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

EU CTR

Registry ID

EUCTR2020-002757-18-NO

Enrollment

130

Registered

2020-10-26

Start date

2020-11-26

Completion date

Unknown

Last updated

2025-09-15

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

Conditions

Heterozygous Familial Hypercholesterolemia MedDRA version: 20.0 Level: LLT Classification code 10057079 Term: Heterozygous familial hypercholesterolemia System Organ Class: 100000004850

Interventions

Trade Name: Leqvio 284 mg solution for injection in pre-filled syringe Product Name: inclisiran sodium Product Code: KJX839 Pharmaceutical Form: Solution for injection in pre-filled syringe INN or Pr

Eligibility

Sex/Gender

All

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 Other inclusion criteria are listed in the clinical study protocol Are the trial subjects under 18? yes Number of subjects for this age range: 130 F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range

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 exclusion criteria are listed in the clinical study protocol

Design outcomes

Primary

Measure	Time frame
Main Objective: Demonstrate superiority of inclisiran compared to placebo in reducing LDL-C [percent change] at Day 330 (Year 1);Secondary Objective: - Demonstrate superiority of inclisiran compared to placebo in reducing LDL-C [time-adjusted percent change] over Year 1 - Demonstrate superiority of inclisiran compared to placebo in reducing LDL-C [absolute change] at Day 330 (Year 1) - Demonstrate superiority of inclisiran compared to placebo in reducing Apo B, lipoprotein (a) [Lp(a)], non-high density lipoprotein cholesterol (non-HDL-C), and total cholesterol [percent change] at Day 330 (Year 1) Other secondary objectives are stated in the clinical study protocol ;Primary end point(s): Percentage (%) change in low-density lipoprotein cholesterol (LDL-C);Timepoint(s) of evaluation of this end point: baseline to Day 330	—

Measure

Time frame

Main Objective: Demonstrate superiority of inclisiran compared to placebo in reducing LDL-C [percent change] at Day 330 (Year 1);Secondary Objective: - Demonstrate superiority of inclisiran compared to placebo in reducing LDL-C [time-adjusted percent change] over Year 1 - Demonstrate superiority of inclisiran compared to placebo in reducing LDL-C [absolute change] at Day 330 (Year 1) - Demonstrate superiority of inclisiran compared to placebo in reducing Apo B, lipoprotein (a) [Lp(a)], non-high density lipoprotein cholesterol (non-HDL-C), and total cholesterol [percent change] at Day 330 (Year 1) Other secondary objectives are stated in the clinical study protocol ;Primary end point(s): Percentage (%) change in low-density lipoprotein cholesterol (LDL-C);Timepoint(s) of evaluation of this end point: baseline to Day 330

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Secondary

Measure	Time frame
Secondary end point(s): - Time-adjusted % change in LDL-C - Absolute change in LDL-C - % change in apolipoprotein B (Apo B), lipoprotein (a) [Lp (a)], non-high density lipoprotein cholesterol (non-HDL-C), and total cholesterol Other secondary endpoint can be found in the clinical study protocol ;Timepoint(s) of evaluation of this end point: - Baseline, after Day 90 up to Day 330 - Baseline and Day 330 - Baseline and Day 330	—

Measure

Time frame

Secondary end point(s): - Time-adjusted % change in LDL-C - Absolute change in LDL-C - % change in apolipoprotein B (Apo B), lipoprotein (a) [Lp (a)], non-high density lipoprotein cholesterol (non-HDL-C), and total cholesterol Other secondary endpoint can be found in the clinical study protocol ;Timepoint(s) of evaluation of this end point: - Baseline, after Day 90 up to Day 330 - Baseline and Day 330 - Baseline and Day 330

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Countries

Argentina, Brazil, Canada, Croatia, Czechia, Czech Republic, France, Germany, Greece, Hungary, Israel, Italy, Jordan, Lebanon, Malaysia, Netherlands, Norway, Poland, Russian Federation, Slovakia, Slovenia, South Africa, Spain, Switzerland, Taiwan, Turkey, United States

Contacts

Public ContactMedisinsk informasjon

Novartis Norge AS

medinfo.nordics@novartis.com+47 23 05 20 00

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026