Study to evaluate efficacy and safety of inclisiran in adolescents with homozygous familial hypercholesterolemia

Two part (double-blind) inclisiran versus placebo [Year 1] followed by open-label inclisiran [Year 2] randomized multicentre study to evaluate safety, tolerability, and efficacy of inclisiran in adolescents (12 to less than 18 years) with homozygous familial hypercholesterolemia and elevated LDL-cholesterol (ORION-13)

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2020-002755-38-SI

Enrollment

Registered

2020-12-02

Start date

2020-12-30

Completion date

Unknown

Last updated

2024-12-03

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

Conditions

Homozygous Familial Hypercholesterolemia MedDRA version: 20.0 Level: LLT Classification code 10057080 Term: Homozygous familial hypercholesterolemia System Organ Class: 10010331 - Congenital, familial and genetic disorders

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: Homozygous Familial Hypercholesterolemia (HoFH) diagnosed by genetic confirmation. Fasting LDL-C >130 mg/dL (3.4 mmol/L) at screening. On maximally tolerated dose of statin (investigator’s discretion) with or without other lipid-lowering therapy; stable for = 30 days before screening. Estimated glomerular filtration rate (eGFR) >30 mL/min/1.73 m2 at screening. Are the trial subjects under 18? yes Number of subjects for this age range: 12 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: Documented evidence of a null (negative) mutation in both LDLR alleles. Heterozygous familial hypercholesterolemia (HeFH). Active liver disease. Secondary hypercholesterolemia, e.g. hypothyroidism or nephrotic syndrome. Major adverse cardiovascular events within 1 month prior to randomization. Previous treatment with monoclonal antibodies directed towards PCSK9 (within 90 days of screening). Treatment with mipomersen or lomitapide (within 5 months of screening). Recent and/or planned use of other investigational medicinal products or devices.

Design outcomes

Primary

Measure	Time frame
Main Objective: The primary objective is to demonstrate superiority of inclisiran compared to placebo in reducing LDL-C [percent change] at Day 330 (Year 1) in adolescents (aged 12 to <18 years) with HoFH and elevated LDL-C ;Secondary Objective: Evaluate the effect of inclisiran compared to placebo on reducing LDL-C [time-adjusted percent change] over Year 1. Evaluate the effect of inclisiran compared to placebo (for Year 1) and long-term (up to Day 720), on lowering LDL-C, other lipoprotein and lipid parameters, and PCSK9 over time. Evaluate the safety and tolerability of inclisiran compared to placebo (for Year 1) and long-term (up to Day 720), in adolescents (aged 12 to <18 years) with HoFH.;Primary end point(s): Percentage (%) change in low-density lipoprotein cholesterol (LDL-C) from baseline to Day 330.;Timepoint(s) of evaluation of this end point: Baseline and Day 330	—

Measure

Time frame

Main Objective: The primary objective is to demonstrate superiority of inclisiran compared to placebo in reducing LDL-C [percent change] at Day 330 (Year 1) in adolescents (aged 12 to <18 years) with HoFH and elevated LDL-C ;Secondary Objective: Evaluate the effect of inclisiran compared to placebo on reducing LDL-C [time-adjusted percent change] over Year 1. Evaluate the effect of inclisiran compared to placebo (for Year 1) and long-term (up to Day 720), on lowering LDL-C, other lipoprotein and lipid parameters, and PCSK9 over time. Evaluate the safety and tolerability of inclisiran compared to placebo (for Year 1) and long-term (up to Day 720), in adolescents (aged 12 to <18 years) with HoFH.;Primary end point(s): Percentage (%) change in low-density lipoprotein cholesterol (LDL-C) from baseline to Day 330.;Timepoint(s) of evaluation of this end point: Baseline and Day 330

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Secondary

Measure	Time frame
Secondary end point(s): Time-adjusted percent change in LDL-C from baseline after Day 90 and up to Day 330. Percent change and absolute change in LDL-C from baseline up to Day 720. Percent change and absolute change in other lipoprotein and lipid parameters from baseline up to Day 720. Percent change and absolute change in proprotein convertase subtilisin/kexin type 9 (PCSK9) from baseline up to Day 720.;Timepoint(s) of evaluation of this end point: Baseline, after Day 90 up to Day 330. Baseline, up to Day 720. Baseline, up to Day 720. Baseline, up to Day 720.	—

Measure

Time frame

Secondary end point(s): Time-adjusted percent change in LDL-C from baseline after Day 90 and up to Day 330. Percent change and absolute change in LDL-C from baseline up to Day 720. Percent change and absolute change in other lipoprotein and lipid parameters from baseline up to Day 720. Percent change and absolute change in proprotein convertase subtilisin/kexin type 9 (PCSK9) from baseline up to Day 720.;Timepoint(s) of evaluation of this end point: Baseline, after Day 90 up to Day 330. Baseline, up to Day 720. Baseline, up to Day 720. Baseline, up to Day 720.

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Countries

Canada, France, Germany, Greece, Italy, Lebanon, Netherlands, Serbia, Slovenia, South Africa, Switzerland, Türkiye, United States, Viet Nam

Contacts

Public ContactMedical Information Desk

Novartis Pharma Services Inc. Podružnica v Sloveniji

medinfo.slo@novartis.com+38613 007550

Outcome results

None listed

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