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Study to Evaluate Efficacy and Safety of Inclisiran in Children With Heterozygous Familial Hypercholesterolemia
NCT06597019 · Novartis
In plain English
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Official title
Two Part (Double-blind Inclisiran Versus Placebo [Year 1] Followed by Open-label Inclisiran [Year 2]) Randomized Multicenter Study to Evaluate Safety, Tolerability and Efficacy of Inclisiran in Children (6 to Less Than 12 Years) With Heterozygous Familial Hypercholesterolemia and Elevated LDL- Cholesterol
About this study
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.
Eligibility criteria
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
Study design
Enrollment target: 51 participants
Allocation: randomized
Masking: double
Age groups: child
Timeline
Starts: 2024-12-09
Estimated completion: 2029-04-15
Last updated: 2025-11-21
Interventions
Drug: InclisiranDrug: Placebo
Primary outcomes
- • Percentage change in LDL-C from baseline to Day 330 (Year 1) (Baseline and Day 330)
Sponsor
Novartis Pharmaceuticals · industry
Contacts & investigators
ContactNovartis Pharmaceuticals · contact · novartis.email@novartis.com · 1-888-669-6682
ContactNovartis Pharmaceuticals · contact · +41613241111
InvestigatorNovartis Pharmaceuticals · study_director, Novartis Pharmaceuticals
All locations (66)
UC San Francisco Medical CenterRecruiting
San Francisco, California, United States
UC San Francisco Medical CenterRecruiting
San Francisco, California, United States
Children's National HospitalNot Yet Recruiting
Washington D.C., District of Columbia, United States
Childrens National HospitalRecruiting
Washington D.C., District of Columbia, United States
Excel Medical Clinical Trials LLCRecruiting
Boca Raton, Florida, United States
Icahn School of Med at Mt SinaiRecruiting
New York, New York, United States
Primary Childrens Medical CenterNot Yet Recruiting
Salt Lake City, Utah, United States
Primary Childrens Medical CenterRecruiting
Salt Lake City, Utah, United States
Virginia Commonwealth UniversityRecruiting
Richmond, Virginia, United States
West Virginia Childrens HospitalRecruiting
Morgantown, West Virginia, United States
West Virginia Childrens HospitalRecruiting
Morgantown, West Virginia, United States
Novartis Investigative SiteRecruiting
Buenos Aires, Argentina
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CABA, Argentina
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Salzburg, Austria
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Vienna, Austria
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Brussels, Belgium
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Leuven, Belgium
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Fortaleza, Ceará, Brazil
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Rio de Janeiro, Rio de Janeiro, Brazil
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Porto Alegre, Rio Grande do Sul, Brazil
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São Paulo, São Paulo, Brazil
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Beijing, Beijing Municipality, China
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Shanghai, China
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Prague, Czechia
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Prague, Czechia
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Marseille, France
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Nantes, France
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Paris, France
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Freiburg im Breisgau, Baden-Wurttemberg, Germany
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Frankfurt am Main, Hesse, Germany
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Hanover, Germany
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Athens, Greece
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Ioannina, Greece
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Hong Kong, Hong Kong
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Budapest, Hungary
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Jerusalem, Israel
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Ramat Gan, Israel
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Milan, MI, Italy
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Roma, RM, Italy
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Torino, TO, Italy
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Verona, VR, Italy
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Kuala Lumpur, Kuala Lumpur, Malaysia
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Amsterdam, North Holland, Netherlands
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Bialystok, Poland
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Gdansk, Poland
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Lodz, Łódź Voivodeship, Poland
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Coimbra, Portugal
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Lisbon, Portugal
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Porto, Portugal
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Porto, Portugal
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Bloemfontein, Free State, South Africa
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Elche, Alicante, Spain
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Cadiz, Andalusia, Spain
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Esplugues, Barcelona, Spain
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Badalona, Catalonia, Spain
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Pamplona, Navarre, Spain
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Barcelona, Spain
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Málaga, Spain
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Seville, Spain
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Taipei, Taiwan
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Taipei, Taiwan
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Adana, Saricam, Turkey (Türkiye)
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Ankara, Yenimahalle, Turkey (Türkiye)
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Izmir, Turkey (Türkiye)
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West Midlands, Birmingham, United Kingdom
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London, United Kingdom