-related tests and procedures are done, you and your child will be asked to read and sign a consent form during a Screening Visit. During this visit, tests will be done and questions will be asked to determine if your child is able to participate in the study.
If your child passes the tests during the Screening Visit, you will also be asked to return to Cincinnati Children’s for another visit, known as a Baseline Visit.
At the Baseline Visit, your child will be randomly assigned by chance to 1 of 3 different dosing groups to receive either 40 mg of the study drug, 80 mg of the study drug, or placebo (an inactive substance, like a sugar pill). Your child will be provided with a 2-week supply of his or her assigned study drug before you go home.
There is a 33% chance your child will receive placebo instead of the study drug. Neither you nor your study doctor will know which group you or your child have been assigned to. However, in case of emergency, the study doctor can obtain this information.
The morning after the Baseline visit, your child will take 2 capsules between 7:00 am and 10:00 am. Your child will continue to take 2 capsules every morning at approximately that same time for 6 weeks. The date and time of dosing will need to be recorded on the study drug packaging every morning.
During the 6-week dosing period, your child will also have 3 study visits (at the end of Weeks 2, 4 and 6). Your child will need to bring the study drug packaging to each of these visits.
The final study visit will happen 2 weeks after your child take the final dose of the study drug. At the end of this visit, participation in this study will be over.
Participants, parents or guardians will be given a consent form that thoroughly explains all of the details of the study. A member of the study staff will review the consent form with you and your child and will be sure that all of your questions are answered.