Why are we doing this research?
Cincinnati Children’s is conducting a research study, sometimes known as a clinical trial or clinical study, to find out if a drug, called pitavastatin, used to lower cholesterol can improve artery health in children and teens who are overweight with high blood lipids (cholesterol and fats). These children and teens may have a condition known as combined dyslipidemia of obesity (CDO).
CDO is abnormal levels of blood lipids that may occur in people who are overweight. When people have CDO for many years, the lipids may build-up in the arteries, causing them to thicken and narrow (called atherosclerosis). This can then lead to serious health problems later in life, such as heart attack or stroke.
What is the Pediatric Heart Network (PHN)?
This is a Pediatric Heart Network (PHN) research study. PHN is a group of hospitals in the US and Canada that conducts research studies in children with heart disease and is supported by the National Heart, Lung, and Blood Institute of the National Institutes of Health.
Who can participate?
Children and teens, 10 to 17 years old, who are overweight and have high cholesterol may be eligible for participation.
What will happen in the study?
If your child qualifies for this study and you decide you want him/her to participate, your child will come to Cincinnati Children’s 9 times over the next 2 years. Study visits will last between 1 and 4 hours.
The following will happen during one or more study visits:
- Review of medical and family history: a review of your child’s medical records, medicines, supplements, medical history and family history
- Physical exam: your child’s height, weight, waist and blood pressure will be measured
- Questionnaires/assessments: you and/or your child will answer questions about your child’s body development and lifestyle behaviors (such as eating, screen time, and physical activity), household income, your education, and whether or not you work outside of the home
- Vascular measures: the thickness and stiffness of your child’s blood vessels will be measured
- Blood draws: samples of your child’s blood will be taken for lab and safety tests (your child will not be able to eat or drink, other than water, for 12 hours before each blood draw)
- Urine collection: samples of your child’s urine will be collected for a urinalysis and pregnancy (if your child is female) testing
- Lifestyle (diet and exercise) counseling: your child’s regular doctors and/or the research team will talk to you and/or your child about a healthy lifestyle as a part of his/her regular care (your child will be provided with a physical activity wrist monitor to wear)
You and your child will be given a parental permission form/child assent form, which explain the details of the study, to review and sign. A member of the research team will review the forms with you and your child and will be sure all your questions are answered.
Your child will first come to Cincinnati Children’s for a screening visit, which will determine whether or not he/she qualifies for this study.
If your child qualifies, he/she will be scheduled for a baseline visit (within 6 weeks of the screening visit). During this visit, your child will be "randomized" (by chance, like flipping a coin) into one of 2 study drug groups to receive either:
- Pitavastatin: a type of cholesterol-lowering medicine, called a statin, which lowers “bad” cholesterol levels (LDL) that increase the risk of heart disease and heart attacks, and increases “good” cholesterol levels (HDL) slightly, which may help to lessen build-ups in arteries
- Placebo: looks just like the study drug but contains no active ingredient
Your child will have an equal chance of receiving either pitavastatin or placebo. Neither you, your child, nor the researchers conducting this study will know what group your child is in.
You and your child will be taught how to take his/her assigned study drug, which will be in the form of a pill. Your child will take the study drug once a day by mouth for 2 years. It is important that your child takes the study drug as instructed.
At the end of the visit, you will be given enough of your child’s study drug to last until the next study visit (the study drug may also be mailed to you).
There will be 7 more study visits after the baseline visit, typically 3 to 6 months apart. At each of these visits, your child will complete some tests and procedures and the research team will ask about side effects and/or health problems. You will also need to return all unused or leftover study drug to the research team to be counted at each of the visits.
When all participants in the study have finished their 2 years of study drug, the research team will find out what study group your child was in and will tell you and your child’s doctor.
What are the good things that can happen from this research?
Being in this study may or may not help your child right now. If your child is randomized to receive pitavastatin, it is possible that his/her lipid levels could improve. However, we cannot guarantee or promise this will happen.
When we finish the study, we hope to know more about how to treat children with CDO. This may help other children with CDO later on.
What are the bad things that can happen from this research?
There are some possible risks and side effects associated with this study and study drug. All possible risks will be discussed with you if you are interested in learning more about the study.
Will you/your child be paid to be in this research study?
Participants will receive up to $500 and parents/guardians will receive up to $325 for their time and travel. Participants will also receive a welcome bag containing items such as a water bottle, cookbook and fitness tracker which will be theirs to keep at the end of the study.