Study to Evaluate Efficacy and Safety of Inclisiran in Children With Heterozygous Familial Hypercholesterolemia
Recruiting · NCT06597019 · Interventional (participants receive a specific treatment) · Lead sponsor: Novartis Pharmaceuticals
View the official record on ClinicalTrials.gov →Interventions studied
InclisiranPlacebo
What this trial is about
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).
Who can take part
Age range
6 Years to 11 Years
Sex
All (male and female)
Healthy volunteers
No - a diagnosis or condition is required
Phase
Phase 3
Study type
Interventional (participants receive a specific treatment)
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
Where it is running
60 locations listed across 7 US states.
- UC San Francisco Medical Center - San Francisco, California, United States
- UC San Francisco Medical Center - San Francisco, California, United States
- Children's National Hospital - Washington D.C., District of Columbia, United States
- Childrens National Hospital - Washington D.C., District of Columbia, United States
- Excel Medical Clinical Trials LLC - Boca Raton, Florida, United States
- Icahn School of Med at Mt Sinai - New York, New York, United States
- Primary Childrens Medical Center - Salt Lake City, Utah, United States
- Primary Childrens Medical Center - Salt Lake City, Utah, United States
- Virginia Commonwealth University - Richmond, Virginia, United States
- West Virginia Childrens Hospital - Morgantown, West Virginia, United States
- West Virginia Childrens Hospital - Morgantown, West Virginia, United States
- Novartis Investigative Site - Athens, Greece
- Novartis Investigative Site - Barcelona, Spain
- Novartis Investigative Site - Bialystok, Poland
- Novartis Investigative Site - Brussels, Belgium
- Novartis Investigative Site - Budapest, Hungary
- Novartis Investigative Site - Buenos Aires, Argentina
- Novartis Investigative Site - CABA, Argentina
- Novartis Investigative Site - Coimbra, Portugal
- Novartis Investigative Site - Gdansk, Poland
- Novartis Investigative Site - Hanover, Germany
- Novartis Investigative Site - Hong Kong, Hong Kong
- Novartis Investigative Site - Ioannina, Greece
- Novartis Investigative Site - Izmir, Turkey (Türkiye)
- Novartis Investigative Site - Jerusalem, Israel
- Novartis Investigative Site - Leuven, Belgium
- Novartis Investigative Site - Lisbon, Portugal
- Novartis Investigative Site - London, United Kingdom
- Novartis Investigative Site - Marseille, France
- Novartis Investigative Site - Málaga, Spain
- Novartis Investigative Site - Nantes, France
- Novartis Investigative Site - Paris, France
- Novartis Investigative Site - Porto, Portugal
- Novartis Investigative Site - Porto, Portugal
- Novartis Investigative Site - Prague, Czechia
- Novartis Investigative Site - Prague, Czechia
- Novartis Investigative Site - Ramat Gan, Israel
- Novartis Investigative Site - Salzburg, Austria
- Novartis Investigative Site - Seville, Spain
- Novartis Investigative Site - Shanghai, China
- Novartis Investigative Site - Taipei, Taiwan
- Novartis Investigative Site - Taipei, Taiwan
- Novartis Investigative Site - Vienna, Austria
- Novartis Investigative Site - Elche, Alicante, Spain
- Novartis Investigative Site - Cadiz, Andalusia, Spain
- Novartis Investigative Site - Freiburg im Breisgau, Baden-Wurttemberg, Germany
- Novartis Investigative Site - Badalona, Barcelona, Spain
- Novartis Investigative Site - Esplugues, Barcelona, Spain
- Novartis Investigative Site - Beijing, Beijing Municipality, China
- Novartis Investigative Site - West Midlands, Birmingham, United Kingdom
- Novartis Investigative Site - Fortaleza, Ceará, Brazil
- Novartis Investigative Site - Bloemfontein, Free State, South Africa
- Novartis Investigative Site - Frankfurt am Main, Hesse, Germany
- Novartis Investigative Site - Kuala Lumpur, Kuala Lumpur, Malaysia
- Novartis Investigative Site - Milan, MI, Italy
- Novartis Investigative Site - Pamplona, Navarre, Spain
- Novartis Investigative Site - Amsterdam, North Holland, Netherlands
- Novartis Investigative Site - Roma, RM, Italy
- Novartis Investigative Site - Porto Alegre, Rio Grande do Sul, Brazil
- Novartis Investigative Site - Rio de Janeiro, Rio de Janeiro, Brazil
Showing 60 of 66 listed sites. See the official record for the complete list.
Read the full protocol, contacts and eligibility on ClinicalTrials.gov →