Needed: 8 to 45 Year Olds with Hypertrophic Cardiomyopathy

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 if an investigational drug can safely be used in people who have Hypertrophic Cardiomyopathy (HCM), or are at risk for developing HCM. We also want to see if the investigational drug slows the harmful changes in the heart due to HCM.

“Investigational” means that the United States Food and Drug Administration (FDA) has not approved the drug being tested for the treatment of HCM.

Who can participate?

Children, teens and adults, 8 to 45 years old, who have HCM, or are at risk for developing HCM, may be eligible for participation.


  • Cardiology - Heart

What will happen in the study?

If you or your child qualifies, and you decide you want to participate in the study, you will come to Cincinnati Children’s for 7 study visits over a period of 2 years. A few people may need extra visits when starting the investigational study drug.

You or your child will also have phone calls, each lasting about 30 minutes, with the study coordinator every 3 months while in the study.

The following is a list of some of the tests and procedures that will happen during the study:


  • Questions about medical history and development
  • Questions about family history
  • Questions about quality of life and daily activities
  • Physical exam – including height, weight and vital signs (blood pressure, heart rate, breathing rate and temperature)
  • Blood samples - for laboratory tests 
  • Urine samples - for pregnancy testing (if you are female and able to become pregnant)
  • Electrocardiogram (EKG or ECG) – a test that measures the electrical activity of the heart
  • Echocardiogram (echo) – a test using sound waves that takes a picture of the heart and blood vessels
  • Cardiac MRI – a test using magnets that takes detailed pictures of the heart 
  • Exercise test – a test requiring you or your child to run on a treadmill or ride a bicycle while your blood pressure, heart rate and EKG are recorded
  • Provided a FitBit - very small activity monitor that clips on to clothes and tracks the amount of steps taken/or stairs climbed 
The first visit, called the screening or baseline visit, will take about 5 hours to complete. Tests and procedures completed during the screening/baseline visit will confirm if you or your child can participate in this study.

If you or your child is eligible to participate, you will be randomly assigned (by chance) to receive either the study drug or placebo. Placebo looks just like the study drug but does not contain any medication. You or your child will need to take your assigned drug 2 times a day, by mouth, for 2 years.

You, as a participant or as a parent/guardian, will be given a consent form that thoroughly explains all of the details of the study and information about the study drug. A member of the study staff will review the consent form with you and will be sure that all of your questions are answered.


What are the good things that can happen from this research?

You or your child may not receive any direct benefit from participating in this study. You may gain a better understanding of Hypertrophic Cardiomyopathy and how it may affect you and/or your children.

What are the bad things that can happen from this research?

A detailed list of possible side effects will be provided to those participants, parents or guardians interested in knowing more about this study.

Will you/your child be paid to be in this research study?

Participants may be paid up to $350.00 for their time and travel.


Contact Us.Paula Casson
Cincinnati Children’s Hospital Medical Center
Heart Institute
3333 Burnet Avenue
Cincinnati, OH 45229-3039

Study Doctor

Contact Us.John L. Jefferies, MD, MPH, FAAP, FACC
Cincinnati Children’s Hospital Medical Center
Heart Institute