Familial Hypercholesterolemia (FH)
Clinical Trials Update
Did you know that studying individuals with familial hypercholesterolemia led to understanding the role of LDL cholesterol in the development of heart disease, the discovery of PCSK9 gene mutations, and the development of breakthrough treatments such as statins and PCSK9 inhibitors?

Not to mention that data from the FH Foundation’s CASCADE FH ® Registry has helped us create stronger medical guidelines, as well as improve the identification and care of FH with health systems and insurance companies.

Research is an important backbone to improving the care for individuals with FH and a key pillar of our work at the FH Foundation. There are multiple ways to contribute to research as you will see from many of the exciting opportunities below.
 
The CHEERS research study is being conducted by Dr. Heather Zierhut and Dr. Julia Steinberger at the University of Minnesota.

You are eligible to participate in this study if:
  1. You are the ​biological parent of a child with FH
  2. You have NOT had genetic counseling about high cholesterol that runs in the family

If you are interested in taking part, we would ask that you fill out a survey and participate in an educational interview that happens once over the telephone and lasts for about 45 minutes. You will be compensated with a $50 Visa gift card as a thank you for your time. Thereafter, we will ask you to fill out surveys at one month, six months, and one year after the interview. For each completed survey, you will be compensated $25 (study total = $150). If you decide to participate, please contact Drs. Zierhut and Steinberger at 612-626-6743 or cheers@umn.edu . to receive your unique access code so that you can take the initial survey and schedule your interview on their website.  

To complete the initial survey and schedule your appointment or to find more information, you can visit the Cheers study website or email cheers@umn.edu .
 
What We Are Learning from the CASCADE FH Registry

We recently hosted a webinar on the impact that participating in a patient registry can have on improving the scientific understanding and care for familial hypercholesterolemia. We shared what the FH Foundation is learning from the national CASCADE FH Registry and were joined by the Cystic Fibrosis (CF) Foundation to share their experience with their registry and how it has changed the landscape for patients affected by CF. You can watch a recording of the webinar here .
 
Interested in joining the CASCADE FH Registry Patient Portal? It is easy and will only take 10-15 minutes of your time. In the patient portal you can:
  • Track heart health, medications and more
  • Build a family tree
  • Link up with the FH Community
  • Participate in clinical and genetic research
 
Your data is anonymous. Combined together with data from the over 1,000 other individuals with FH in the Patient Portal, you can help tell the story of FH.

It is important to note that you can be registered in both the clinical registry at a CASCADE FH site and the Patient Portal. In fact, they both provide scientists with different but equally important information about the care and treatment of FH. 
For more information or to get help enrolling, please contact FHregistry@theFHfoundation.org .
FH & Pregnancy: Help Other Moms and Babies Through Research
MotherToBaby  is currently enrolling women in their observational  High Cholesterol and Pregnancy study . You may qualify for the study if you have FH, are pregnant, and currently reside in the U.S. or Canada. The study is observational; you will not be asked to travel or make any changes to your healthcare routine,  including your medications .

Highlight of Clinical Trials for FH

Clinical trials are looking for individuals with FH and other forms of hypercholesterolemia to join. This is how new treatments become available – with the generous participation of clinical trial participants. But we know that often doctors don’t ask individuals with FH if they are interested in joining a trial – it is up to you to seek out these opportunities.

If you are interested in joining a clinical trial, click on the link below to see trials that are actively enrolling. Contact us if you need help connecting with a clinical trial.

Pediatric Trials
Clinical trials are ongoing for PCSK9 inhibitor treatment of children with FH age 8 or 10 to 17 years old. There are trial sites across the US and around the world, from Florida to California, from Australia to the UK, Canada to Brazil, and many places in between.

Homozygous FH and Severe FH
This is an exciting and hopeful time for HoFH treatment. There are several trials ongoing or soon to begin for individuals with HoFH and severe FH, including trials for two different approaches to PCSK9 inhibition, gene therapy, a trial for a new drug target known as ANGPTL3, and another approach known as HDL Acute Lipid Optimization.

If you have questions, or need help connecting with a clinical trial, please contact us at info@theFHfoundation.org .