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siRNA

Inclisiran for High Cholesterol (ORION-16 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

Heterozygous Familial Hypercholesterolemia (HeFH) diagnosed either by genetic testing or on phenotypic criteria

Fasting LDL-C >130 mg/dL (3.4 mmol/L) at screening

Must not have

Homozygous familial hypercholesterolemia (HoFH)

Active liver disease

Timeline

Screening 3 days

Treatment Varies

Follow Up 3 days

Awards & highlights

Pivotal Trial

Summary

This trial will study the effects of a new drug, inclisiran, on adolescents with a genetic condition that causes high cholesterol.

Who is the study for?

This trial is for adolescents with a condition called heterozygous familial hypercholesterolemia (HeFH) and high LDL cholesterol levels. They should be on the highest dose of statins they can handle, possibly with other cholesterol drugs, for at least 30 days before joining. Those with homozygous familial hypercholesterolemia, liver disease, secondary causes of high cholesterol like hypothyroidism or nephrotic syndrome, recent heart events, or previous PCSK9 inhibitor treatments cannot join.

What is being tested?

The study tests Inclisiran's ability to lower bad cholesterol in teens with HeFH compared to a placebo. It's a phase III trial which means it’s checking if Inclisiran is safe and works well after earlier studies showed promise. Participants will randomly receive either Inclisiran or a dummy drug without knowing which one.

What are the potential side effects?

Inclisiran could cause side effects such as injection site reactions (like pain or swelling), flu-like symptoms, muscle pain, headache and possible allergic reactions. The exact side effects in adolescents are being studied so there might be more that aren't known yet.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have been diagnosed with HeFH through genetic testing or based on symptoms.

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My fasting LDL cholesterol is above 130 mg/dL.

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My kidney function, measured by eGFR, is above 30.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have been diagnosed with a genetic condition that causes very high cholesterol.

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I have an active liver condition.

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I have high cholesterol due to another health condition like thyroid issues or kidney problems.

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I have not had a major heart problem in the last 3 months.

Timeline

Screening ~ 3 days1 visit

Treatment ~ Varies

Follow Up ~ 3 days1 visit

Screening ~ 3 days

Treatment ~ Varies

Follow Up ~3 days

This trial's timeline: 3 days for screening, Varies for treatment, and 3 days for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Percentage (%) change in low-density lipoprotein cholesterol (LDL-C) from baseline to Day 330

Secondary study objectives

% change and absolute change in LDL-C from baseline up to Day 720

% change and absolute change in other lipoproteins 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

+3 more

Side effects data

From 2019 Phase 3 trial • 482 Patients • NCT03397121

12%

Nasopharyngitis

Injection site reaction

Upper respiratory tract infection

Back pain

Influenza

Acute myocardial infarction

Aortic valve stenosis

100%

80%

60%

40%

20%

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

~3430

0 / 7Eligibility criteria met