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siRNA
Inclisiran for High Cholesterol (ORION-13 Trial)
Phase 3
Waitlist Available
Research Sponsored by Novartis Pharmaceuticals
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Homozygous Familial Hypercholesterolemia (HoFH) diagnosed by genetic confirmation
Estimated glomerular filtration rate (eGFR) >30 mL/min/1.73 m2 at screening
Must not have
Documented evidence of a null (negative) mutation in both LDLR alleles
Active liver disease
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline, after day 90 up to day 330
Awards & highlights
Pivotal Trial
Summary
This trial tests inclisiran, a medication that lowers bad cholesterol, in adolescents with a genetic condition causing very high cholesterol. Inclisiran works by blocking a protein that increases cholesterol levels, leading to significant reductions in bad cholesterol.
Who is the study for?
This trial is for adolescents with a genetic condition called homozygous familial hypercholesterolemia (HoFH) and high LDL cholesterol levels, despite being on the highest dose of statins they can tolerate. They should not have used certain other cholesterol drugs recently and must have decent kidney function.
What is being tested?
The study tests Inclisiran, a new medication aimed at lowering LDL cholesterol, against a placebo in young people with severe inherited high cholesterol. It's designed to see if it's safe and effective in reducing their cholesterol levels.
What are the potential side effects?
While specific side effects for Inclisiran are not listed here, common ones may include injection site reactions, allergic responses, liver enzyme changes, muscle pain or weakness, and potential interactions with other lipid-lowering therapies.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I have been genetically diagnosed with Homozygous Familial Hypercholesterolemia.
Select...
My kidney function is above the minimum level required.
Exclusion Criteria
You may be eligible for the trial if you check “No” for criteria below:Select...
My tests show a negative mutation in both LDLR genes.
Select...
I have an ongoing liver condition.
Select...
I have not had a major heart problem in the last month.
Select...
I have a genetic condition that causes high cholesterol.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ baseline, after day 90 up to day 330
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline, after day 90 up to day 330
Treatment Details
Study Objectives
Study objectives can provide a clearer picture of what you can expect from a treatment.Primary study objectives
Low-Density Lipoproteins
Secondary study objectives
% change and absolute change in LDL-C from baseline up to Day 720
% change and absolute change in other lipoprotein and lipid parameters from baseline up to Day 720
% change and absolute change in proprotein convertase subtilisin/kexin type 9 (PCSK9) from baseline up to Day 720
+1 moreSide effects data
From 2019 Phase 3 trial • 482 Patients • NCT0339712112%
Nasopharyngitis
9%
Injection site reaction
7%
Upper respiratory tract infection
7%
Back pain
5%
Influenza
1%
Acute myocardial infarction
1%
Aortic valve stenosis
100%
80%
60%
40%
20%
0%
Study treatment Arm
Placebo
Inclisiran
Awards & Highlights
Pivotal Trial
The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
Trial Design
2Treatment groups
Experimental Treatment
Placebo Group
Group I: InclisiranExperimental Treatment1 Intervention
Year 1 - inclisiran sodium 300 mg subcutaneous injection (given at Days 1, 90, and 270) Day 360 only - placebo subcutaneous injection Year 2 - inclisiran sodium 300 mg subcutaneous injection (given at Days 450 and 630)
Group II: PlaceboPlacebo Group1 Intervention
Year 1 - placebo subcutaneous injection (given at Days 1, 90 and 270) Year 2 - inclisiran sodium 300 mg subcutaneous injection (given at Days 360, 450, and 630)
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Inclisiran
2021
Completed Phase 3
~2820
Research Highlights
Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.Mechanism Of Action
Side Effect Profile
Prior Approvals
Other Research
The most common treatments for hypercholesterolemia include statins, ezetimibe, and PCSK9 inhibitors. Statins work by inhibiting HMG-CoA reductase, an enzyme involved in cholesterol synthesis in the liver, leading to reduced LDL-C levels.
Ezetimibe reduces cholesterol absorption in the intestines, further lowering LDL-C. PCSK9 inhibitors, such as Inclisiran, work by inhibiting the PCSK9 protein, which increases the number of LDL receptors on liver cells, enhancing the clearance of LDL-C from the bloodstream.
These treatments are crucial for hypercholesterolemia patients as they significantly lower LDL-C levels, reducing the risk of cardiovascular events and improving overall heart health.
PCSK9 Inhibitors in Secondary Prevention-An Opportunity for Personalized Therapy.PCSK9 inhibition in LDL cholesterol reduction: genetics and therapeutic implications of very low plasma lipoprotein levels.
PCSK9 Inhibitors in Secondary Prevention-An Opportunity for Personalized Therapy.PCSK9 inhibition in LDL cholesterol reduction: genetics and therapeutic implications of very low plasma lipoprotein levels.
Find a Location
Who is running the clinical trial?
Novartis PharmaceuticalsLead Sponsor
2,913 Previous Clinical Trials
4,253,150 Total Patients Enrolled
24 Trials studying Hypercholesterolemia
29,452 Patients Enrolled for Hypercholesterolemia
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- My tests show a negative mutation in both LDLR genes.I have not taken mipomersen or lomitapide in the last 5 months.I have been genetically diagnosed with Homozygous Familial Hypercholesterolemia.I have an ongoing liver condition.I have not taken PCSK9 inhibitors in the last 90 days.I have high cholesterol due to another health condition like thyroid issues or kidney problems.Your cholesterol level is too high when you haven't eaten for a while.I have not had a major heart problem in the last month.I have been on the highest dose of statin I can tolerate for at least 30 days.My kidney function is above the minimum level required.I have a genetic condition that causes high cholesterol.
Research Study Groups:
This trial has the following groups:- Group 1: Inclisiran
- Group 2: Placebo
Awards:
This trial has 1 awards, including:- Pivotal Trial - The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
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