Study to Evaluate Efficacy and Safety of Inclisiran in Adolescents With Homozygous Familial Hypercholesterolemia (ORION-13)

December 18, 2025 updated by: Novartis Pharmaceuticals

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 Homozygous Familial Hypercholesterolemia and Elevated LDL-cholesterol (ORION-13)

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

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Detailed Description

This was a two-part (double-blind, inclisiran versus placebo [Year 1] followed by open-label inclisiran [Year 2]) multicenter study in adolescents (aged 12 to < 18 years) with HoFH and elevated LDL-C (> 130 mg/dL; 3.4 mmol/L) on stable, individualized, optimal standard of care (SoC) background lipid-lowering therapy (including maximally tolerated statin treatment, at the Investigator's discretion) to evaluate the safety, tolerability, and efficacy of inclisiran in this pediatric patient population.

Following an approximately 4-week screening/run-in period, the study had 2 sequential parts as follows:

Part 1/Year 1: 12 months double-blind, parallel group period, in which participants were randomized in a 2:1 ratio to receive either inclisiran sodium 300 mg subcutaneous (s.c.) or placebo. The primary endpoint was assessed at Day 330.

Part 2/Year 2: 12 months single arm, open-label follow-up period, with all participants receiving inclisiran sodium 300 mg s.c.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
13

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Canada
    • Quebec
      • Québec, Quebec, Canada, G1V 4W2
        • Novartis Investigative Site
  • France
      • Bron, France, 69677
        • Novartis Investigative Site
  • Greece
      • Athens, Greece, 18547
        • Metropolitan Hospital
    • GR
      • Ioannina, GR, Greece, 455 00
        • University General Hospital of Ioannina
  • Lebanon
      • Beirut, Lebanon, 1107 2020
        • American University of Beirut Medical Center
      • El Achrafiyé, Lebanon, 166830
        • Hotel Dieu de France Hospital
  • Malaysia
      • Kuala Lumpur, Malaysia, 59100
        • Novartis Investigative Site
  • Netherlands
      • Amsterdam, Netherlands, 1105 AZ
        • Novartis Investigative Site
  • Turkey (Türkiye)
      • Izmir, Turkey (Türkiye), 35100
        • Novartis Investigative Site
    • TUR
      • Istanbul, TUR, Turkey (Türkiye), 34098
        • Novartis Investigative Site
  • United States
    • Florida
      • Boca Raton, Florida, United States, 33434
        • Excel Medical Clinical Trials LLC

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

12 years to 17 years (Child)

Accepts Healthy Volunteers

No

Description

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
  • Male or female participants >=12 to <18 years of age at screening

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
  • 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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Double

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: Part 1 - Inclisiran
Inclisiran sodium 300 mg subcutaneous (sc) injection (given at Days 1, 90 and 270)
Drug: Inclisiran
Inclisiran sodium 300 mg (equivalent to 284 mg inclisiran) in 1.5 mL solution for subcutaneous injection
Other Names:
  • KJX839
Placebo Comparator: Part 1 - Placebo
Placebo sc injection (given at Day 1, 90 and 270)
Drug: Placebo
Sterile normal saline (0.9% sodium chloride in water for injection) for subcutaneous injection
Other Names:
  • Saline solution
Experimental: Part 2 - Inclisiran (Total)
Inclisiran sodium 300 mg sc injection (given at Days 450 and 630). In addition, participants assigned to placebo in Part 1 received inclisiran sodium 300 mg sc injection on Day 360, while participants assigned to inclisiran in Part 1 received placebo sc injection on Day 360.
Drug: Inclisiran
Inclisiran sodium 300 mg (equivalent to 284 mg inclisiran) in 1.5 mL solution for subcutaneous injection
Other Names:
  • KJX839

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
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)
Baseline and Day 330

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
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
Baseline, after Day 90 up to Day 330
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.
Baseline, up to Day 720
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.
Baseline, up to Day 720
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.
Baseline, up to Day 720
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.
Baseline, up to Day 720
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.
Baseline, up to Day 720
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.
Baseline, up to Day 720
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.
Baseline, up to Day 720
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.
Baseline, up to Day 720
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.
Baseline, up to Day 720
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.
Baseline, up to Day 720
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.
Baseline, up to Day 720
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.
Baseline, up to Day 720
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.
Baseline, up to Day 720
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.
Baseline, up to Day 720
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.
Baseline, up to Day 720
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.
Baseline, up to Day 720
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.
Baseline, up to Day 720
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.
Baseline, up to Day 720
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.
Baseline, up to Day 720
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.
Baseline, up to Day 720

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Novartis Pharmaceuticals

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Novartis Pharmaceuticals, Novartis Pharmaceuticals

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
February 16, 2021

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
October 30, 2023

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
November 18, 2024

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
December 2, 2020

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
December 2, 2020

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
December 9, 2020

Study Record Updates

Last Update Posted (Estimated)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
January 13, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
December 18, 2025

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
December 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • CKJX839C12302
  • 2020-002755-38 (EudraCT Number)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.

This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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