Study to Evaluate Efficacy and Safety of Inclisiran in Adolescents With Heterozygous Familial Hypercholesterolemia

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 Adolescents (12 to Less Than 18 Years) With Heterozygous Familial Hypercholesterolemia and Elevated LDL-cholesterol (ORION-16)

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

LBCTR

Registry ID

LBCTR2021034776

Enrollment

Registered

2025-08-14

Start date

2021-08-31

Completion date

Unknown

Last updated

2026-02-02

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Heterozygous Familial Hypercholesterolemia heterozygous familial hypercholesterolemia

Interventions

Drug: Inclisiran Drug: Placebo

Informed Consent/assent form discussion

Inclusion/exclusion assessment

physical examination

neurological examination

blood and urine samples collection

IMP dispensation

ICF, IMP , Lab tests, physical exams

Eligibility

Sex/Gender

All

Age

12 Years to 17 Years

Inclusion criteria

Inclusion criteria: 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 Fasting triglycerides 30 mL/min/1.73 m2 at screening

Exclusion criteria

Exclusion criteria: Homozygous familial hypercholesterolemia (HoFH) Active liver disease Secondary hypercholesterolemia, e.g. hypothyroidism or nephrotic syndrome Major adverse cardiovascular events within 3 months prior to randomization Previous treatment with monoclonal antibodies directed towards PCSK9 (within 90 days of screening) Recent and/or planned use of other investigational medicinal products or devices Other protocol-defined inclusion/exclusion criteria may apply

Design outcomes

Primary

Measure	Time frame
Name: Percentage (%) change in low-density lipoprotein cholesterol (LDL-C);Timepoints: baseline to Day 330 ;Measure: baseline to Day 330 ;Name: Demonstrate superiority of inclisiran compared to placebo in reducing LDL-C [percent change];Timepoints: Day 330 (Year 1);Measure: Day 330 (Year 1)	—

Secondary

Measure	Time frame
Name: Time-adjusted % change in LDL-C from baseline;Timepoints: Baseline, after Day 90 up to Day 330;Measure: Baseline, after Day 90 up to Day 330;Name: Absolute change in LDL-C from baseline to Day 330;Timepoints: Baseline and Day 330;Measure: Baseline and Day 330;Name: % change in apolipoprotein B (Apo B), lipoprotein (a) [Lp(a)], non-high density lipoprotein cholesterol (non-HDL-C), and total cholesterol from baseline to Day 330;Timepoints: Baseline and Day 330;Measure: Baseline and Day 330;Name: % change and absolute change in LDL-C from baseline up to Day 720;Timepoints: Baseline, up to Day 720;Measure: Baseline, up to Day 720;Name: % change and absolute change in other lipoproteins and lipid parameters;Timepoints: Baseline, up to Day 720;Measure: Baseline, up to Day 720;Name: % change and absolute change in proprotein convertase subtilisin/kexin type 9 (PCSK9);Timepoints: Baseline, up to Day 720;Measure: Baseline, up to Day 720	—

Measure

Time frame

Name: Time-adjusted % change in LDL-C from baseline;Timepoints: Baseline, after Day 90 up to Day 330;Measure: Baseline, after Day 90 up to Day 330;Name: Absolute change in LDL-C from baseline to Day 330;Timepoints: Baseline and Day 330;Measure: Baseline and Day 330;Name: % change in apolipoprotein B (Apo B), lipoprotein (a) [Lp(a)], non-high density lipoprotein cholesterol (non-HDL-C), and total cholesterol from baseline to Day 330;Timepoints: Baseline and Day 330;Measure: Baseline and Day 330;Name: % change and absolute change in LDL-C from baseline up to Day 720;Timepoints: Baseline, up to Day 720;Measure: Baseline, up to Day 720;Name: % change and absolute change in other lipoproteins and lipid parameters;Timepoints: Baseline, up to Day 720;Measure: Baseline, up to Day 720;Name: % change and absolute change in proprotein convertase subtilisin/kexin type 9 (PCSK9);Timepoints: Baseline, up to Day 720;Measure: Baseline, up to Day 720

—

Countries

Australia, Germany, Hungary, Lebanon, Norway, Spain, United States of America

Contacts

Public ContactSelim Jambart

Hotel Dieu De France

sjambart@dm.net.lb961 3 406 001

Outcome results

None listed

Source: LBCTR (via WHO ICTRP) · Data processed: Feb 7, 2026