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Completed

NCT Number: NCT04652726

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

This was 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).

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Key information

Conditions

Familial Hypercholesterolemia - Heterozygous

Age range

12 year–17 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Novartis Investigative Site, Formosa, Formosa Province, Argentina

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About this study

This was 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 was to demonstrate superiority of inclisiran compared to placebo in reducing LDL-C (percent change) at Day 330.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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 <400 mg/dL (4.5 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

Exclusion criteria

  • Homozygous familial hypercholesterolemia (HoFH)
  • Active liver disease
  • Secondary hypercholesterolemia, e.g. hypothyroidism or nephrotic syndrome
  • 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

Treatment and study plan

Inclisiran

Drug

Inclisiran sodium 300 mg (equivalent to 284 mg inclisiran) in 1.5 mL solution for subcutaneous injection

Other names: KJX839

Placebo

Drug

Sterile normal saline (0.9% sodium chloride in water for subcutaneous injection)

Other names: Saline solution

Primary outcomes

  1. Percentage Change in LDL-C From Baseline to Day 330 (Part 1/Year 1)

    Time frame: Baseline and Day 330

    Percentage change in low-density lipoprotein cholesterol (LDL-C) from baseline to Day 330 (Year 1)

Secondary outcomes

  1. Time-adjusted Percent Change in LDL-C From Baseline After Day 90 and up to Day 330 (Part 1/Year 1)

    Time frame: Baseline, after Day 90 up to Day 330

    Time-adjusted percent change in LDL-C (after Day 90 and up to Day 330), calculated as the average of percent changes from baseline to Days 150, 270 and 330

  2. Absolute Change in LDL-C From Baseline to up Day 330 (Part 1/Year 1)

    Time frame: Baseline and Day 330

    Absolute change in LDL-C from baseline to Day 330.

  3. Percent Change in Apo B From Baseline up to Day 330 (Part 1/Year 1)

    Time frame: Baseline and Day 330

    Percentage change in apolipoprotein B (Apo B) from baseline to Day 330.

  4. Percent Change in Lp(a) From Baseline up to Day 330 (Part 1/Year 1)

    Time frame: Baseline and Day 330

    Percentage change in lipoprotein (a) [Lp(a)] from baseline to Day 330.

  5. Percent Change in Non-HDL-C From Baseline up to Day 330 (Part 1/Year 1)

    Time frame: Baseline and Day 330

    Percentage change in non-high density lipoprotein cholesterol (non-HDL-C) from baseline to Day 330.

  6. Percent Change in Total Cholesterol From Baseline up to Day 330 (Part 1/Year 1)

    Time frame: Baseline and Day 330

    Percentage change in total cholesterol from baseline to Day 330.

  7. Percent Change in LDL-C From Baseline up to Day 720

    Time frame: Baseline, up to Day 720

    Percentage change in LDL-C from baseline to each assessment time up to Day 720.

  8. Absolute Change in LDL-C From Baseline up to Day 720

    Time frame: Baseline, up to Day 720

    Absolute change in LDL-C from baseline to each assessment time up to Day 720.

  9. Percent Change in Apo B From Baseline up to Day 720

    Time frame: Baseline, up to Day 720

    Percentage change in apolipoprotein B (Apo B) from baseline to each assessment time up to Day 720.

  10. Absolute Change in Apo B From Baseline up to Day 720

    Time frame: Baseline, up to Day 720

    Absolute change in apolipoprotein B (Apo B) from baseline to each assessment time up to Day 720.

  11. Percent Change in Lp(a) From Baseline up to Day 720

    Time frame: Baseline, up to Day 720

    Percentage change in lipoprotein (a) [Lp(a)] from baseline to each assessment time up to Day 720.

  12. Absolute Change in Lp(a) From Baseline up to Day 720

    Time frame: Baseline, up to Day 720

    Absolute change in lipoprotein (a) [Lp(a)] from baseline to each assessment time up to Day 720.

  13. Percent Change in Non-HDL-C From Baseline up to Day 720

    Time frame: Baseline, up to Day 720

    Percentage change in non-high density lipoprotein cholesterol (non-HDL-C) from baseline to each assessment time up to Day 720.

  14. Absolute Change in Non-HDL-C From Baseline up to Day 720

    Time frame: Baseline, up to Day 720

    Absolute change in non-high density lipoprotein cholesterol (non-HDL-C) from baseline to each assessment time up to Day 720.

  15. Percent Change in Total Cholesterol From Baseline up to Day 720

    Time frame: Baseline, up to Day 720

    Percentage change in total cholesterol from baseline to each assessment time up to Day 720.

  16. Absolute Change in Total Cholesterol From Baseline up to Day 720

    Time frame: Baseline, up to Day 720

    Absolute change in total cholesterol from baseline to each assessment time up to Day 720.

  17. Percent Change in Triglycerides From Baseline up to Day 720

    Time frame: Baseline, up to Day 720

    Percentage change in triglycerides from baseline to each assessment time up to Day 720.

  18. Absolute Change in Triglycerides From Baseline up to Day 720

    Time frame: Baseline, up to Day 720

    Absolute change in triglycerides from baseline to each assessment time up to Day 720.

  19. Percent Change in HDL-C From Baseline up to Day 720

    Time frame: Baseline, up to Day 720

    Percentage change in high density lipoprotein cholesterol (HDL-C) from baseline to each assessment time up to Day 720.

  20. Absolute Change in HDL-C From Baseline up to Day 720

    Time frame: Baseline, up to Day 720

    Absolute change in high density lipoprotein cholesterol (HDL-C) from baseline to each assessment time up to Day 720.

  21. Percent Change in VLDL-C From Baseline up to Day 720

    Time frame: Baseline, up to Day 720

    Percentage change in very low density lipoprotein cholesterol (VLDL-C) from baseline to each assessment time up to Day 720.

  22. Absolut Change in VLDL-C From Baseline up to Day 720

    Time frame: Baseline, up to Day 720

    Absolute change in very low density lipoprotein cholesterol (VLDL-C) from baseline to each assessment time up to Day 720.

  23. Percent Change in Apo A1 From Baseline up to Day 720

    Time frame: Baseline, up to Day 720

    Percentage change in apolipoprotein A1 (Apo A1) from baseline to each assessment time up to Day 720.

  24. Absolute Change in Apo A1 From Baseline up to Day 720

    Time frame: Baseline, up to Day 720

    Absolute change in apolipoprotein A1 (Apo A1) from baseline to each assessment time up to Day 720.

  25. Percent Change in PCSK9 From Baseline up to Day 720

    Time frame: Baseline, up to Day 720

    Percentage change in proprotein convertase subtilisin/kexin type 9 (PCSK9) from baseline to each assessment time up to Day 720.

  26. Absolut Change in PCSK9 From Baseline up to Day 720

    Time frame: Baseline, up to Day 720

    Absolute change in proprotein convertase subtilisin/kexin type 9 (PCSK9) from baseline to each assessment time up to Day 720.

Sponsors and collaborators

Lead sponsor

Novartis Pharmaceuticals

Industry

Registry information

Official study 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)

Acronym: ORION-16

Important dates

Study start
2021
Primary completion
2023
Study completion
2024
First posted
Dec 3, 2020
Registry last updated
Jan 13, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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