ORION 19- Inclisiran in children (2 to less than 12 years)
Research type
Research Study
Full title
Two part (double-blind inclisiran versus placebo [Year 1] followed by open-label inclisiran [Year 2]) randomized multicenter study to evaluate safety, tolerability, and efficacy of inclisiran in children (2 to less than 12 years) with homozygous familial hypercholesterolemia and elevated LDL-cholesterol (ORION-19).
IRAS ID
1011068
Contact name
Sarah Mofid
Contact email
Sponsor organisation
Novartis Pharma AG
Research summary
There are two main types of cholesterol in the blood: the HDL-cholesterol (also known as the “good” cholesterol) and the LDL-cholesterol (also known as the “bad” cholesterol). Homozygous familial hypercholesterolemia (HoFH) is a rare inherited disease that causes highly elevated levels of bad cholesterol to circulate in the blood since birth. When bad cholesterol is circulating in the blood at increased levels, it can accumulate in the walls of the blood vessels causing thick fatty deposits (called plaques) in the arteries leading to cardiovascular disease (CVD). CVD is a condition that involves blood vessels becoming blocked, which can then cause a heart attack, stroke, or reduced circulation of blood to other body parts. In HoFH this can occur when patients are young, for example in their twenties or thirties.
Inclisiran is a drug designed to lower LDL-cholesterol (“bad cholesterol”) and is already approved for use in adults with higher levels of bad cholesterol. It is also currently being studied in adolescents with HoFH. The purpose of this study is to evaluate if inclisiran can safely and
effectively lower the bad cholesterol in younger children with HoFH.REC name
East Midlands - Derby Research Ethics Committee
REC reference
25/EM/0004
Date of REC Opinion
13 Feb 2025
REC opinion
Further Information Favourable Opinion