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Long-term safety and tolerability of inclisiran for patients with familial hypercholesterolemia

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)

Phase 3 Interventional Novartis · NCT05682378

This study is testing if the cholesterol-lowering drug inclisiran is safe and well-tolerated for people with familial hypercholesterolemia who have already tried it in earlier studies.

Quick facts

PhasePhase 3
Study typeInterventional
Enrollment195 (estimated)
Ages12 Years to 100 Years
SexAll
SponsorNovartis Industry-sponsored
Locations52 sites (Boca Raton, Florida and 51 other locations)
Trial IDNCT05682378 on ClinicalTrials.gov

What this trial studies

This open-label, single-arm, multicenter study aims to evaluate the long-term safety and tolerability of inclisiran in participants diagnosed with heterozygous or homozygous familial hypercholesterolemia who have previously completed the ORION-16 or ORION-13 studies. Participants will have continued access to inclisiran treatment while the study monitors their health outcomes over an extended period. The study is designed to gather data on the ongoing effects of inclisiran in this specific patient population.

Who should consider this trial

Good fit: Ideal candidates are male and female participants with a diagnosis of heterozygous or homozygous familial hypercholesterolemia who have completed the ORION-16 or ORION-13 studies and benefited from inclisiran treatment.

Not a fit: Patients who experienced serious safety or tolerability issues related to inclisiran in previous studies or those with uncontrolled serious diseases may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could provide valuable insights into the long-term safety of inclisiran, potentially leading to improved management of familial hypercholesterolemia.

How similar studies have performed: Other studies involving inclisiran have shown promising results, indicating that this approach has potential based on previous findings.

Eligibility criteria

Show full inclusion / exclusion 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

Where this trial is running

Boca Raton, Florida and 51 other locations

+2 more sites — see ClinicalTrials.gov for the full list.

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Heterozygous or Homozygous Familial HypercholesterolemiaKJX839heterozygous familial hypercholesterolemiahomozygous familial hypercholesterolemiafamilial hypercholesterolemiaFHinclisiranpediatric
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.