Long-term Safety and Tolerability of Inclisiran in Participants With HeFH or HoFH Who Have Completed the Pediatric ORION-16, ORION-13, ORION-20, or ORION-19 Studies (V-PEDS-OLE)

An Open-label, Single Arm, Multicenter Extension Study to Evaluate Long-term Safety and Tolerability of Inclisiran in Participants With Heterozygous or Homozygous Familial Hypercholesterolemia Who Have Completed the Pediatric ORION-16, ORION-13, ORION-20 or ORION-19 Studies (VICTORION-PEDS-OLE)

The purpose of this open-label, single arm, multicenter extension study is to evaluate the long-term safety and tolerability of inclisiran in participants with HeFH or HoFH who have completed the ORION-16 (CKJX839C12301), ORION-13 (CKJX839C12302), ORION-20 (CKJX839C12303) or ORION-19 (CKJX839C12304) studies.

Study Overview

• Status: Recruiting
• Conditions: Heterozygous or Homozygous Familial Hypercholesterolemia
• Intervention / Treatment: Drug: Inclisiran

Detailed Description

This is an open-label, single arm, multicenter study designed to evaluate long-term safety and tolerability of inclisiran. In addition, the study will provide participants the opportunity to have continued access to treatment with inclisiran.

• Study Type: Interventional
• Enrollment (Estimated): 195
• Phase: Phase 3

Contacts and Locations

• Study Contact: Name: Novartis Pharmaceuticals; Phone Number: 1-888-669-6682; Email: novartis.email@novartis.com
• Study Contact Backup: Name: Novartis Pharmaceuticals; Phone Number: +41613241111

Study Locations

• Argentina
  • Formosa Province
    • Formosa, Formosa Province, Argentina, P3600
      • Active, not recruiting
      • Novartis Investigative Site
• Brazil
  • Ceará
    • Fortaleza, Ceará, Brazil, 60430-275
      • Active, not recruiting
      • Novartis Investigative Site
  • São Paulo
    • São Paulo, São Paulo, Brazil, 05403 000
      • Active, not recruiting
      • Novartis Investigative Site
    • São Paulo, São Paulo, Brazil, 04023-900
      • Completed
      • Novartis Investigative Site
• Canada
  • Quebec
    • Québec, Quebec, Canada, G1V 4W2
      • Active, not recruiting
      • Novartis Investigative Site
• Czechia
  • Prague, Czechia, 128 08
    • Active, not recruiting
    • Novartis Investigative Site
  • Prague, Czechia, 150 06
    • Active, not recruiting
    • Novartis Investigative Site
• France
  • Besançon, France, 25030
    • Active, not recruiting
    • Novartis Investigative Site
  • Toulouse, France, 31059
    • Active, not recruiting
    • Novartis Investigative Site
• Germany
  • Freiburg im Breisgau, Germany, 79100
    • Completed
    • Novartis Investigative Site
  • Hesse
    • Frankfurt am Main, Hesse, Germany, 60590
      • Active, not recruiting
      • Novartis Investigative Site
• Greece
  • Athens, Greece, 115 27
    • Completed
    • Novartis Investigative Site
  • Athens, Greece, 185 47
    • Active, not recruiting
    • Novartis Investigative Site
• Hungary
  • Baranya
    • Pécs, Baranya, Hungary, 7623
      • Completed
      • Novartis Investigative Site
• Israel
  • Jerusalem, Israel, 9112001
    • Active, not recruiting
    • Novartis Investigative Site
  • Ramat Gan, Israel, 5265601
    • Active, not recruiting
    • Novartis Investigative Site
• Italy
  • MI
    • Milan, MI, Italy, 20162
      • Completed
      • Novartis Investigative Site
  • MO
    • Modena, MO, Italy, 41124
      • Completed
      • Novartis Investigative Site
  • RM
    • Roma, RM, Italy, 00161
      • Completed
      • Novartis Investigative Site
    • Roma, RM, Italy, 00165
      • Active, not recruiting
      • Novartis Investigative Site
• Jordan
  • Irbid, Jordan, 22110
    • Active, not recruiting
    • Novartis Investigative Site
• Lebanon
  • Beirut, Lebanon, 113-0236
    • Active, not recruiting
    • Novartis Investigative Site
  • Beirut, Lebanon, 166830
    • Active, not recruiting
    • Novartis Investigative Site
• Malaysia
  • Kuala Lumpur, Malaysia, 59100
    • Active, not recruiting
    • Novartis Investigative Site
  • Selangor
    • Sungai Buloh, Selangor, Malaysia, 47000
      • Active, not recruiting
      • Novartis Investigative Site
• Netherlands
  • North Holland
    • Amsterdam, North Holland, Netherlands, 1105 AZ
      • Active, not recruiting
      • Novartis Investigative Site
  • South Holland
    • Rotterdam, South Holland, Netherlands, 3015 GD
      • Active, not recruiting
      • Novartis Investigative Site
• Norway
  • Oslo, Norway, 0514
    • Active, not recruiting
    • Novartis Investigative Site
• Poland
  • Gdansk, Poland, 80 952
    • Active, not recruiting
    • Novartis Investigative Site
  • Łódź Voivodeship
    • Lodz, Łódź Voivodeship, Poland, 93-338
      • Completed
      • Novartis Investigative Site
• Russia
  • Kemerovo, Russia, 650002
    • Completed
    • Novartis Investigative Site
  • Moscow, Russia, 127412
    • Completed
    • Novartis Investigative Site
• Slovakia
  • Poprad, Slovakia, 058 01
    • Active, not recruiting
    • Novartis Investigative Site
• Slovenia
  • Ljubljana, Slovenia, 1000
    • Active, not recruiting
    • Novartis Investigative Site
• South Africa
  • Free State
    • Bloemfontein, Free State, South Africa, 9301
      • Completed
      • Novartis Investigative Site
  • Western Cape
    • Cape Town, Western Cape, South Africa, 7130
      • Completed
      • Novartis Investigative Site
• Spain
  • A Coruña, Spain, 15001
    • Completed
    • Novartis Investigative Site
  • Córdoba, Spain, 14004
    • Active, not recruiting
    • Novartis Investigative Site
  • Navarre
    • Pamplona, Navarre, Spain, 31008
      • Active, not recruiting
      • Novartis Investigative Site
  • Principality of Asturias
    • Oviedo, Principality of Asturias, Spain, 33011
      • Completed
      • Novartis Investigative Site
• Switzerland
  • Geneva, Switzerland, 1211
    • Active, not recruiting
    • Novartis Investigative Site
• Taiwan
  • Taipei, Taiwan, 111045
    • Recruiting
    • Novartis Investigative Site
• Turkey (Türkiye)
  • Izmir, Turkey (Türkiye), 35100
    • Active, not recruiting
    • Novartis Investigative Site
  • Fatih
    • Istanbul, Fatih, Turkey (Türkiye), 34098
      • Active, not recruiting
      • Novartis Investigative Site
  • Saricam
    • Adana, Saricam, Turkey (Türkiye), 01330
      • Active, not recruiting
      • Novartis Investigative Site
  • Yenimahalle
    • Ankara, Yenimahalle, Turkey (Türkiye), 06500
      • Active, not recruiting
      • Novartis Investigative Site
• United Kingdom
  • Middlesex, United Kingdom, UB9 6JH
    • Active, not recruiting
    • Novartis Investigative Site
• United States
  • Florida
    • Boca Raton, Florida, United States, 33434
      • Completed
      • Excel Medical Clinical Trials LLC
  • New York
    • New York, New York, United States, 10029
      • Active, not recruiting
      • Icahn School of Med at Mt Sinai
  • Ohio
    • Cincinnati, Ohio, United States, 45229-3039
      • Completed
      • Cincinnati Childrens Hospital MC
  • Pennsylvania
    • Pittsburgh, Pennsylvania, United States, 15224
      • Active, not recruiting
      • Childrens Hosp Pittsburgh UPMC
  • Utah
    • Salt Lake City, Utah, United States, 84113
      • Completed
      • Primary Childrens Medical Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 12 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Key inclusion:

• Male and female participants with a diagnosis of HeFH or HoFH who completed the ORION-16, ORION-13, ORION-20 or ORION-19 studies
• Per investigator's clinical judgment, participant derived benefit from treatment with inclisiran in the ORION-16, ORION-13, ORION-20 or ORION-19 studies

Key exclusion:

• Participants who in the feeder ORION-16, ORION-13, ORION-20, or ORION-19 studies either screen failed or permanently discontinued from the treatment/study for any reason or had serious safety or tolerability issues related to inclisiran treatment
• Any uncontrolled or serious disease, or any medical, physical, or surgical condition, that may either interfere with participation in the clinical study or interpretation of clinical study results, and/or put the participant at significant risk

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Inclisiran; Participants who have completed ORION-16 or ORION-13 receive inclisiran sodium 300mg (equivalent to 284mg inclisiran*) in 1.5mL solution. Participants who have completed ORION-19 and ORION-20 receive Inclisiran dose levels based on weight.	Drug: Inclisiran; Solution for injection; Other Names: KJX839

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of participants with treatment-emergent adverse events (TEAEs) and serious adverse events (TESAEs)	Safety and tolerability: TEAEs, TESAEs (incidence, severity, relationship to study drug and discontinuation due to TEAEs)	From Day 1 in the study up to the end of study visit; up to 1080 days

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percentage and absolute change in LDL-C from baseline in the feeder study to end of study	Evaluate the long-term effect of inclisiran (from baseline of feeder study to end of study) in lowering LDL-C	Baseline (of feeder study) and Day 1080

Collaborators and Investigators

• Sponsor: Novartis Pharmaceuticals
• Investigators: Study Director: Novartis Pharmaceuticals, Novartis Pharmaceuticals

Study record dates

Study Major Dates

• Study Start (Actual): February 10, 2023
• Primary Completion (Estimated): March 25, 2032
• Study Completion (Estimated): March 28, 2032

Study Registration Dates

• First Submitted: December 27, 2022
• First Submitted That Met QC Criteria: December 27, 2022
• First Posted (Actual): January 12, 2023

Study Record Updates

• Last Update Posted (Actual): May 15, 2026
• Last Update Submitted That Met QC Criteria: May 13, 2026
• Last Verified: May 1, 2026

More Information

Terms related to this study

• Keywords: pediatric; FH; heterozygous familial hypercholesterolemia; familial hypercholesterolemia; inclisiran; KJX839; homozygous familial hypercholesterolemia
• Additional Relevant MeSH Terms: Metabolism, Inborn Errors; Genetic Diseases, Inborn; Metabolic Diseases; Hyperlipidemias; Dyslipidemias; Lipid Metabolism Disorders; Lipid Metabolism, Inborn Errors; Hyperlipoproteinemias; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Nutritional and Metabolic Diseases; Homozygous Familial Hypercholesterolemia; Hyperlipoproteinemia Type II; ALN-PCS
• Other Study ID Numbers: CKJX839C12001B; 2022-002316-23 (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