Heterozygous Familial Hypercholesterolemia Trial Shows Positive Results of Therapy in Teenagers

Researchers in Canada have just released positive results from their heterozygous familial hypercholesterolemia (HeFH) clinical trial. This investigation was examining the effect of Repatha on teenagers with the condition. The trial was called HAUSER-RCT.

The results from the study were published in the New England Journal of Medicine.


HAUSER-RCT was a placebo-controlled, randomized investigation of 157 HeFH patients. The average patient age in this trial was 13.7. The participants were 85% white and 56% female. Previously, a study found this therapy to be well tolerated for patients with homozygous FH or severe HeFH.

The HAUSER-RCT study examined whether adding an injection (420 mg) of Repatha each month in addition to the patients typical lipid-lowering therapies, would help in lowering LDL cholesterol. Typical therapy for HeFH uses ezetimibe and statins, however their effectiveness is not universal across patients. Some patients experience a limited response to the drugs or negative side effects. Additionally, adherence is an issue.

Thankfully, a new option may be efficacious. The results from this trial were incredible.

The average LDL levels decreased 44.5% (77.5 mg/dL) compared to 6.2% (9.0 mg/dL) in the placebo group. This was achieved in just 24 weeks of treatment.  Additionally, non-HDL cholesterol was also improved as was apolipoproteien B.

There were no concerns with safety in this trial.

Looking Forward

This therapy was approved back in 2015 by the FDA to reduce LDL cholesterol for those who don’t respond to standard therapies. It’s indication was expanded in 2017 to serve as an aid to reduce cardiovascular risk.

There is currently an open-label extension study taking place for this therapy. Researchers are still working to examine the longterm effects of the treatment.

You can read more about this HeFH trial here.

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