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

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)

Status

Recruiting

Phases

Phase 3

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT05682378

Acronym

V-PEDS-OLE

Enrollment

195

Registered

2023-01-12

Start date

2023-02-10

Completion date

2032-03-28

Last updated

2026-09-18

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Heterozygous or Homozygous Familial Hypercholesterolemia

Keywords

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

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.

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.

Interventions

DRUGInclisiran

Solution for injection

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

2 Years to 100 Years

Healthy volunteers

Inclusion criteria

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

Design outcomes

Primary

Measure	Time frame	Description
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)

Secondary

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

Countries

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, United States

Contacts

CONTACTNovartis Pharmaceuticals

novartis.email@novartis.com1-888-669-6682

STUDY_DIRECTORNovartis Pharmaceuticals

Novartis Pharmaceuticals

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Sep 19, 2026