Long-term Safety and Tolerability of Inclisiran in Participants With HeFH or HoFH Who Have Completed the Adolescent ORION-16 or ORION-13 Studies

NCT ID: NCT05682378

Last Updated: 2025-12-31

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE3

Total Enrollment

131 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-02-10

Study Completion Date

2028-03-02

Brief Summary

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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 or ORION-13 studies.

Detailed Description

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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.

Conditions

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Heterozygous or Homozygous Familial Hypercholesterolemia

Keywords

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

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Inclisiran

Inclisiran sodium 300mg (equivalent to 284mg inclisiran\*) in 1.5mL solution

Group Type EXPERIMENTAL

Inclisiran

Intervention Type DRUG

Inclisiran sodium 300mg (equivalent to 284mg inclisiran\*) in 1.5mL solution administered subcutaneously in pre-filled syringe

Interventions

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Inclisiran

Inclisiran sodium 300mg (equivalent to 284mg inclisiran\*) in 1.5mL solution administered subcutaneously in pre-filled syringe

Intervention Type DRUG

Other Intervention Names

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KJX839

Eligibility Criteria

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Inclusion Criteria

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

Key exclusion:

* Participants who in the feeder inclisiran ORION-16 and ORION-13 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
Minimum Eligible Age

12 Years

Maximum Eligible Age

100 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Novartis Pharmaceuticals

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Novartis Pharmaceuticals

Role: STUDY_DIRECTOR

Novartis Pharmaceuticals

Locations

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Excel Medical Clinical Trials LLC

Boca Raton, Florida, United States

Site Status

Icahn School of Med at Mt Sinai

New York, New York, United States

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Cincinnati Childrens Hospital MC

Cincinnati, Ohio, United States

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Childrens Hosp Pittsburgh UPMC

Pittsburgh, Pennsylvania, United States

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Primary Childrens Medical Center

Salt Lake City, Utah, United States

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Novartis Investigative Site

Formosa, Formosa Province, Argentina

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Fortaleza, Ceará, Brazil

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São Paulo, São Paulo, Brazil

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São Paulo, São Paulo, Brazil

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Québec, Quebec, Canada

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Prague, , Czechia

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Prague, , Czechia

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Besançon, , France

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Toulouse, , France

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Frankfurt am Main, Hesse, Germany

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Freiburg im Breisgau, , Germany

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Athens, , Greece

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Athens, , Greece

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Pécs, Baranya, Hungary

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Jerusalem, , Israel

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Ramat Gan, , Israel

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Milan, MI, Italy

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Modena, MO, Italy

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Roma, RM, Italy

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Roma, RM, Italy

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Irbid, , Jordan

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Beirut, , Lebanon

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Beirut, , Lebanon

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Sungai Buloh, Selangor, Malaysia

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Kuala Lumpur, , Malaysia

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Amsterdam, North Holland, Netherlands

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Rotterdam, South Holland, Netherlands

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Oslo, , Norway

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Gdansk, , Poland

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Lodz, Łódź Voivodeship, Poland

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Kemerovo, , Russia

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Moscow, , Russia

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Poprad, , Slovakia

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Ljubljana, , Slovenia

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Bloemfontein, Free State, South Africa

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Cape Town, Western Cape, South Africa

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Pamplona, Navarre, Spain

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Oviedo, Principality of Asturias, Spain

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A Coruña, , Spain

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Córdoba, , Spain

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Geneva, , Switzerland

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Taipei, , Taiwan

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Istanbul, Fatih, Turkey (Türkiye)

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Adana, Saricam, Turkey (Türkiye)

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Ankara, Yenimahalle, Turkey (Türkiye)

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Izmir, , Turkey (Türkiye)

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Novartis Investigative Site

Middlesex, , United Kingdom

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Countries

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United States Argentina Brazil Canada Czechia France Germany Greece Hungary Israel Italy Jordan Lebanon Malaysia Netherlands Norway Poland Russia Slovakia Slovenia South Africa Spain Switzerland Taiwan Turkey (Türkiye) United Kingdom

Other Identifiers

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2022-002316-23

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

CKJX839C12001B

Identifier Type: -

Identifier Source: org_study_id