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

Study Overview
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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.

Official Title
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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)

Conditions
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Heterozygous or Homozygous Familial Hypercholesterolemia

Intervention / Treatment
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* Drug: Inclisiran


Participation Criteria
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Eligibility Criteria
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Key inclusion: 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

Ages Eligible for Study
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Minimum Age: 12 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Inclisiran<br>Inclisiran sodium 300mg (equivalent to 284mg inclisiran*) in 1.5mL solution | Drug: Inclisiran<br>* Inclisiran sodium 300mg (equivalent to 284mg inclisiran*) in 1.5mL solution administered subcutaneously in pre-filled syringe<br>* Other names: KJX839;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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 Outcome Measures

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

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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KJX839, heterozygous familial hypercholesterolemia, homozygous familial hypercholesterolemia, familial hypercholesterolemia, FH, inclisiran, pediatric