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

Sponsor

Novartis Pharmaceuticals (Industry)

Overall Status

Not yet recruiting

CT.gov ID

NCT05682378

Collaborator

(none)

165

Enrollment

Arm

57.4

Anticipated Duration (Months)

Study Details

Study Description

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 or ORION-13 studies.

Condition or Disease	Intervention/Treatment	Phase
Heterozygous or Homozygous Familial Hypercholesterolemia	Drug: Inclisiran	Phase 3

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.

Study Design

Study Type:

Interventional

Anticipated Enrollment :

165 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

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)

Anticipated Study Start Date :

Feb 6, 2023

Anticipated Primary Completion Date :

Nov 19, 2027

Anticipated Study Completion Date :

Nov 19, 2027

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Inclisiran Inclisiran sodium 300mg (equivalent to 284mg inclisiran*) in 1.5mL solution	Drug: Inclisiran Inclisiran sodium 300mg (equivalent to 284mg inclisiran*) in 1.5mL solution administered subcutaneously in pre-filled syringe Other Names: KJX839

Arm

Intervention/Treatment

Experimental: Inclisiran

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

Drug: Inclisiran

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

Other Names:

KJX839

Outcome Measures

Primary Outcome Measures

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 Outcome Measures

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

Eligibility Criteria

Criteria

Ages Eligible for Study:

12 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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