Recruiting
Phase 3

Inclisiran

Sponsor:

Novartis Pharmaceuticals

Code:

NCT05682378

Conditions

Heterozygous or Homozygous Familial Hypercholesterolemia

Eligibility Criteria

Sex: All

Age: 2 - 70+

Healthy Volunteers: Not accepted

Interventions

Inclisiran

Study Details

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 (CKJX839C12301), ORION-13 (CKJX839C12302), ORION-20 (CKJX839C12303) or ORION-19 (CKJX839C12304) studies.

Conditions

Heterozygous or Homozygous Familial Hypercholesterolemia

Study ID

NCT05682378

Start date

Feb 10, 2023

Status verified date

Sep, 2026

Completion date

Mar 28, 2032

Anticipated

Primary completion date

Mar 25, 2032

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 2 - 70+

Healthy Volunteers: Not accepted

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

Study Design

Enrollment

195 participants

Anticipated

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Inclisiran

Participants who have completed ORION-16 or ORION-13 receive inclisiran sodium 300mg (equivalent to 284mg inclisiran\*) in 1.5mL solution.

Participants who have completed ORION-19 and ORION-20 receive Inclisiran dose levels based on weight.

Interventions

Inclisiran

Solution for injection

Primary outcome measure

  • Number of participants with treatment-emergent adverse events (TEAEs) and serious adverse events (TESAEs) [ Time Frame: From Day 1 in the study up to the end of study visit; up to 1080 days ]

Central Contacts and Locations

Central contacts

Novartis Pharmaceuticals

1-888-669-6682novartis.email@novartis.com

Novartis Pharmaceuticals

+41613241111

Locations

Primary Childrens Medical Center

Recruiting

Salt Lake City, Utah, United States, 84113

Contacts

Linda Lambert

Linda.lambert@hsc.utah.edu

Principal Investigator:

Adam Ware

More Information

Sponsor

Novartis Pharmaceuticals

Last update posted

Sep 18, 2026

Last verified

Sep, 2026

Keywords

  • KJX839
  • heterozygous familial hypercholesterolemia
  • homozygous familial hypercholesterolemia
  • familial hypercholesterolemia
  • FH
  • inclisiran
  • pediatric

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-24. This information was provided to ClinicalTrials.gov by Novartis Pharmaceuticals on 2026-09-18. Recruitment status is synced daily from ClinicalTrials.gov and may not reflect the sponsor's current status. Confirm during your call.