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

NCT05682378 | Recruiting Phase 3 FDA Drug

• 2 other identifiers: CKJX839C12001B2022-002316-23
• Study Type: interventional
• Target: 195
• Locations: 25 countries
• Sites: 51

Brief Summary

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.

Conditions

Heterozygous or Homozygous Familial Hypercholesterolemia

Keywords

KJX839; heterozygous familial hypercholesterolemia; homozygous familial hypercholesterolemia; familial hypercholesterolemia; FH; inclisiran; pediatric

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (1)

• Percentage and absolute change in LDL-C from baseline in the feeder study to end of study

  Baseline (of feeder study) and Day 1080

Study Arms (1)

Inclisiran

EXPERIMENTAL

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

Interventions

Inclisiran DRUG

Solution for injection

Also known as: KJX839

Inclisiran

Eligibility Criteria

• Age: 12 Years - 100 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (52)

Excel Medical Clinical Trials LLC

Boca Raton, Florida, 33434, United States

COMPLETED

Icahn School of Med at Mt Sinai

New York, New York, 10029, United States

ACTIVE NOT RECRUITING

Cincinnati Childrens Hospital MC

Cincinnati, Ohio, 45229-3039, United States

COMPLETED

Childrens Hosp Pittsburgh UPMC

Pittsburgh, Pennsylvania, 15224, United States

ACTIVE NOT RECRUITING

Primary Childrens Medical Center

Salt Lake City, Utah, 84113, United States

COMPLETED

Novartis Investigative Site

Formosa, Formosa Province, P3600, Argentina

ACTIVE NOT RECRUITING

Novartis Investigative Site

Fortaleza, Ceará, 60430-275, Brazil

ACTIVE NOT RECRUITING

Novartis Investigative Site

São Paulo, São Paulo, 04023-900, Brazil

COMPLETED

Novartis Investigative Site

São Paulo, São Paulo, 05403 000, Brazil

ACTIVE NOT RECRUITING

Novartis Investigative Site

Québec, Quebec, G1V 4W2, Canada

ACTIVE NOT RECRUITING

Novartis Investigative Site

Prague, 128 08, Czechia

ACTIVE NOT RECRUITING

Novartis Investigative Site

Prague, 150 06, Czechia

RECRUITING

Novartis Investigative Site

Besançon, 25030, France

ACTIVE NOT RECRUITING

Novartis Investigative Site

Toulouse, 31059, France

ACTIVE NOT RECRUITING

Novartis Investigative Site

Frankfurt am Main, Hesse, 60590, Germany

ACTIVE NOT RECRUITING

Novartis Investigative Site

Freiburg im Breisgau, 79100, Germany

COMPLETED

Novartis Investigative Site

Athens, 115 27, Greece

COMPLETED

Novartis Investigative Site

Athens, 185 47, Greece

ACTIVE NOT RECRUITING

Novartis Investigative Site

Pécs, Baranya, 7623, Hungary

COMPLETED

Novartis Investigative Site

Jerusalem, 9112001, Israel

ACTIVE NOT RECRUITING

Novartis Investigative Site

Ramat Gan, 5265601, Israel

ACTIVE NOT RECRUITING

Novartis Investigative Site

Milan, MI, 20162, Italy

COMPLETED

Novartis Investigative Site

Modena, MO, 41124, Italy

COMPLETED

Novartis Investigative Site

Roma, RM, 00161, Italy

COMPLETED

Novartis Investigative Site

Roma, 165, Italy

ACTIVE NOT RECRUITING

Novartis Investigative Site

Irbid, 22110, Jordan

ACTIVE NOT RECRUITING

Novartis Investigative Site

Beirut, 113-0236, Lebanon

ACTIVE NOT RECRUITING

Novartis Investigative Site

Beirut, 166830, Lebanon

ACTIVE NOT RECRUITING

Novartis Investigative Site

Sungai Buloh, Selangor, 47000, Malaysia

ACTIVE NOT RECRUITING

Novartis Investigative Site

Kuala Lumpur, 59100, Malaysia

ACTIVE NOT RECRUITING

Novartis Investigative Site

Amsterdam, North Holland, 1105 AZ, Netherlands

ACTIVE NOT RECRUITING

Novartis Investigative Site

Rotterdam, South Holland, 3015 GD, Netherlands

ACTIVE NOT RECRUITING

Novartis Investigative Site

Oslo, 0514, Norway

ACTIVE NOT RECRUITING

Novartis Investigative Site

Gdansk, 80 952, Poland

ACTIVE NOT RECRUITING

Novartis Investigative Site

Lodz, Łódź Voivodeship, 93-338, Poland

COMPLETED

Novartis Investigative Site

Kemerovo, 650002, Russia

COMPLETED

Novartis Investigative Site

Moscow, 127412, Russia

COMPLETED

Novartis Investigative Site

Poprad, 058 01, Slovakia

ACTIVE NOT RECRUITING

Novartis Investigative Site

Ljubljana, 1000, Slovenia

ACTIVE NOT RECRUITING

Novartis Investigative Site

Bloemfontein, Free State, 9301, South Africa

COMPLETED

Novartis Investigative Site

Cape Town, Western Cape, 7130, South Africa

COMPLETED

Novartis Investigative Site

Pamplona, Navarre, 31008, Spain

ACTIVE NOT RECRUITING

Novartis Investigative Site

Oviedo, Principality of Asturias, 33011, Spain

COMPLETED

Novartis Investigative Site

A Coruña, 15001, Spain

COMPLETED

Novartis Investigative Site

Córdoba, 14004, Spain

ACTIVE NOT RECRUITING

Novartis Investigative Site

Geneva, 1211, Switzerland

ACTIVE NOT RECRUITING

Novartis Investigative Site

Taipei, 111045, Taiwan

ACTIVE NOT RECRUITING

Novartis Investigative Site

Istanbul, Fatih, 34098, Turkey (Türkiye)

ACTIVE NOT RECRUITING

Novartis Investigative Site

Adana, Saricam, 01330, Turkey (Türkiye)

ACTIVE NOT RECRUITING

Novartis Investigative Site

Ankara, Yenimahalle, 06500, Turkey (Türkiye)

ACTIVE NOT RECRUITING

Novartis Investigative Site

Izmir, 35100, Turkey (Türkiye)

ACTIVE NOT RECRUITING

Novartis Investigative Site

Middlesex, UB9 6JH, United Kingdom

ACTIVE NOT RECRUITING

MeSH Terms

Conditions

Homozygous Familial Hypercholesterolemia; Hyperlipoproteinemia Type II

Interventions

ALN-PCS

Condition Hierarchy (Ancestors)

Lipid Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Hyperlipoproteinemias; Hyperlipidemias; Dyslipidemias; Lipid Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Novartis Pharmaceuticals

  Novartis Pharmaceuticals

  STUDY DIRECTOR

Central Study Contacts

Novartis Pharmaceuticals

CONTACT

1-888-669-6682; novartis.email@novartis.com

Novartis Pharmaceuticals

CONTACT

+41613241111

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 27, 2022
• First Posted: January 12, 2023
• Study Start: February 10, 2023
• Primary Completion (Estimated): March 25, 2032
• Study Completion (Estimated): March 28, 2032
• Last Updated: May 1, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will share

Locations

PL (2); GB (1); JO (1); BR (3); RU (2); SL (1); IL (2); LE (2); DE (2); NO (1); US (5); TW (1); MY (2); FR (2); HU (1); CZ (2); IT (4); AR (1); GR (2); ES (4); CH (1); NL (2); ZA (2); CA (1); TU (4)