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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)

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) - CKJX839C12301 ORION-16

Status
Active, not recruiting
Phases
Phase 3
Study type
Interventional
Source
NL-OMON
Registry ID
NL-OMON54301
Enrollment
24
Registered
2020-11-20
Start date
2021-04-23
Completion date
Unknown
Last updated
2025-08-18

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

Conditions

elevated LDL-cholesterol heterozygous 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

Sponsors

Novartis
Lead Sponsor

Eligibility

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 days before screening • Estimated glomerular filtration rate (eGFR) > 30 mL/min/1.73 m2 at screening Other inclusion criteria are listed in the protocol

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

MeasureTime 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

MeasureTime frame
- 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) - 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 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)