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)

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) - CKJX839C12302 ORION13

Status

Recruiting

Phases

Phase 3

Study type

Interventional

Source

NL-OMON

Registry ID

NL-OMON54036

Enrollment

Registered

2020-12-21

Start date

2021-05-07

Completion date

Unknown

Last updated

2026-06-29

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

Conditions

elevated LDL-cholesterol homozygous familial hypercholesterolemia

Interventions

Participants will be randomized 2:1 to double-blind s.c. injections of inclisiran sodium 300 mg or placebo in Part 1 (Year 1) of the study, and subsequently all participants will receive open-label

Eligibility

Age

12 Years to 17 Years

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. Other inclusion criteria are noted in the protocol.

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
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	—

Secondary

Measure	Time frame
- Demonstrate superiority of inclisiran compared to placebo in 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, Apo B, lipoprotein (a) [Lp(a)], non-high density lipoprotein cholesterol (non-HDL-Cother lipoprotein and lipid parameters, and PCSK9 over time - Evaluate the safety and tolerability profile of inclisiran, compared to placebo (for Year 1) and long-term (up to Day 720), in adolescents (aged 12 to	—

Measure

Time frame

- Demonstrate superiority of inclisiran compared to placebo in 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, Apo B, lipoprotein (a) [Lp(a)], non-high density lipoprotein cholesterol (non-HDL-Cother lipoprotein and lipid parameters, and PCSK9 over time - Evaluate the safety and tolerability profile of inclisiran, compared to placebo (for Year 1) and long-term (up to Day 720), in adolescents (aged 12 to

—

Countries

Netherlands

Outcome results

None listed

Source: NL-OMON (via WHO ICTRP) · Data processed: Jul 3, 2026