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Study to Evaluate Efficacy and Safety of Inclisiran in Children With Heterozygous Familial Hypercholesterolemia

NCT ID: NCT06597019

Last Updated: 2025-11-13

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

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Recruitment Status

RECRUITING

Clinical Phase

PHASE3

Total Enrollment

51 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-12-09

Study Completion Date

2029-04-15

Brief Summary

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This is a pivotal phase III study designed to evaluate safety, tolerability, and efficacy of inclisiran in children (aged 6 to \<12 years) with heterozygous familial hypercholesterolemia (HeFH) and elevated low density lipoprotein cholesterol (LDLC).

Detailed Description

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This is a two-part (1 year double-blind inclisiran versus placebo / 1 year open-label inclisiran) multicenter study designed to evaluate safety, tolerability, and efficacy of inclisiran in children (aged 6 to \<12 years) with heterozygous familial hypercholesterolemia (HeFH) and elevated low density lipoprotein cholesterol (LDL-C) on stable standard of care background lipid-lowering therapy.

Conditions

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Familial Hypercholesterolemia - Heterozygous

Keywords

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Heterozygous familial hypercholesterolemia (HeFH), LDL-cholesterol (LDL-C), Children, pediatric, small interfering ribonucleic acid (siRNA), inclisiran, Familial Hypercholesterolemia, Heterozygous FH, Hypercholesterolemia, Lipoprotein(a), Hyperlipidemia, Dyslipidemia, Heart Failure, Cardiovascular Diseases, Cholesterol, Aortic Stenosis

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Parallel (Year 1) to single-group (Year 2)
Primary Study Purpose

TREATMENT

Blinding Strategy

DOUBLE

Participants Investigators
Masked (Year 1) to No Masking (Year 2)

Study Groups

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Inclisiran

Year 1 - inclisiran sodium subcutaneous injection (given at Days 1, 90, and 270) Day 360 only - placebo subcutaneous injection Year 2 - inclisiran sodium subcutaneous injection (given at Days 450 and 630)

Group Type EXPERIMENTAL

Inclisiran

Intervention Type DRUG

Inclisiran (inclisiran sodium 300 mg subcutaneous (s.c.) for participants with body weight ≥23 kg and inclisiran sodium 180 mg s.c. for participants with body weight \<23 kg. The dose level is based on the participant's body weight on Day 1 (for Part 1) and Day 360 (for Part 2), respectively.

Placebo

Year 1 - placebo subcutaneous injection (given at Days 1, 90 and 270) Year 2 - inclisiran sodium subcutaneous injection (given at Days 360, 450, and 630)

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Sterile normal saline (0.9% sodium chloride in water for subcutaneous injection)

Interventions

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Inclisiran

Inclisiran (inclisiran sodium 300 mg subcutaneous (s.c.) for participants with body weight ≥23 kg and inclisiran sodium 180 mg s.c. for participants with body weight \<23 kg. The dose level is based on the participant's body weight on Day 1 (for Part 1) and Day 360 (for Part 2), respectively.

Intervention Type DRUG

Placebo

Sterile normal saline (0.9% sodium chloride in water for subcutaneous injection)

Intervention Type DRUG

Other Intervention Names

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KJX839 saline solution

Eligibility Criteria

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Inclusion Criteria

* Male or female participants, 6 to \<12 years of age at screening
* HeFH diagnosed either by genetic testing or on phenotypic criteria
* Fasting LDL-C \>130 mg/dL (3.4 mmol/L) at screening
* For participants 8 to \<12 years, on an optimal dose of statin (investigator's discretion) unless statin intolerant, with or without other lipid-lowering therapy (e.g. ezetimibe). For participants \<8 years, the use of background lipid-lowering treatment is based on investigator's discretion.
* Participants on lipid-lowering therapies (such as statin and/or e.g. ezetimibe) must be on a stable dose for ≥30 days before screening with no planned medication or dose changes during study participation.

Exclusion Criteria

* Previous treatment (within 90 days of screening) with monoclonal antibodies directed towards PCSK9
* Secondary hypercholesterolemia, e.g. hypothyroidism or nephrotic syndrome
* Homozygous familial hypercholesterolemia (HoFH)
* Body weight \<16 kg at the screening and/or randomization (Day 1) visit
* Active liver disease defined as any known current infectious, neoplastic, or metabolic pathology of the liver or unexplained alanine aminotransferase (ALT), aspartate aminotransferase (AST) elevation \>3x ULN, or total bilirubin elevation \>2x ULN (except patients with Gilbert's syndrome)
* Pregnant or nursing females
* Recent and/or planned use of other investigational medicinal products or devices
Minimum Eligible Age

6 Years

Maximum Eligible Age

11 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Novartis Pharmaceuticals

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Novartis Pharmaceuticals

Role: STUDY_DIRECTOR

Novartis Pharmaceuticals

Locations

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UC San Francisco Medical Center

San Francisco, California, United States

Site Status RECRUITING

UC San Francisco Medical Center

San Francisco, California, United States

Site Status RECRUITING

Children's National Hospital

Washington D.C., District of Columbia, United States

Site Status NOT_YET_RECRUITING

Childrens National Hospital

Washington D.C., District of Columbia, United States

Site Status RECRUITING

Excel Medical Clinical Trials LLC

Boca Raton, Florida, United States

Site Status RECRUITING

Icahn School of Med at Mt Sinai

New York, New York, United States

Site Status RECRUITING

Primary Childrens Medical Center

Salt Lake City, Utah, United States

Site Status NOT_YET_RECRUITING

Primary Childrens Medical Center

Salt Lake City, Utah, United States

Site Status RECRUITING

Virginia Commonwealth University

Richmond, Virginia, United States

Site Status RECRUITING

West Virginia Childrens Hospital

Morgantown, West Virginia, United States

Site Status RECRUITING

West Virginia Childrens Hospital

Morgantown, West Virginia, United States

Site Status RECRUITING

Novartis Investigative Site

Buenos Aires, , Argentina

Site Status RECRUITING

Novartis Investigative Site

CABA, , Argentina

Site Status RECRUITING

Novartis Investigative Site

Salzburg, , Austria

Site Status RECRUITING

Novartis Investigative Site

Vienna, , Austria

Site Status RECRUITING

Novartis Investigative Site

Brussels, , Belgium

Site Status RECRUITING

Novartis Investigative Site

Leuven, , Belgium

Site Status RECRUITING

Novartis Investigative Site

Fortaleza, Ceará, Brazil

Site Status RECRUITING

Novartis Investigative Site

Rio de Janeiro, Rio de Janeiro, Brazil

Site Status RECRUITING

Novartis Investigative Site

Porto Alegre, Rio Grande do Sul, Brazil

Site Status RECRUITING

Novartis Investigative Site

São Paulo, São Paulo, Brazil

Site Status RECRUITING

Novartis Investigative Site

Beijing, Beijing Municipality, China

Site Status RECRUITING

Novartis Investigative Site

Shanghai, , China

Site Status RECRUITING

Novartis Investigative Site

Prague, , Czechia

Site Status RECRUITING

Novartis Investigative Site

Prague, , Czechia

Site Status RECRUITING

Novartis Investigative Site

Marseille, , France

Site Status RECRUITING

Novartis Investigative Site

Nantes, , France

Site Status RECRUITING

Novartis Investigative Site

Paris, , France

Site Status RECRUITING

Novartis Investigative Site

Freiburg im Breisgau, Baden-Wurttemberg, Germany

Site Status RECRUITING

Novartis Investigative Site

Hanover, , Germany

Site Status RECRUITING

Novartis Investigative Site

Athens, , Greece

Site Status RECRUITING

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Ioannina, , Greece

Site Status RECRUITING

Novartis Investigative Site

Hong Kong, , Hong Kong

Site Status RECRUITING

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Budapest, , Hungary

Site Status RECRUITING

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Jerusalem, , Israel

Site Status RECRUITING

Novartis Investigative Site

Ramat Gan, , Israel

Site Status RECRUITING

Novartis Investigative Site

Milan, MI, Italy

Site Status RECRUITING

Novartis Investigative Site

Roma, RM, Italy

Site Status RECRUITING

Novartis Investigative Site

Torino, TO, Italy

Site Status RECRUITING

Novartis Investigative Site

Verona, VR, Italy

Site Status RECRUITING

Novartis Investigative Site

Kuala Lumpur, Kuala Lumpur, Malaysia

Site Status RECRUITING

Novartis Investigative Site

Amsterdam, North Holland, Netherlands

Site Status RECRUITING

Novartis Investigative Site

Bialystok, , Poland

Site Status RECRUITING

Novartis Investigative Site

Gdansk, , Poland

Site Status RECRUITING

Novartis Investigative Site

Lodz, Łódź Voivodeship, Poland

Site Status RECRUITING

Novartis Investigative Site

Coimbra, , Portugal

Site Status RECRUITING

Novartis Investigative Site

Lisbon, , Portugal

Site Status RECRUITING

Novartis Investigative Site

Porto, , Portugal

Site Status RECRUITING

Novartis Investigative Site

Porto, , Portugal

Site Status RECRUITING

Novartis Investigative Site

Bloemfontein, Free State, South Africa

Site Status RECRUITING

Novartis Investigative Site

Elche, Alicante, Spain

Site Status RECRUITING

Novartis Investigative Site

Cadiz, Andalusia, Spain

Site Status RECRUITING

Novartis Investigative Site

Esplugues, Barcelona, Spain

Site Status RECRUITING

Novartis Investigative Site

Badalona, Catalonia, Spain

Site Status RECRUITING

Novartis Investigative Site

Pamplona, Navarre, Spain

Site Status RECRUITING

Novartis Investigative Site

Barcelona, , Spain

Site Status RECRUITING

Novartis Investigative Site

Málaga, , Spain

Site Status RECRUITING

Novartis Investigative Site

Seville, , Spain

Site Status RECRUITING

Novartis Investigative Site

Taipei, , Taiwan

Site Status RECRUITING

Novartis Investigative Site

Taipei, , Taiwan

Site Status RECRUITING

Novartis Investigative Site

Adana, Saricam, Turkey (Türkiye)

Site Status RECRUITING

Novartis Investigative Site

Ankara, Yenimahalle, Turkey (Türkiye)

Site Status RECRUITING

Novartis Investigative Site

Izmir, , Turkey (Türkiye)

Site Status RECRUITING

Novartis Investigative Site

West Midlands, Birmingham, United Kingdom

Site Status RECRUITING

Novartis Investigative Site

London, , United Kingdom

Site Status RECRUITING

Countries

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United States Argentina Austria Belgium Brazil China Czechia France Germany Greece Hong Kong Hungary Israel Italy Malaysia Netherlands Poland Portugal South Africa Spain Taiwan Turkey (Türkiye) United Kingdom

Central Contacts

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Novartis Pharmaceuticals

Role: CONTACT

Phone: 1-888-669-6682

Email: [email protected]

Novartis Pharmaceuticals

Role: CONTACT

Phone: +41613241111

Facility Contacts

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Luis Gay

Role: primary

Laura Dapkus Humphries

Role: primary

Carlos Carhuas

Role: primary

Role: primary

Claire Hennum

Role: primary

Maria Morban

Role: primary

Linda Lambert

Role: primary

Role: primary

Megan Beatley

Role: primary

Robin Hoffer

Role: primary

Robin Hoffer

Role: primary

Other Identifiers

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2024-514594-21

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

CKJX839C12303

Identifier Type: -

Identifier Source: org_study_id