Allison Cirino

Clinical trial seeks to change the course of hypertrophic cardiomyopathy

Allison L. Cirino, MS, CGC, Brigham and Women’s Hospital

February is American Heart Month. This month reminds us about the importance of a healthy lifestyle, regular medical care, and knowing your family health history. One important way to acknowledge American Heart Month is to consider participating in a clinical trial or, if you’re a clinician, referring your patient to one. Clinical trials can provide invaluable guidance for the treatment and prevention of many different forms of heart disease, including genetic heart diseases.

The increased availability of genetic testing has created a tremendous opportunity to identify individuals at risk for many genetic heart conditions well before the condition develops. That’s a crucial first step. But it’s not enough on its own. We also need preventive treatments to slow the condition’s progression or, better yet, stop it from ever developing. That’s where the research and medical community often falls short. Clinical trials seek to identify these much-needed treatments so that we can follow through to help individuals at risk for genetic heart conditions.

The VANISH trial is an NIH-sponsored, international, randomized placebo-controlled trial studying the use of the prescription medicine valsartan to halt or slow the progression of hypertrophic cardiomyopathy (HCM). HCM is a genetic heart condition that is characterized by thickening of the heart muscle wall. For many, HCM can be readily managed, but for some it can lead to heart failure or sudden death. A person may be eligible for the trial if he or she:

  • has a mutation in one of the genes known to cause HCM
  • is between the ages of 8 and 45
  • has been diagnosed with HCM or is at risk for developing HCM
  • has little to no symptoms caused by HCM

Participation lasts approximately two years and involves having genetic testing (if it hasn’t been done already) at no cost to the participant, taking the study medication every day, and making about 3 or 4 visits to the hospital. If there is no local participating site, travel assistance may be available. The VANISH team will work closely with the participant’s primary cardiologist to ensure the best possible experience for all involved.

The VANISH trial is a key first step to gaining the critical knowledge and experience needed to develop the first treatments that actually prevent HCM. Important trials like this involve collaboration on many levels—from the team of experts who design and conduct the trial, to the doctors who refer their patients, to, above all, the participants who generously donate their time and enthusiasm.

For more information about the trial, including a list of sites, please visit (trial NCT01912534) and contact Allison Cirino or Stephanie Harris.

We hope that you will join us in our effort!