Testing a New Cholesterol Injection for Children With Familial Hypercholesterolemia

Plain-English translation of NCT06597019 on ClinicalTrials.gov ↗ · Source last updated May 2026 · Translation generated July 2026 · How we translate trials

Phase 3 — Testing in thousands of people, comparing the treatment against what doctors currently use. This is the last big step before approval.

What is this trial?

This trial is testing a new medication called to see if it can help lower cholesterol in children with familial hypercholesterolemia — a genetic condition where the body has trouble controlling cholesterol levels. The study will compare this medication to a placebo (a dummy injection with no medicine) during the first year, then everyone will receive the active treatment during the second year.

Many children with familial hypercholesterolemia have dangerously high cholesterol even when taking standard cholesterol medicines. Researchers want to know if this medication can safely and effectively lower cholesterol further in this young group.

Do you qualify?

You likely qualify if…

you are between 6 and 12 years old at the time you join
you have been diagnosed with heterozygous familial hypercholesterolemia (either through genetic testing or clinical signs)
your fasting LDL cholesterol is above 130 mg/dL at screening
you weigh at least 16 kilograms (about 35 pounds)
if you are 8 years or older, you are already taking a statin or other cholesterol-lowering medicine at a stable dose

You likely don't qualify if…

you have received a PCSK9 inhibitor (a different type of cholesterol injection) within the past 3 months
you have homozygous familial hypercholesterolemia (the more severe genetic form)
you have active liver disease or severely abnormal liver function tests
you are pregnant or nursing
you have received or plan to receive another experimental medication or device during the study

What participation looks like

If you join, you'll come to research visits over 2 years. In the first year, you'll receive injections at specific timepoints — some children will get the new medication and others will get a placebo. In the second year, everyone receives the active treatment. The injections are given under the skin, and researchers will regularly check your cholesterol levels and overall health to make sure the treatment is safe and working.

AI-generated summary from trial data · Jul 7, 2026 · Not medical advice

Trial locations(66 sites)

United States

UC San Francisco Medical Center, San Francisco, California

Children's National Hospital, Washington D.C., District of Columbia

Childrens National Hospital, Washington D.C., District of Columbia

Excel Medical Clinical Trials LLC, Boca Raton, Florida