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Anti-sense oligonucleotide
Inclisiran for Hypercholesterolemia (ORION-19 Trial)
San Francisco, CA
Phase 3
Recruiting
Research Sponsored by Novartis Pharmaceuticals
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
* Male or female participants, 2 to \<12 years of age at screening
* HoFH diagnosed by genetic confirmation
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline, after day 90 up to day 330
Awards & highlights
Pivotal Trial
Summary
This trial will test the safety, side effects, and effectiveness of a drug called inclisiran in children aged 2 to under 12 years who have a genetic condition that causes high cholesterol levels.
See full description
Who is the study for?
This trial is for children aged 2 to less than 12 with homozygous familial hypercholesterolemia (HoFH), a genetic condition causing very high cholesterol. Participants must have genetically confirmed HoFH but can't join if they have certain mutations in both LDLR alleles.Check my eligibility
What is being tested?
The study tests the safety and effectiveness of Inclisiran, a medication aimed at lowering cholesterol levels, compared to a placebo. It's designed for young patients with severe inherited high cholesterol.See study design
What are the potential side effects?
While specific side effects for this age group are not detailed here, Inclisiran may generally cause injection site reactions, allergic responses, flu-like symptoms, and possibly liver enzyme elevations.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I am between 2 and 11 years old.
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My HoFH diagnosis was confirmed through genetic testing.
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I do not have negative mutations in both LDLR genes.
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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
Percentage change in LDL-C from baseline to Day 330 (Year 1)
Secondary study objectives
Absolute change in Apo B, Apo A1 from baseline to each assessment time up to Day 720 (Year 2)
Absolute change in LDL-C, total cholesterol, non-HDL-C, triglycerides, HDL-C, VLDL-C from baseline to each assessment time up to Day 720 (Year 2)
Absolute change in Lp(a) from baseline to each assessment time up to Day 720 (Year 2)
+6 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%
Aortic valve stenosis
1%
Acute myocardial infarction
100%
80%
60%
40%
20%
0%
Study treatment Arm
Inclisiran
Placebo
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 subcutaneous injection (given at Days 1, 90, and 270) Day 360 only - placebo subcutaneous injection Year 2 - inclisiran sodium 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 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
~3580
Find a Location
Closest Location:UC San Francisco Medical Center· San Francisco, CA
Who is running the clinical trial?
Novartis PharmaceuticalsLead Sponsor
2,959 Previous Clinical Trials
4,274,224 Total Patients Enrolled
25 Trials studying Hypercholesterolemia
29,730 Patients Enrolled for Hypercholesterolemia