Heterozygous or Homozygous Familial HypercholesterolemiaPhase 3V-PEDS-OLENCT05682378
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 study testing Inclisiran in people with Heterozygous or Homozygous Familial Hypercholesterolemia, run by Novartis Pharmaceuticals at 52 sites in 26 countries.
Trial 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 more about this study
- Who is it for?
- Ages 2 to 100 years
- Conditions the study is for
- Heterozygous or Homozygous Familial Hypercholesterolemia
- What is being tested?
- Inclisiran
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 195 participants
- Where
- 52 sitesin 26 countries
- Study dates
- 10 Feb 2023to 28 Mar 2032
- Phase
- Phase 3
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 to 100 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
52 sites in 26 countries. Contact the study team or a site near you to ask about taking part.
Novartis Pharmaceuticals contact
52 sites in 26 countries
United States5 sites, 1 recruiting
- Completed
Excel Medical Clinical Trials LLC
Boca Raton, Florida, 33434
- Completed
Cincinnati Childrens Hospital MC
Cincinnati, Ohio, 45229-3039
- Active, not recruiting
Icahn School of Med at Mt Sinai
New York, New York, 10029
- Completed
Childrens Hosp Pittsburgh UPMC
Pittsburgh, Pennsylvania, 15224
- Recruiting
Primary Childrens Medical Center
Salt Lake City, Utah, 84113
Linda Lambert, Linda.lambert@hsc.utah.edu
Adam Ware
Italy4 sites
- Completed
Novartis Investigative Site
Milan, MI, 20162
- Completed
Novartis Investigative Site
Modena, MO, 41124
- Completed
Novartis Investigative Site
Roma, RM, 00161
- Active, not recruiting
Novartis Investigative Site
Roma, RM, 00165
Spain4 sites
- Completed
Novartis Investigative Site
A Coruña, 15001
- Active, not recruiting
Novartis Investigative Site
Córdoba, 14004
- Completed
Novartis Investigative Site
Oviedo, Principality of Asturias, 33011
- Active, not recruiting
Novartis Investigative Site
Pamplona, Navarre, 31008
Turkey (Türkiye)4 sites
- Active, not recruiting
Novartis Investigative Site
Adana, Saricam, 01330
- Active, not recruiting
Novartis Investigative Site
Ankara, Yenimahalle, 06500
- Active, not recruiting
Novartis Investigative Site
Istanbul, Fatih, 34098
- Active, not recruiting
Novartis Investigative Site
Izmir, 35100
Brazil3 sites
- Active, not recruiting
Novartis Investigative Site
Fortaleza, Ceará, 60430-275
- Completed
Novartis Investigative Site
São Paulo, São Paulo, 04023-900
- Active, not recruiting
Novartis Investigative Site
São Paulo, São Paulo, 05403-000
Czechia2 sites
- Active, not recruiting
Novartis Investigative Site
Prague, 128 08
- Active, not recruiting
Novartis Investigative Site
Prague, 150 06
France2 sites
- Active, not recruiting
Novartis Investigative Site
Besançon, 25030
- Active, not recruiting
Novartis Investigative Site
Toulouse, 31059
Germany2 sites
- Active, not recruiting
Novartis Investigative Site
Frankfurt am Main, Hesse, 60590
- Completed
Novartis Investigative Site
Freiburg im Breisgau, 79100
Greece2 sites
- Completed
Novartis Investigative Site
Athens, 115 27
- Active, not recruiting
Novartis Investigative Site
Athens, 185 47
Israel2 sites
- Active, not recruiting
Novartis Investigative Site
Jerusalem, 9112001
- Active, not recruiting
Novartis Investigative Site
Ramat Gan, 5265601
Lebanon2 sites
- Active, not recruiting
Novartis Investigative Site
Beirut, 1100 2070
- Active, not recruiting
Novartis Investigative Site
Beirut, 113-0236
Malaysia2 sites
- Active, not recruiting
Novartis Investigative Site
Kuala Lumpur, 59100
- Active, not recruiting
Novartis Investigative Site
Sungai Buloh, Selangor, 47000
Netherlands2 sites
- Active, not recruiting
Novartis Investigative Site
Amsterdam, North Holland, 1105 AZ
- Active, not recruiting
Novartis Investigative Site
Rotterdam, South Holland, 3015 GD
Poland2 sites
- Active, not recruiting
Novartis Investigative Site
Gdansk, 80-952
- Completed
Novartis Investigative Site
Lodz, Łódź Voivodeship, 93-338
Russia2 sites
- Completed
Novartis Investigative Site
Kemerovo, 650002
- Completed
Novartis Investigative Site
Moscow, 127412
South Africa2 sites
- Active, not recruiting
Novartis Investigative Site
Bloemfontein, Free State, 9301
- Completed
Novartis Investigative Site
Cape Town, Western Cape, 7130
Argentina1 site
- Active, not recruiting
Novartis Investigative Site
Formosa, Formosa Province, P3600MPD
Canada1 site
- Active, not recruiting
Novartis Investigative Site
Québec, Quebec, G1V 4W2
Slovakia1 site
- Active, not recruiting
Novartis Investigative Site
Poprad, 058 01
Slovenia1 site
- Active, not recruiting
Novartis Investigative Site
Ljubljana, 1000
Taiwan1 site, 1 recruiting
- Recruiting
Novartis Investigative Site
Taipei, 111045
United Kingdom1 site
- Active, not recruiting
Novartis Investigative Site
Middlesex, UB9 6JH
About this study
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 Pediatric ORION-16, ORION-13, ORION-20 or ORION-19 Studies (VICTORION-PEDS-OLE)
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.
- Being tested
- Inclisiran
- Keywords
- KJX839, heterozygous familial hypercholesterolemia, homozygous familial hypercholesterolemia, familial hypercholesterolemia, FH, inclisiran, pediatric
- Medical subject headings
- Homozygous Familial Hypercholesterolemia, Hyperlipoproteinemia Type II
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
InclisiranDrug
Also called KJX839
Solution for injection
| Study group | Role | What they receive |
|---|---|---|
| Inclisiran | Experimental | 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. Receives: Drug: Inclisiran |
What is being measured
The primary outcome is the main question the study is designed to answer.
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)
Measured: 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
Measured: Baseline (of feeder study) and Day 1080
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Jan 2023
- Study start10 Feb 2023
- Primary completion25 Mar 2032 (estimated)
- Study completion28 Mar 2032 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Heterozygous or Homozygous Familial HypercholesterolemiaStudies of the same condition, open and under way.
- More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 195 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Novartis Pharmaceuticals, Study director, Novartis Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05682378
- Sponsor's protocol ID
- CKJX839C12001B
- Other IDs
- 2022-002316-23
- Acronym
- V-PEDS-OLE
- Status last verified
- Sep 2026
- First submitted
- 27 Dec 2022
- Last updated
- 18 Sep 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- 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