Also called interventional studies, clinical trials test new treatments or new combinations of treatments. Clinical trials help researchers evaluate the safety and effectiveness of therapies to improve the quality of care for affected individuals. To locate a clinical trial near you, enter your information into the search criteria below.



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Below is a table with current pediatric and adult clinical trials for familial hypercholesterolemia and homozygous familial hypercholesterolemia.

Name Adult or Pediatric Population ClinicalTrials.gov  Identifier
CASCADE FH™ Registry Adults and Pediatric Heterozygous FH and Homozygous FH NCT01960244
Evolocumab (AMG 145) Pediatric Heterozygous FH and Homozygous FH NCT02392559
NCT02624869
Alirocumab Adults and Pediatric Heterozygous FH NCT02890992
NCT02957682
Bempedoic Acid (ETC-1002) Adults Heterozygous FH and Statin Intolerant NCT02991118
NCT02988115
MGL-3196 Adults Heterozygous FH NCT03038022
IONIS ANGPTL3-LRx Adults Heterozygous FH and Homozygous FH NCT02709850
ALN-PCSSC Adults Homozygous FH NCT02963311
Gemcabene Adults Homozygous FH NCT02722408
NCT02634151
Genetic AAV directed hLDLR Gene Therapy Adults Homozygous FH NCT03018678
NCT02651675
Evolocumab Pregnancy Exposure Registry Adults Hypercholesterolemia Pregnancy NCT02957604
NCT02906124
Last update: August 2017


Why participate in FH clinical research?


By participating in clinical research, you will play an active role in your own healthcare and contribute to the greater good. Because FH is a family disorder, clinical research offers hope and the opportunity to have a powerful impact on the lives of your family members and your future generations.

The FH Foundation seeks to (1) encourage the patient community to step forward and participate in FH research and (2) add to the current body of knowledge about FH.


Observational Studies


Observational studies are one type of clinical trial. There are no treatments or actions in an observational study. This type of study can help inform how to best diagnose, treat, and manage a disease or a condition.

The FH Foundation’s CASCADE FHTM Registry is an observational study aimed at helping FH individuals and researchers better understand the condition. For this study, patients provide data directly to the FH Foundation about the burden of disease, knowledge of FH, barriers to care, and experiences with current and new therapies.

Click to learn more about the CASCADE FH Registry or contact the FH Foundation at 626-583-4674 or visit /contact-us/.