HeFH Study

About this HeFH trial

Madrigal Pharmaceuticals is conducting a Phase 2 clinical research study designed to examine the effect of the investigational drug MGL-3196 in patients with HeFH who are receiving maximally tolerated statin therapy but require additional lowering of low density lipoprotein cholesterol (LDL-C).

Heterozygous familial hypercholesterolemia (HeFH) is a genetic disorder characterized by severe debilitating dyslipidemia and early onset CV disease. Individuals with HeFH typically have LDL-C levels approximately double that of unaffected siblings. HeFH is most commonly caused by mutations of the LDLR gene. If untreated, early onset coronary artery disease can develop in HeFH patients. The prevalence of HeFH is estimated to be 1 in 500 and may be as high as 1 in 200. Despite treatment with newer therapies (ie, proprotein convertase subtilisin/kexin type 9 [PCSK9] inhibitors) and standard care (which includes statins and ezetimibe), some HeFH patients are not achieving their LDL-C goal. For more information on HeFH, see the Familial Hypercholesterolemia Foundation website.

This Phase 2 study is to test the safety and effectiveness of the study drug called MGL-3196. MGL-3196 has previously been tested in humans and was well tolerated. MGL-3196 works by interacting with a form of thyroid hormone receptor in the liver to reduce elevated LDL cholesterol levels associated with HeFH. MGL-3196 is an investigational drug. This means that it is still being studied and that it has not been approved by the U.S. Food and Drug Administration (FDA) for the treatment of HeFH. The FDA only allows MGL-3196 to be used in research.

The purpose of this study is to determine:

  • How well MGL-3196 works for the treatment of HeFH compared to a placebo (a placebo looks like the study drug but does not contain any active ingredients).
  • The safety of the study drug in participants with HeFH.

About two thirds of patients in the study will be assigned to MGL-3196 and one third to placebo. Neither you nor your doctor will know the treatment assignment. The study lasts about 16 weeks.

You may be eligible to participate if you have been diagnosed with HeFH by genetic testing or have met the diagnostic criteria.