Study of Efficacy, Safety, Tolerability and Quality of Life of Inclisiran (KJX839) vs Placebo, on Top of Ongoing Individually Optimized Lipid-lowering Therapy, in Participants With Hypercholesterolemia

NCT ID: NCT05192941

Last Updated: 2025-06-11

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Clinical Phase

PHASE4

Total Enrollment

1776 participants

Study Classification

INTERVENTIONAL

Study Start Date

2022-04-08

Study Completion Date

2025-03-19

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

Study of efficacy, safety, tolerability and quality of life of inclisiran (KJX839) vs placebo, on top of ongoing individually optimized lipid-lowering therapy, in participants with hypercholesterolemia

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

The purpose of this study is to demonstrate the superiority of inclisiran compared to placebo, both on top of ongoing individually optimized lipid-lowering therapy (LLT), on reaching a participant's LDL-C target (\< 55 mg/dL or \< 70 mg/dL, depending on the cardiovascular risk category), according to the 2019 ESC/EAS guidelines for the management of dyslipidemias as well as on patient-relevant safety, tolerability outcomes and quality of life.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Hypercholesterolemia

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Double-blind investigational treatment inclisiran/placebo: subcutaneous injections of inclisiran sodium 300 mg (equivalent to 284 mg inclisiran) or placebo, in 1.5 mL solution.

Open label study treatment rosuvastatin: all participants will receive rosuvastatin, starting at the lowest indicated dose and titrating up until they reach their individual LDL-C target or MTD (Maximum tolerated dose) of statin, whichever occurs first.
Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors
Participants will be randomized at the baseline visit (Day 1) to one of the following two double-blind treatment groups in a 1:1 ratio.

1. Inclisiran sodium 300 mg s.c.
2. Corresponding placebo Each participant will receive one injection of blinded inclisiran or placebo on Day 1, a second injection of blinded inclisiran or placebo on Day 90 and a subsequent injection of blinded inclisiran or placebo on Day 270, unless there is a need for discontinuing inclisiran/placebo in the course of the study

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

Inclisiran sodium 300 mg s.c. + open label rosuvastatin

Participants will be randomized at the baseline visit (Day 1) to one of the following two double-blind treatment groups in a 1:1 ratio.

1. Inclisiran sodium 300 mg s.c.
2. Corresponding placebo

Open label study treatment rosuvastatin: all participants will receive rosuvastatin, starting at the lowest indicated dose and titrating up until they reach their individual LDL-C target or MTD of statin, whichever occurs first.

Group Type EXPERIMENTAL

Inclisiran Sodium

Intervention Type DRUG

Subcutaneously injected on Day 1, Day 90, and Day 270

Corresponding placebo + open label rosuvastatin

Participants will be randomized at the baseline visit (Day 1) to one of the following two double-blind treatment groups in a 1:1 ratio.

1. Inclisiran sodium 300 mg s.c.
2. Corresponding placebo

Open label study treatment rosuvastatin: all participants will receive rosuvastatin, starting at the lowest indicated dose and titrating up until they reach their individual LDL-C target or MTD of statin, whichever occurs first.

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Placebo to inclisiran 300 mg subcutaneously

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

Inclisiran Sodium

Subcutaneously injected on Day 1, Day 90, and Day 270

Intervention Type DRUG

Placebo

Placebo to inclisiran 300 mg subcutaneously

Intervention Type DRUG

Other Intervention Names

Discover alternative or legacy names that may be used to describe the listed interventions across different sources.

KJX839

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

Participants eligible for inclusion in this study must meet all of the following criteria:

1. Written informed consent must be obtained before any assessment is performed.
2. Male or female participants ≥18 years of age.
3. Participants categorized as very high or high CV risk, as defined below:

•Very high risk participants with at least one of the following: A. Documented Atherosclerotic Cardiovascular Disease (ASCVD) i) Acute Coronary Syndrome: Unstable angina or myocardial infarction ii) Stable angina iii) Unequivocally documented ASCVD upon prior imaging v) Stroke and Transient Ischaemic Attack (TIA) vi) Peripheral Artery Disease (PAD) B. Diabetes mellitus (DM) with target organ damage (defined as microalbuminuria, retinopathy, or neuropathy), or at least ≥ 3 major risk factors, or early onset of Type 1 DM of long duration (\> 20 years) C. A calculated SCORE2 ≥ 7.5% for age \< 50 years; SCORE2 ≥ 10% for age 50-69 years; SCORE2-OP ≥ 15% for age ≥ 70 years to estimate 10-year risk of fatal and non-fatal CVD D. Pre-existing diagnosis of heterozygous familial hypercholesterolemia (HeFH) with ASCVD or with another major risk factor.

OR

•High risk participants with at least one of the following: A. Markedly elevated single risk factors, in particular total cholesterol \> 310 mg/dL, LDL-C \> 190 mg/dL, or blood pressure ≥ 180/110 mmHg B. Pre-existing diagnosis of HeFH without other major risk factors C. DM without target organ damage (defined as microalbuminuria, retinopathy, or neuropathy), with DM duration ≥ 10 years or other additional risk factor D. Moderate chronic kidney disease (eGFR 30-59 mL/min/1.73m2) E. A calculated SCORE2 2.5 to \<7.5% for age under 50 years; SCORE2 5 to \<10% for age 50-69 years; SCORE2-OP 7.5 to \<15% for age ≥70 years to estimate 10-year risk of fatal and non-fatal CVD as defined by the cardiovascular risk categories in the 2019 ESC/EAS guideline (Mach et al 2020)
4. LDL-C levels:

1. in participants with very high cardiovascular risk: serum LDL-C ≥55 mg/dL
2. in participants with high cardiovascular risk: serum LDL-C ≥70 mg/dL
5. Participant on a stable dose of a statin for ≥ 30 days.
6. Up to approximately 20% of total participants can be on a stable dose (for ≥ 30 days prior to screening) of another LLT on top of statin such as a cholesterol absorbing inhibitor or a bile acid sequestrant, or alternatively, an adenosine triphosphate citrate lyase (ACL) inhibitor, as indicated.
7. Fasting triglyceride \< 400 mg/dL.

At Baseline:
8. Fasting triglyceride \< 400 mg/dL.
9. Before randomization, despite being treated with the individual MTD of a statin for ≥ 30 days and, if applicable, with another LLT on top of statin (stable for ≥ 30 days),

1. in participants with very high cardiovascular risk: serum LDL-C ≥ 55mg/dL.
2. in participants with high cardiovascular risk: serum LDL-C ≥ 70mg/dL.

Exclusion Criteria

1. Severe concomitant non-CV disease that is expected to reduce life expectancy to less than 2 years at screening or baseline visit.
2. Participants on more than one other lipid-lowering drug on top of statin at screening visit.
3. Pre-existing diagnosis of homozygous familial hypercholesterolemia at screening or baseline visit.
4. Secondary hypercholesterolemia, e.g. hypothyroidism or nephrotic syndrome at screening or baseline visit.
5. Previous (within 90 days of screening), current or planned treatment with a monoclonal antibody (mAb) directed towards PCSK9 (e.g. evolocumab, alirocumab) at screening or baseline visit.
6. Previous exposure to inclisiran or any other non-mAb PCSK9 targeted therapy, either as an investigational or marketed drug within 2 years prior to screening or baseline visit.
7. Previous, current or planned treatment with LDL-apheresis at screening or baseline visit.
8. Participants with known intolerance to rosuvastatin at screening or baseline visit.
9. History of hypersensitivity to any of the study treatments, inclisiran or rosuvastatin, or its excipients or to drugs of similar chemical classes at screening or baseline visit.
10. Participants taking gemfibrozil at screening or baseline visit.
11. Liver and CK: (a) Active liver disease defined as any current infectious, neoplastic, or metabolic pathology of the liver or (b) unexplained alanine aminotransferase (ALT), aspartate aminotransferase (AST) elevation \>3x ULN, or total bilirubin elevation \> 2x ULN (except for participants with Gilbert's syndrome), or (c) creatine kinase (CK) \>5x ULN at screening or baseline visit.
12. Participant with severe renal impairment defined by eGFR \<30 mL/min/1.73m2 as calculated by the Modification in Diet in Renal Disease (MDRD) formula at screening or baseline visit.
13. Acute coronary syndrome, ischemic stroke or TIA, coronary revascularization or peripheral arterial revascularization procedure or amputation due to atherosclerotic disease \< 3 months prior to the screening or baseline visit.
14. Planned or expected cardiac, cerebrovascular or peripheral artery surgery or coronary revascularization within the study duration.
15. Heart failure New York Heart Association (NYHA) class IV at screening or baseline visit.
16. History of malignancy that required surgery (excluding local and wide-local excision), radiation therapy and/or systemic therapy (excluding systemic adjuvant therapies given to prevent cancer recurrence eg: hormonotherapy for prostate or breast cancer) during the 3 years prior to screening or baseline visit.
17. Participant with myopathy at screening or baseline visit.
18. Participant receiving concomitant ciclosporin at screening or baseline visit.
19. Participants that are predisposed to the development of renal failure secondary to rhabdomyolysis (e.g. sepsis, hypotension, major surgery, trauma, severe metabolic, endocrine and electrolyte disorders; or uncontrolled seizures).
20. Participants with rare hereditary problems of galactose intolerance, the Lapp lactase deficiency or glucose-galactose-malabsorption.
21. Unwillingness or inability (e.g. physical or cognitive) to comply with study procedures (including study visits, fasting blood draws and compliance with study treatment regimens), and medication administration (injections) and schedule. Participant should be able and willing to read, understand and answer questionnaires.
22. Any surgical or medical condition, which in the opinion of the investigator, may place the participant at higher risk from his/her participation in the study, or is likely to prevent the participant from complying with the requirements of the study or completing the study at screening or baseline visit.
23. Use of other investigational drugs within 5 half-lives, 30 days or until the expected pharmacodynamic effect has returned to baseline (e.g. biologics), whichever is longer or longer if required by local regulation, prior to screening visit.
24. Pregnant or nursing (lactating) women at screening or baseline visit.
25. Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using highly effective methods of contraception while taking study treatment, which includes rosuvastatin, and for 5 days (= 5 times the terminal half-life of rosuvastatin) after stopping medication. Highly effective contraception methods include:

* Total abstinence (when this is in line with the preferred and usual lifestyle of the participant. Periodic abstinence (e.g. calendar, ovulation, symptothermal, post-ovulation methods) and withdrawal are not acceptable methods of contraception.
* Female sterilization (have had surgical bilateral oophorectomy with or without hysterectomy), total hysterectomy, or bilateral tubal ligation at least six weeks before taking study treatment. In case of oophorectomy alone, only when the reproductive status of the woman has been confirmed by follow up hormone level assessment.
* Male sterilization (at least 6 months prior to screening). For female participants on the study, the vasectomized male partner should be the sole partner for that participant.
* Use of oral, (estrogen and progesterone), injected, or implanted hormonal methods of contraception or placement of an intrauterine device (IUD) or intrauterine system (IUS), or other forms of hormonal contraception that have comparable efficacy (failure rate \< 1%), for example hormone vaginal ring or transdermal hormone contraception.

In case of use of oral contraception women should have been stable on the same pill for a minimum of 3 months before taking study treatment.

Women are considered post-menopausal if they have had 12 months of natural (spontaneous) amenorrhea with an appropriate clinical profile (e.g. age appropriate history of vasomotor symptoms). Women are considered not of child bearing potential if they are post-menopausal or have had surgical bilateral oophorectomy (with or without hysterectomy), total hysterectomy or bilateral tubal ligation at least six weeks ago. In the case of oophorectomy alone, only when the reproductive status of the woman has been confirmed by follow-up hormone level assessments and she is considered not of child bearing potential.

If local regulations deviate from the contraception methods listed above to prevent pregnancy, local regulations apply and will be described in the local ICF.
Minimum Eligible Age

18 Years

Maximum Eligible Age

99 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

Novartis Pharmaceuticals

INDUSTRY

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Responsibility Role SPONSOR

Principal Investigators

Learn about the lead researchers overseeing the trial and their institutional affiliations.

Novartis Pharmaceuticals

Role: STUDY_DIRECTOR

Novartis Pharmaceuticals

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

Novartis Investigative Site

Burgas, Bulgaria, Bulgaria

Site Status

Novartis Investigative Site

Pleven, Bulgaria, Bulgaria

Site Status

Novartis Investigative Site

Sofia, Bulgaria, Bulgaria

Site Status

Novartis Investigative Site

Burgas, Burgas, Bulgaria

Site Status

Novartis Investigative Site

Gabrovo, , Bulgaria

Site Status

Novartis Investigative Site

Pleven, , Bulgaria

Site Status

Novartis Investigative Site

Plovdiv, , Bulgaria

Site Status

Novartis Investigative Site

Rousse, , Bulgaria

Site Status

Novartis Investigative Site

Sofia, , Bulgaria

Site Status

Novartis Investigative Site

Sofia, , Bulgaria

Site Status

Novartis Investigative Site

Sofia, , Bulgaria

Site Status

Novartis Investigative Site

Stara Zagora, , Bulgaria

Site Status

Novartis Investigative Site

Varna, , Bulgaria

Site Status

Novartis Investigative Site

Veliko Tarnovo, , Bulgaria

Site Status

Novartis Investigative Site

Chlumec nad Cidlinou, Chlumec Nad Cidlinou, Czechia

Site Status

Novartis Investigative Site

Pardubice, Czech Republic, Czechia

Site Status

Novartis Investigative Site

Trutnov, CZE, Czechia

Site Status

Novartis Investigative Site

Přerov, Olomoucký kraj, Czechia

Site Status

Novartis Investigative Site

Slaný, Slany, Czechia

Site Status

Novartis Investigative Site

Hlučín, , Czechia

Site Status

Novartis Investigative Site

Hodonín, , Czechia

Site Status

Novartis Investigative Site

Mělník, , Czechia

Site Status

Novartis Investigative Site

Olomouc, , Czechia

Site Status

Novartis Investigative Site

Olomouc, , Czechia

Site Status

Novartis Investigative Site

Pärnu, , Estonia

Site Status

Novartis Investigative Site

Tallinn, , Estonia

Site Status

Novartis Investigative Site

Tallinn, , Estonia

Site Status

Novartis Investigative Site

Tartu, , Estonia

Site Status

Novartis Investigative Site

Amiens, , France

Site Status

Novartis Investigative Site

Chambéry, , France

Site Status

Novartis Investigative Site

Le Chesnay, , France

Site Status

Novartis Investigative Site

Le Kremlin-Bicêtre, , France

Site Status

Novartis Investigative Site

Lille, , France

Site Status

Novartis Investigative Site

Marseille, , France

Site Status

Novartis Investigative Site

Nantes, , France

Site Status

Novartis Investigative Site

Paris, , France

Site Status

Novartis Investigative Site

Valenciennes, , France

Site Status

Novartis Investigative Site

Mannheim, Baden-Wurttemberg, Germany

Site Status

Novartis Investigative Site

Lichtenfels, Bavaria, Germany

Site Status

Novartis Investigative Site

Regensburg, Bavaria, Germany

Site Status

Novartis Investigative Site

Hamburg, City state of Hamburg, Germany

Site Status

Novartis Investigative Site

Frankfurt am Main, Hesse, Germany

Site Status

Novartis Investigative Site

Frankfurt am Main, Hesse, Germany

Site Status

Novartis Investigative Site

Frankfurt am Main, Hesse, Germany

Site Status

Novartis Investigative Site

Langen, Hesse, Germany

Site Status

Novartis Investigative Site

Göttingen, Lower Saxony, Germany

Site Status

Novartis Investigative Site

Hanover, Lower Saxony, Germany

Site Status

Novartis Investigative Site

Hanover, Lower Saxony, Germany

Site Status

Novartis Investigative Site

Winsen, Lower Saxony, Germany

Site Status

Novartis Investigative Site

Cologne, North Rhine-Westphalia, Germany

Site Status

Novartis Investigative Site

Löhne, North Rhine-Westphalia, Germany

Site Status

Novartis Investigative Site

Kaiserslautern, Rhineland-Palatinate, Germany

Site Status

Novartis Investigative Site

Bad Gottleuba, Saxony, Germany

Site Status

Novartis Investigative Site

Dresden, Saxony, Germany

Site Status

Novartis Investigative Site

Leipzig, Saxony, Germany

Site Status

Novartis Investigative Site

Schleswig, Schleswig-Holstein, Germany

Site Status

Novartis Investigative Site

Bad Homburg, , Germany

Site Status

Novartis Investigative Site

Bad Krozingen, , Germany

Site Status

Novartis Investigative Site

Bad Oeynhausen, , Germany

Site Status

Novartis Investigative Site

Bamberg, , Germany

Site Status

Novartis Investigative Site

Berlin, , Germany

Site Status

Novartis Investigative Site

Berlin, , Germany

Site Status

Novartis Investigative Site

Berlin, , Germany

Site Status

Novartis Investigative Site

Berlin, , Germany

Site Status

Novartis Investigative Site

Berlin, , Germany

Site Status

Novartis Investigative Site

Berlin, , Germany

Site Status

Novartis Investigative Site

Bochum, , Germany

Site Status

Novartis Investigative Site

Bremen, , Germany

Site Status

Novartis Investigative Site

Coburg, , Germany

Site Status

Novartis Investigative Site

Cologne, , Germany

Site Status

Novartis Investigative Site

Dessau, , Germany

Site Status

Novartis Investigative Site

Dresden, , Germany

Site Status

Novartis Investigative Site

Duisburg, , Germany

Site Status

Novartis Investigative Site

Erfurt, , Germany

Site Status

Novartis Investigative Site

Erfurt, , Germany

Site Status

Novartis Investigative Site

Essen, , Germany

Site Status

Novartis Investigative Site

Essen, , Germany

Site Status

Novartis Investigative Site

Essen, , Germany

Site Status

Novartis Investigative Site

Fulda, , Germany

Site Status

Novartis Investigative Site

Gelnhausen, , Germany

Site Status

Novartis Investigative Site

Gladbeck, , Germany

Site Status

Novartis Investigative Site

Hamburg, , Germany

Site Status

Novartis Investigative Site

Haßloch, , Germany

Site Status

Novartis Investigative Site

Hennigsdorf, , Germany

Site Status

Novartis Investigative Site

Hoyerswerda, , Germany

Site Status

Novartis Investigative Site

Kassel, , Germany

Site Status

Novartis Investigative Site

Kiel, , Germany

Site Status

Novartis Investigative Site

Lüneburg, , Germany

Site Status

Novartis Investigative Site

Magdeburg, , Germany

Site Status

Novartis Investigative Site

Magdeburg, , Germany

Site Status

Novartis Investigative Site

Mainz, , Germany

Site Status

Novartis Investigative Site

Mannheim, , Germany

Site Status

Novartis Investigative Site

Markkleeberg, , Germany

Site Status

Novartis Investigative Site

Mühldorf, , Germany

Site Status

Novartis Investigative Site

München, , Germany

Site Status

Novartis Investigative Site

Münster, , Germany

Site Status

Novartis Investigative Site

Nuremberg, , Germany

Site Status

Novartis Investigative Site

Papenburg, , Germany

Site Status

Novartis Investigative Site

Pirna, , Germany

Site Status

Novartis Investigative Site

Potsdam, , Germany

Site Status

Novartis Investigative Site

Reinfeld, , Germany

Site Status

Novartis Investigative Site

Rostock, , Germany

Site Status

Novartis Investigative Site

Rüdersdorf, , Germany

Site Status

Novartis Investigative Site

Saint Ingbert Oberwuerzbach, , Germany

Site Status

Novartis Investigative Site

Singen, , Germany

Site Status

Novartis Investigative Site

Stuttgart, , Germany

Site Status

Novartis Investigative Site

Stuttgart, , Germany

Site Status

Novartis Investigative Site

Wallerfing, , Germany

Site Status

Novartis Investigative Site

Warendorf, , Germany

Site Status

Novartis Investigative Site

Wuppertal, , Germany

Site Status

Novartis Investigative Site

Daugavpils, , Latvia

Site Status

Novartis Investigative Site

Ogre, , Latvia

Site Status

Novartis Investigative Site

Riga, , Latvia

Site Status

Novartis Investigative Site

Riga, , Latvia

Site Status

Novartis Investigative Site

Riga, , Latvia

Site Status

Novartis Investigative Site

Riga, , Latvia

Site Status

Novartis Investigative Site

Krosno, Krosno, Poland

Site Status

Novartis Investigative Site

Tarnów, Lesser Poland Voivodeship, Poland

Site Status

Novartis Investigative Site

Krakow, Maloposkie, Poland

Site Status

Novartis Investigative Site

Szczecin, West Pomeranian Voivodeship, Poland

Site Status

Novartis Investigative Site

Gdynia, , Poland

Site Status

Novartis Investigative Site

Katowice, , Poland

Site Status

Novartis Investigative Site

Krakow, , Poland

Site Status

Novartis Investigative Site

Rzeszów, , Poland

Site Status

Novartis Investigative Site

Skierniewice, , Poland

Site Status

Novartis Investigative Site

Córdoba, Andalusia, Spain

Site Status

Novartis Investigative Site

Málaga, Andalusia, Spain

Site Status

Novartis Investigative Site

Sanlúcar de Barrameda, Andalusia, Spain

Site Status

Novartis Investigative Site

Seville, Andalusia, Spain

Site Status

Novartis Investigative Site

Seville, Andalusia, Spain

Site Status

Novartis Investigative Site

Barcelona, Catalonia, Spain

Site Status

Novartis Investigative Site

Barcelona, Catalonia, Spain

Site Status

Novartis Investigative Site

San Sebastián de los Reyes, Madrid, Spain

Site Status

Novartis Investigative Site

Oviedo, Principality of Asturias, Spain

Site Status

Novartis Investigative Site

Madrid, , Spain

Site Status

Countries

Review the countries where the study has at least one active or historical site.

Bulgaria Czechia Estonia France Germany Latvia Poland Spain

References

Explore related publications, articles, or registry entries linked to this study.

Landmesser U, Laufs U, Schatz U, Winzer EB, Nowak B, Kassner U, Gouni-Berthold I, Esteban A, Lubyayi L, Krueger A, Streich JH, Hentschke C, Achouba A, Banach M. Design and rationale of the VICTORION-Difference study: A phase 4 randomized, double-blind, placebo-controlled clinical trial to assess inclisiran's early efficacy, safety, tolerability, as well as its impact on quality of life in individuals with hypercholesterolemia. Am Heart J. 2025 Nov;289:117-126. doi: 10.1016/j.ahj.2025.05.014. Epub 2025 May 26.

Reference Type DERIVED
PMID: 40436308 (View on PubMed)

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

2024-511263-28

Identifier Type: OTHER

Identifier Source: secondary_id

CKJX839A12402

Identifier Type: -

Identifier Source: org_study_id

More Related Trials

Additional clinical trials that may be relevant based on similarity analysis.

LATAM LOWERS LDL-C
NCT06501443 RECRUITING PHASE4