ClinicalTrials.gg
RecruitingNCT05682378V-PEDS-OLEUpdated Sep 18, 2026

Long-term Safety and Tolerability of Inclisiran in Participants With HeFH or HoFH Who Have Completed the Pediatric ORION-16, ORION-13, ORION-20, or ORION-19 Studies

A Phase 3 interventional study of Inclisiran in Heterozygous or Homozygous Familial Hypercholesterolemia, sponsored by Novartis Pharmaceuticals. Recruiting at 52 sites in 26 countries. Open to participants aged 2 Years to 100 Years. Per ClinicalTrials.gov, last updated 2026-09-18.

Sponsored by Novartis Pharmaceuticals (part of Novartis) · Phase 3, Interventional, and Treatment

From the registry’s dates

  • Started Feb 2023; still recruiting 3 years 9 months later.
Phase
Phase 3
Study type
Interventional
Enrollment
195
Allocation
Not applicable
Ages
2 Years to 100 Years
Sex
All
01

Study 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.

Read the 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.

02

Conditions studied

  • Heterozygous or Homozygous Familial Hypercholesterolemia

Keywords

  • KJX839
  • heterozygous familial hypercholesterolemia
  • homozygous familial hypercholesterolemia
  • familial hypercholesterolemia
  • FH
  • inclisiran
  • pediatric
03

In context

Homozygous Familial Hypercholesterolemia

68 studies on the registry are indexed under Homozygous Familial Hypercholesterolemia; 13 are open to participants now.

This study's planned enrollment of 195 is above the median of 21 across 51 interventional studies indexed under Homozygous Familial Hypercholesterolemia.

Browse Homozygous Familial Hypercholesterolemia studies →

Lead sponsor

Novartis Pharmaceuticals is the lead sponsor of 2,718 studies on the registry; 221 are open to participants now.

Of its 585 completed or terminated interventional studies of FDA-regulated products, 438 (75%) have results posted.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
2 Years to 100 Years
Sexes eligible
All
Accepts healthy volunteers
No

Eligibility 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
05

Study design

Phase
Phase 3
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
195 participants (estimated)

Study 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.

    Drug: Inclisiran

Interventions

  • Solution for injection

    Also known as: KJX839

06

What researchers measure

Primary outcomes

  1. 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)

    Time frame: From Day 1 in the study up to the end of study visit; up to 1080 days

Secondary outcomes

  1. 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

    Time frame: Baseline (of feeder study) and Day 1080

07

Study locations

2 of 52 sites recruiting
  • Excel Medical Clinical Trials LLC
    Boca Raton, Florida 33434, United States
    Completed

    Not enrolling at this site — choose another above.

  • Icahn School of Med at Mt Sinai
    New York, New York 10029, United States
    Active, not recruiting

    Not enrolling at this site — choose another above.

  • Cincinnati Childrens Hospital MC
    Cincinnati, Ohio 45229-3039, United States
    Completed

    Not enrolling at this site — choose another above.

  • Childrens Hosp Pittsburgh UPMC
    Pittsburgh, Pennsylvania 15224, United States
    Completed

    Not enrolling at this site — choose another above.

  • Primary Childrens Medical Center
    Salt Lake City, Utah 84113, United States
    Recruiting
  • Novartis Investigative Site
    Formosa, Formosa Province P3600MPD, Argentina
    Active, not recruiting

    Not enrolling at this site — choose another above.

  • Novartis Investigative Site
    Fortaleza, Ceará 60430-275, Brazil
    Active, not recruiting

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  • Novartis Investigative Site
    São Paulo, São Paulo 04023-900, Brazil
    Completed

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  • Novartis Investigative Site
    São Paulo, São Paulo 05403-000, Brazil
    Active, not recruiting

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  • Novartis Investigative Site
    Québec, Quebec G1V 4W2, Canada
    Active, not recruiting

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  • Novartis Investigative Site
    Prague, 128 08, Czechia
    Active, not recruiting

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  • Novartis Investigative Site
    Prague, 150 06, Czechia
    Active, not recruiting

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  • Novartis Investigative Site
    Besançon, 25030, France
    Active, not recruiting

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  • Novartis Investigative Site
    Toulouse, 31059, France
    Active, not recruiting

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  • Novartis Investigative Site
    Frankfurt am Main, Hesse 60590, Germany
    Active, not recruiting

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  • Novartis Investigative Site
    Freiburg im Breisgau, 79100, Germany
    Completed

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  • Novartis Investigative Site
    Athens, 115 27, Greece
    Completed

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  • Novartis Investigative Site
    Athens, 185 47, Greece
    Active, not recruiting

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  • Novartis Investigative Site
    Pécs, Baranya 7623, Hungary
    Completed

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  • Novartis Investigative Site
    Jerusalem, 9112001, Israel
    Active, not recruiting

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  • Novartis Investigative Site
    Ramat Gan, 5265601, Israel
    Active, not recruiting

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  • Novartis Investigative Site
    Milan, MI 20162, Italy
    Completed

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  • Novartis Investigative Site
    Modena, MO 41124, Italy
    Completed

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  • Novartis Investigative Site
    Roma, RM 00161, Italy
    Completed

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  • Novartis Investigative Site
    Roma, RM 00165, Italy
    Active, not recruiting

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  • Novartis Investigative Site
    Irbid, 22110, Jordan
    Active, not recruiting

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  • Novartis Investigative Site
    Beirut, 1100 2070, Lebanon
    Active, not recruiting

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  • Novartis Investigative Site
    Beirut, 113-0236, Lebanon
    Active, not recruiting

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  • Novartis Investigative Site
    Sungai Buloh, Selangor 47000, Malaysia
    Active, not recruiting

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  • Novartis Investigative Site
    Kuala Lumpur, 59100, Malaysia
    Active, not recruiting

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  • Novartis Investigative Site
    Amsterdam, North Holland 1105 AZ, Netherlands
    Active, not recruiting

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  • Novartis Investigative Site
    Rotterdam, South Holland 3015 GD, Netherlands
    Active, not recruiting

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  • Novartis Investigative Site
    Oslo, 0514, Norway
    Active, not recruiting

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  • Novartis Investigative Site
    Gdansk, 80-952, Poland
    Active, not recruiting

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  • Novartis Investigative Site
    Lodz, Łódź Voivodeship 93-338, Poland
    Completed

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  • Novartis Investigative Site
    Kemerovo, 650002, Russia
    Completed

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  • Novartis Investigative Site
    Moscow, 127412, Russia
    Completed

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  • Novartis Investigative Site
    Poprad, 058 01, Slovakia
    Active, not recruiting

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  • Novartis Investigative Site
    Ljubljana, 1000, Slovenia
    Active, not recruiting

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  • Novartis Investigative Site
    Bloemfontein, Free State 9301, South Africa
    Active, not recruiting

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  • Novartis Investigative Site
    Cape Town, Western Cape 7130, South Africa
    Completed

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  • Novartis Investigative Site
    Pamplona, Navarre 31008, Spain
    Active, not recruiting

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  • Novartis Investigative Site
    Oviedo, Principality of Asturias 33011, Spain
    Completed

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  • Novartis Investigative Site
    A Coruña, 15001, Spain
    Completed

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  • Novartis Investigative Site
    Córdoba, 14004, Spain
    Active, not recruiting

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  • Novartis Investigative Site
    Geneva, 1211, Switzerland
    Completed

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  • Novartis Investigative Site
    Taipei, 111045, Taiwan
    Recruiting
  • Novartis Investigative Site
    Istanbul, Fatih 34098, Turkey (Türkiye)
    Active, not recruiting

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  • Novartis Investigative Site
    Adana, Saricam 01330, Turkey (Türkiye)
    Active, not recruiting

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  • Novartis Investigative Site
    Ankara, Yenimahalle 06500, Turkey (Türkiye)
    Active, not recruiting

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  • Novartis Investigative Site
    Izmir, 35100, Turkey (Türkiye)
    Active, not recruiting

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  • Novartis Investigative Site
    Middlesex, UB9 6JH, United Kingdom
    Active, not recruiting

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08

References and documents

Individual participant data

Plan to share: Yes — Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com

No publications or documents are linked to this record.

09

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Sep 18, 2026, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
10

Registry details

Key details

Study ID
NCT05682378
Responsible party
Sponsor
First posted
Jan 12, 2023
Start date
Feb 10, 2023
Primary completion
Mar 25, 2032 (estimated)
Completion
Mar 28, 2032 (estimated)
Last update
Sep 18, 2026

Study contacts

Novartis Pharmaceuticals
Contact
novartis.email@novartis.com
1-888-669-6682
Novartis Pharmaceuticals
Contact
+41613241111
Novartis Pharmaceuticals
study director · Novartis Pharmaceuticals

Oversight

Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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Studies of the drug tested here: Inclisiran

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