Clinical Trials

For more information about our upcoming Phase 2 clinical trial, please the FAQ section below.

Perhexiline Phase 2 Trial FAQs

We know that drug trials can be confusing. Here is some basic information about our Phase 2 study to get you started.

What is the trial studying?

Our trial is studying whether the drug perhexiline can improve symptoms in patients with Hypertrophic Cardiomyopathy (HCM).  Currently, there are no medications known to improve symptoms in this disease.

What is perhexiline?

Perhexiline is a drug that is being developed to treat symptoms in patients with HCM.  It is taken orally once daily.  It has been used to treat other heart conditions in Australia and New Zealand for more than 30 years, but it has never been available in the United States.

Does everyone in the trial take perhexiline?

Yes. Everyone who enters this clinical trial will receive perhexiline as there are no patients who will receive a placebo.

Who can be in the trial? (What are the criteria for trial participation?)

You may qualify for this research study if you:

  • are above the age of 18
  • are diagnosed with HCM
  • have symptoms which limit your ability to exercise

What will happen in the trial?

The study will involve taking one or more pills (perhexiline) once a day for four months.  During this time, the dosage will be changed by your physician.  There are approximately seven visits over a six month period, and you will be periodically asked to perform cardiopulmonary exercise (CPEX) testing.

What tests are part of the study?

Cardiopulmonary exercise test (CPEX): uses a treadmill or bicycle to assess heart function while exercising.  You will be required to wear a breathing mask during the test in order to measure the amount of oxygen consumed during exercise.

Electrocardiogram (ECG): painless recording of the electrical activity of your heart

Blood draws: about 9 tablespoons total during the study, and never more than 5 tablespoons in one visit

What are the risks of my participation in the trial?

Perhexiline may cause liver or nerve problems if it accumulates in the bloodstream.  This can be prevented if genetic testing reveals that you are susceptible, and you would not be allowed to participate in the trial.  If allowed to enroll, these problems will also be prevented by periodically measuring your blood levels of perhexiline and modifying your dose appropriately.

Perhexiline may cause changes in the electrocardiogram (ECG).  With some drugs, similar changes have been associated with an increase in the risk of suffering a fatal heart abnormal heart rhythm. These irregularities, however, have not been associated with the use of perhexiline.  As an additional precaution you will be monitored during the study for changes in your ECG.  If they occur, your dose may be reduced, or your participation in the study may be stopped.

Are there any costs for participating in the trial?

No, there are no costs to you for participating in the trial.

What if my doctor is not at a participating site?

You can be in the trial and continue to see your current doctors. You do not need to be a patient at the study center to enroll in the trial there. The trial doctors will work closely with your doctors throughout the study to coordinate your care.

I want to be in the trial, what should I do?

Additional information will be available soon at To be notified, please register your email address at the top of this page.

I want to be in the trial but cannot afford to travel to a study center. What should I do?

There is limited financial assistance available. More information will become available soon.