Long-term safety and tolerability of inclisiran in participants with heterozygous familial hypercholesterolaemia or homozygous familial hypercholesterolaemia who have completed the adolescent ORION-16 or ORION-13 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 adolescent ORION-16 or ORION-13 studies (VICTORION-PEDS-OLE)

Status

Not yet recruiting

Phases

Phase 3

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2022-002316-23-DE

Enrollment

165

Registered

2022-10-25

Start date

2023-04-04

Completion date

Unknown

Last updated

2023-09-18

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

Conditions

Familial Hypercholesterolemia MedDRA version: 20.0 Level: LLT Classification code 10049593 Term: Familial hypercholesterolaemia System Organ Class: 100000004850

Interventions

Trade Name: Leqvio 284 mg solution for injection in pre-filled syringe Product Name: inclisiran sodium Product Code: KJX839 Pharmaceutical Form: Solution for injection in pre-filled syringe INN or Pro

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: • Male and female participants with a diagnosis of HeFH or HoFH who completed the ORION-16 or ORION-13 studies, meaning the participant received the last dose of study drug and completed the final study visit as per applicable protocol • Per investigator`s clinical judgment, participant derived benefit from treatment with inclisiran in the ORION-16 or ORION-13 studies • Continuing current lipid-lowering therapies (such as e.g. statin and/or ezetimibe) from the feeder study with no planned medication or dose change during study participation Are the trial subjects under 18? yes Number of subjects for this age range: 103 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 62 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range

Exclusion criteria

Exclusion criteria: • Participants who in the preceding 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 • Active liver disease defined as any known current infectious, neoplastic, or metabolic pathology of the liver

Design outcomes

Primary

Measure	Time frame
Main Objective: The primary objective of the study is to evaluate the long-term safety and tolerability of inclisiran in participants with HeFH or HoFH;Secondary Objective: To evaluate the long-term effect of inclisiran treatment on LDL-C levels;Primary end point(s): Incidence, severity and relationship to study drug of treatment-emergent adverse events (TEAEs) and serious adverse events (TESAEs); vital signs; laboratory parameters ;Timepoint(s) of evaluation of this end point: From day 1 to the end of study	—

Measure

Time frame

Main Objective: The primary objective of the study is to evaluate the long-term safety and tolerability of inclisiran in participants with HeFH or HoFH;Secondary Objective: To evaluate the long-term effect of inclisiran treatment on LDL-C levels;Primary end point(s): Incidence, severity and relationship to study drug of treatment-emergent adverse events (TEAEs) and serious adverse events (TESAEs); vital signs; laboratory parameters ;Timepoint(s) of evaluation of this end point: From day 1 to the end of study

—

Secondary

Measure	Time frame
Secondary end point(s): Percentage change and absolute change in LDL-C from baseline in the feeder study to end of study (EoS) ;Timepoint(s) of evaluation of this end point: End of study	—

Countries

Brazil, Canada, France, Germany, Greece, Hungary, Israel, Italy, Lebanon, Malaysia, Netherlands, Norway, Poland, Russian Federation, Slovenia, South Africa, Spain, Switzerland, Taiwan, Turkey, United Kingdom, United States

Contacts

Public ContactMedizinischer Infoservice (MCC)

Novartis Pharma GmbH

infoservice.novartis@novartis.com+49 911 273-12100

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026