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NCT04652726 Clinical Trial

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

Familial Hypercholesterolemia - Heterozygous Clinical Trial

— ORION-16  

Official title:
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)

Clinical Trial Details — Status: Active, not recruiting

Administrative data
NCT number NCT04652726
Other study ID # CKJX839C12301
Secondary ID 2020-002757-18
Status Active, not recruiting
Phase Phase 3
First received
Last updated
Start date January 27, 2021
Est. completion date December 2, 2024

Study information
Verified date June 2024
Source Novartis
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

This is a pivotal phase III study designed to evaluate safety, tolerability, and efficacy of inclisiran in adolescents with heterozygous familial hypercholesterolemia (HeFH) and elevated low density lipoprotein cholesterol (LDL-C).

Description:

This is a two-part (1 year double-blind inclisiran versus placebo / 1 year open-label inclisiran) multicenter study designed to evaluate safety, tolerability, and efficacy of inclisiran in adolescents with heterozygous familial hypercholesterolemia (HeFH) and elevated low density lipoprotein cholesterol (LDL-C) on stable standard of care background lipid-lowering therapy. The primary objective is to demonstrate superiority of inclisiran compared to placebo in reducing LDL-C (percent change) at Day 330.

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Study Design

Related Conditions & MeSH terms

Intervention

Drug:
• Inclisiran
Inclisiran sodium 300 mg (equivalent to 284 mg inclisiran) in 1.5 mL solution for subcutaneous injection
• Placebo
Sterile normal saline (0.9% sodium chloride in water for subcutaneous injection)
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Sponsors (1)
Lead Sponsor Collaborator
Novartis Pharmaceuticals

Countries where clinical trial is conducted

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

Outcome
Type Measure Description Time frame Safety issue
Primary Percentage (%) change in low-density lipoprotein cholesterol (LDL-C) from baseline to Day 330 Demonstrate superiority of inclisiran compared to placebo in reducing LDL-C [percent change] at Day 330 (Year 1) Baseline and Day 330
Secondary Time-adjusted % change in LDL-C from baseline after Day 90 and up to Day 330 Demonstrate superiority of inclisiran compared to placebo in reducing LDL-C [time-adjusted percent change] over Year 1 Baseline, after Day 90 up to Day 330
Secondary Absolute change in LDL-C from baseline to Day 330 Demonstrate superiority of inclisiran compared to placebo in reducing LDL-C [absolute change] at Day 330 (Year 1) Baseline and Day 330
Secondary % 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 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)
- Hierarchical testing		 Baseline and Day 330
Secondary % change and absolute change in LDL-C from baseline up to Day 720 Evaluate the effect of inclisiran, compared to placebo (for Year 1) and long-term (up to Day 720), on lowering LDL-C over time Baseline, up to Day 720
Secondary % change and absolute change in other lipoproteins and lipid parameters from baseline up to Day 720 Evaluate the effect of inclisiran, compared to placebo (for Year 1) and long-term (up to Day 720), on lowering Apo B, Lp(a), non-HDL-C, total cholesterol, triglycerides, high density lipoprotein cholesterol (HDL-C), very low density lipoprotein cholesterol (VLDL-C), apolipoprotein A1 (Apo A1) over time Baseline, up to Day 720
Secondary % change and absolute change in proprotein convertase subtilisin/kexin type 9 (PCSK9) from baseline up to Day 720 Evaluate the effect of inclisiran, compared to placebo (for Year 1) and long-term (up to Day 720), on lowering PCSK9 over time Baseline, up to Day 720
See also
  Status Clinical Trial Phase
Recruiting NCT02517944 - MYOCARDIAL SILENT INFARCTIONS AND FIBROSIS IN FAMILIAL HYPERCHOLESTEROLEMIA (CHOLCOEUR) N/A
Completed NCT03501875 - Study of Coronary Calcification in Subjects With Autosomal Dominant Familial Hypercholesterolemia Heterozygous N/A
Recruiting NCT04370899 - Early Detection of Familial Hypercholesterolemia in Children
Recruiting NCT04656028 - Genetic Testing and Motivational Counseling for FH N/A
Recruiting NCT05218005 - Advancing Cardiac Care Unit-based Rapid Assessment and Treatment of hypErcholesterolemia N/A