Study of inclisiran for children with homozygous familial high cholesterol
Treatments studied
Part of Genetic & congenital, Heart & circulation, Hormones & metabolism clinical trials.
This trial tests a new medicine called inclisiran to lower very high cholesterol in children aged 2 to 12 who have a specific genetic condition (homozygous familial hypercholesterolemia, or HoFH). You may not need to change your current cholesterol treatments, and the study will check if inclisiran can help reduce your LDL cholesterol further.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are between 2 and 12 years old.
- You have a confirmed genetic diagnosis of homozygous familial hypercholesterolemia (HoFH), but you do NOT have the 'null' mutation in both LDLR genes.
- Your fasting LDL ('bad') cholesterol is above 130 mg/dL at screening.
- You are taking a stable dose of a statin (or you cannot take statins) with or without other cholesterol medicines, or you have been on regular LDL apheresis therapy for at least 3 months.
- You have not used certain other cholesterol drugs (like mipomersen, lomitapide, or PCSK9 antibody treatments) recently.
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
Similar studies
Other trials that look related to this one.
This trial tests a medicine called inclisiran to lower high cholesterol in children ages 6 to 12 with a genetic condition called heterozygous familial hypercholesterolemia (HeFH). It may help children who still have high cholesterol despite taking other treatments.
This study follows people with HeFH or HoFH (inherited high cholesterol) who previously took inclisiran in specific pediatric ORION studies. It mainly checks long-term safety and how well the treatment continues to be tolerated over time.
This trial tests a new version of inclisiran, a cholesterol-lowering medicine, to see if it is safe and works well. The study compares it to the current version. You may qualify if you have moderately high cholesterol but have never taken cholesterol medications.
This trial tests if a new medicine, lerodalcibep, can safely lower cholesterol in children ages 6 to 17 who have a genetic condition called heterozygous familial hypercholesterolemia (HeFH). It aims to help children whose cholesterol stays high despite taking standard cholesterol-lowering drugs.
This study tests two newer cholesterol-lowering medications (inclisiran and alirocumab) together in people who've had heart problems or strokes and cannot tolerate standard cholesterol drugs. The goal is to see if this combination safely lowers cholesterol further and reduces future cardiovascular events.
This study tests a new injectable medicine called inclisiran for people who are already on many medications and have high cholesterol. It is designed for those who cannot lower their cholesterol enough with statins, especially if they have diabetes or other heart risks.
Hear when a new Familial Hypercholesterolemia - Homozygous trial opens
We’ll email you when one opens — at most once a week, no account needed, unsubscribe anytime.