Clinical Research Directory
Browse clinical research sites, groups, and studies.
Study to Evaluate Efficacy and Safety of Inclisiran in Children With Heterozygous Familial Hypercholesterolemia
Sponsor: Novartis Pharmaceuticals
Summary
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).
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
Key Details
Gender
All
Age Range
6 Years - 11 Years
Study Type
INTERVENTIONAL
Enrollment
51
Start Date
2024-12-09
Completion Date
2029-04-15
Last Updated
2025-11-21
Healthy Volunteers
No
Interventions
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.
Placebo
Sterile normal saline (0.9% sodium chloride in water for subcutaneous injection)
Locations (66)
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
Buenos Aires, Argentina
Novartis Investigative Site
CABA, Argentina
Novartis Investigative Site
Salzburg, Austria
Novartis Investigative Site
Vienna, Austria
Novartis Investigative Site
Brussels, Belgium
Novartis Investigative Site
Leuven, Belgium
Novartis Investigative Site
Fortaleza, Ceará, Brazil
Novartis Investigative Site
Rio de Janeiro, Rio de Janeiro, Brazil
Novartis Investigative Site
Porto Alegre, Rio Grande do Sul, Brazil
Novartis Investigative Site
São Paulo, São Paulo, Brazil
Novartis Investigative Site
Beijing, Beijing Municipality, China
Novartis Investigative Site
Shanghai, China
Novartis Investigative Site
Prague, Czechia
Novartis Investigative Site
Prague, Czechia
Novartis Investigative Site
Marseille, France
Novartis Investigative Site
Nantes, France
Novartis Investigative Site
Paris, France
Novartis Investigative Site
Freiburg im Breisgau, Baden-Wurttemberg, Germany
Novartis Investigative Site
Frankfurt am Main, Hesse, Germany
Novartis Investigative Site
Hanover, Germany
Novartis Investigative Site
Athens, Greece
Novartis Investigative Site
Ioannina, Greece
Novartis Investigative Site
Hong Kong, Hong Kong
Novartis Investigative Site
Budapest, Hungary
Novartis Investigative Site
Jerusalem, Israel
Novartis Investigative Site
Ramat Gan, Israel
Novartis Investigative Site
Milan, MI, Italy
Novartis Investigative Site
Roma, RM, Italy
Novartis Investigative Site
Torino, TO, Italy
Novartis Investigative Site
Verona, VR, Italy
Novartis Investigative Site
Kuala Lumpur, Kuala Lumpur, Malaysia
Novartis Investigative Site
Amsterdam, North Holland, Netherlands
Novartis Investigative Site
Bialystok, Poland
Novartis Investigative Site
Gdansk, Poland
Novartis Investigative Site
Lodz, Łódź Voivodeship, Poland
Novartis Investigative Site
Coimbra, Portugal
Novartis Investigative Site
Lisbon, Portugal
Novartis Investigative Site
Porto, Portugal
Novartis Investigative Site
Porto, Portugal
Novartis Investigative Site
Bloemfontein, Free State, South Africa
Novartis Investigative Site
Elche, Alicante, Spain
Novartis Investigative Site
Cadiz, Andalusia, Spain
Novartis Investigative Site
Esplugues, Barcelona, Spain
Novartis Investigative Site
Badalona, Catalonia, Spain
Novartis Investigative Site
Pamplona, Navarre, Spain
Novartis Investigative Site
Barcelona, Spain
Novartis Investigative Site
Málaga, Spain
Novartis Investigative Site
Seville, Spain
Novartis Investigative Site
Taipei, Taiwan
Novartis Investigative Site
Taipei, Taiwan
Novartis Investigative Site
Adana, Saricam, Turkey (Türkiye)
Novartis Investigative Site
Ankara, Yenimahalle, Turkey (Türkiye)
Novartis Investigative Site
Izmir, Turkey (Türkiye)
Novartis Investigative Site
West Midlands, Birmingham, United Kingdom
Novartis Investigative Site
London, United Kingdom