Serving the HCM Spectrum Disorder Community Since 1996

VANISH - A drug trial for gene positive or have HCM

A clinical trial is currently underway to investigate whether a drug called valsartan can delay the onset of Hypertrophic Cardiomyopathy (HCM) or slow the progression of HCM. Studies in animals with HCM have suggested that medications like valsartan may be able to decrease changes to the heart. With this study, we would like to see if valsartan can be safely used in people with HCM or people at risk for developing HCM .We also want to see if valsartan improves how the heart is working. Here are some facts about this very important clinical trial.

Who is in charge of the clinical trial?

This trial is funded by the National Heart lung and Blood Institute (NHLBI) of the National Institutes of Health (NIH). The trial is led by investigators with expertise in HCM and clinical trials.


Where is the study taking place?

Study visits will take place at centers located across the Unites States and Canada, including:


Stanford University, Palo Alto, CA


University of Colorado Denver, Denver, CO


University of Chicago, Chicago, IL


Johns Hopkins University, Baltimore, MD


Brigham and Women’s Hospital, Boston, MA

Boston Children’s Hospital, Boston, MA

Massachusetts General Hospital, Boston, MA


University of Michigan, Ann Arbor, MI


Washington University School of Medicine, St. Louis, MO


Cleveland Clinic Foundation, Cleveland OH

Cincinnati Children’s Hospital Medical Center, Cincinnati, OH


Hospital of the University of Pennsylvania, Philadelphia, PA

Children’s Hospital of Philadelphia, Philadelphia, PA


Vanderbilt University, Nashville, TN


Toronto Hospital for Sick Children, Toronto, ON

University of Toronto/Toronto General Hospital, Toronto, ON



The following sites are also contributing to the trial, but will not be conducting study visits:

Yale University, New Haven, CT

Columbia University Medical Center, New York, NY

More centers may be joining the VANISH trial.

What is the trial studying?

The trial is studying whether a drug, valsartan, can delay the onset of Hypertrophic Cardiomyopathy (HCM) or slow the progression of HCM. Currently there are no medications that can delay or prevent HCM.

What is valsartan?

Valsartan is an FDA approved drug which is safe for treating high blood pressure in adults and children at the doses that will be used in this study.  Most people do not have side effects from taking this medication for their high blood pressure. Valsartan has not been studied or approved to treat HCM.

Does everyone in the trial take valsartan?

No. You have a 50% chance of being placed in the valsartan group and 50% chance of being placed in the placebo (sugar pill) group. Neither you nor your doctor will know which group you are in.

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

You may qualify for this research study if you:

  • are between the ages of 8-45 years
  • have a mutation in one of the genes that is known to cause HCM
  • are diagnosed with HCM or are at risk for developing HCM
  • have little to no symptoms caused by HCM

If you have not had genetic testing, it can be provided free of charge by the study.


What will happen in the trial?

The study will involve taking a pill (valsartan or inactive sugar pill) twice a day for approximately two years.  There are up to seven visits over the two year period but only 3 to 4 of the visits need to be done at the study center. Some of the visits can be replaced with phone calls or visits to a local doctor.


What tests are part of the study?

Cardiac MRI: takes detailed picture of the heart using large magnets within a large tube

Echocardiogram (echo): ultrasound of the heart

Exercise test: uses a treadmill or bicycle to assess heart function while exercising

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


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?

Contact the study coordinator at the study center nearest you. The study coordinator can provide you with more details about the trial, answer your questions and help decide if you are eligible for the trial.


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. You can contact the study coordinator to discuss your eligibility for the trial and how to apply for financial help with study-related travel expenses.

To find out more about the clinical trial and to find a study center:

For more information please contact Stephanie Harris of Brigham and Women’s Hospital at 617.525.7168 or sharris19@partners.org.

For additional information on this trial view the following HCMA Webinar with Dr. Caroline Ho