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

Phase 3

Recruiting

• Conditions: Heterozygous or Homozygous Familial Hypercholesterolemia
• Interventions: Drug: Inclisiran

• Registration Number: NCT05682378
• Lead Sponsor: Novartis Pharmaceuticals

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.

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 Timeline

• Study First Submitted: 2022-12-27
• Study First Submitted QC: 2022-12-27
• Study First Posted: 2023-01-12
• Study Start: 2023-02-10
• Status Verified: 2025-03
• Last Update Submitted: 2025-03-18
• Last Update Posted: 2025-03-19
• Primary Completion: 2028-03-02
• Study Completion: 2028-03-02

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 154

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Inclisiran	Inclisiran	Inclisiran sodium 300mg (equivalent to 284mg inclisiran\*) in 1.5mL solution

Primary Outcome Measures

Name	Time	Method
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

Name	Time	Method
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

Trial Locations

Locations (6)

Excel Medical Clinical Trials LLC; 🇺🇸 Boca Raton, Florida, United States

Icahn School of Med at Mt Sinai; 🇺🇸 New York, New York, United States

Cincinnati Childrens Hospital MC; 🇺🇸 Cincinnati, Ohio, United States

Childrens Hosp Pittsburgh UPMC; 🇺🇸 Pittsburgh, Pennsylvania, United States

Primary Childrens Medical Center; 🇺🇸 Salt Lake City, Utah, United States

Novartis Investigative Site; 🇬🇧 Middlesex, United Kingdom