CKJX839C12303: A phase III study with inclisiran in children with HeFH (Orion20)
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 (6 to less than 12 years) with heterozygous familial hypercholesterolemia and elevated LDL-cholesterol (ORION-20)
IRAS ID
1008920
Contact name
Sarah Mofid
Contact email
Sponsor organisation
Novartis Pharma AG
Clinicaltrials.gov Identifier
-
Research summary
Heterozygous familial hypercholesterolemia (HeFH) is an inherited disease that causes elevated levels of “bad” cholesterol (LDL cholesterol) to circulate in the blood since birth which can cause thick fatty deposits (called plaques) in the arteries leading to so-called atherosclerotic cardiovascular disease (CVD). Inclisiran is a drug designed to lower bad cholesterol. The purpose of this study is to evaluate if inclisiran can safely and effectively lower the bad cholesterol in younger children with HeFH.
The study will be conducted in multiple centres across the world, and approximately 51 participants from the age of 6 to younger than 12 will be included.
During the first year, participants will receive an injection of inclisiran or placebo (placebo looks like the trial drug but has no drug in it) by chance (which is called randomisation).
A computer program will randomly assign each participant to one of the treatments: with a “2 in 3 chance” of receiving inclisiran or a “1 in 3 chance” of receiving placebo during the first year. Neither the doctor nor the participant will know which drug the participant will be assigned.
The study treatments will be given as an injection under the skin on day 1, 90, and then again on day 270. At the completion of year one, on day 360, a ‘switch day’ will take place and on that day participants who were receiving placebo will receive inclisiran and participants who were receiving inclisiran will receive placebo (placebo is only given on day 360 so that it is not known whether the participant received inclisiran or placebo in year one).
From then on, all participants will receive inclisiran on day 450 and 630. At the end of year two, on day 720, the study will be completed.REC name
East Midlands - Derby Research Ethics Committee
REC reference
25/EM/0005
Date of REC Opinion
13 Feb 2025
REC opinion
Further Information Favourable Opinion