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 · tracked via the Priya Life Science UK tracker

Sponsor

Novartis Pharmaceuticals

Phase

Phase 3

Started

2023-02-10

Last updated

2026-09-18

Condition(s) studied

Heterozygous or Homozygous Familial Hypercholesterolemia

Investigational drug(s) / intervention(s)

Inclisiran

Inclisiran: Solution for injection

Study 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.

Eligibility

Sex

ALL

Min age

2 Years

Max age

100 Years

Healthy volunteers

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

Primary outcome measure(s)

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

Trial sites (52)

Facility	City	Region	Status
Excel Medical Clinical Trials LLC	Boca Raton	Florida	Completed
Icahn School of Med at Mt Sinai	New York	New York	Active Not Recruiting
Cincinnati Childrens Hospital MC	Cincinnati	Ohio	Completed
Childrens Hosp Pittsburgh UPMC	Pittsburgh	Pennsylvania	Completed
Primary Childrens Medical Center	Salt Lake City	Utah	Recruiting
Novartis Investigative Site	Formosa	Formosa Province	Active Not Recruiting
Novartis Investigative Site	Fortaleza	Ceará	Active Not Recruiting
Novartis Investigative Site	São Paulo	São Paulo	Completed
Novartis Investigative Site	São Paulo	São Paulo	Active Not Recruiting
Novartis Investigative Site	Québec	Quebec	Active Not Recruiting
Novartis Investigative Site	Prague	Czechia	Active Not Recruiting
Novartis Investigative Site	Prague	Czechia	Active Not Recruiting
Novartis Investigative Site	Besançon	France	Active Not Recruiting
Novartis Investigative Site	Toulouse	France	Active Not Recruiting
Novartis Investigative Site	Frankfurt am Main	Hesse	Active Not Recruiting
Novartis Investigative Site	Freiburg im Breisgau	Germany	Completed
Novartis Investigative Site	Athens	Greece	Completed
Novartis Investigative Site	Athens	Greece	Active Not Recruiting
Novartis Investigative Site	Pécs	Baranya	Completed
Novartis Investigative Site	Jerusalem	Israel	Active Not Recruiting
Novartis Investigative Site	Ramat Gan	Israel	Active Not Recruiting
Novartis Investigative Site	Milan	MI	Completed
Novartis Investigative Site	Modena	MO	Completed
Novartis Investigative Site	Roma	RM	Completed
Novartis Investigative Site	Roma	RM	Active Not Recruiting
Novartis Investigative Site	Irbid	Jordan	Active Not Recruiting
Novartis Investigative Site	Beirut	Lebanon	Active Not Recruiting
Novartis Investigative Site	Beirut	Lebanon	Active Not Recruiting
Novartis Investigative Site	Sungai Buloh	Selangor	Active Not Recruiting
Novartis Investigative Site	Kuala Lumpur	Malaysia	Active Not Recruiting
Novartis Investigative Site	Amsterdam	North Holland	Active Not Recruiting
Novartis Investigative Site	Rotterdam	South Holland	Active Not Recruiting
Novartis Investigative Site	Oslo	Norway	Active Not Recruiting
Novartis Investigative Site	Gdansk	Poland	Active Not Recruiting
Novartis Investigative Site	Lodz	Łódź Voivodeship	Completed
Novartis Investigative Site	Kemerovo	Russia	Completed
Novartis Investigative Site	Moscow	Russia	Completed
Novartis Investigative Site	Poprad	Slovakia	Active Not Recruiting
Novartis Investigative Site	Ljubljana	Slovenia	Active Not Recruiting
Novartis Investigative Site	Bloemfontein	Free State	Active Not Recruiting

+ 12 more sites — see the full list on the official registry below.

On this site

📄 Leqvio (inclisiran) drug profile →

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Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT05682378 on ClinicalTrials.gov ↗ ← All trials in the UK