Study to Evaluate Safety, Tolerability and Efficacy of Inclisiran in Children With Homozygous Familial Hypercholesterolemia
Start Date
2/28/2025
Completion Date
4/15/2029
Summary
This is a pivotal phase III study designed to evaluate safety, tolerability, and efficacy of inclisiran in children (aged 2 to \<12 years) with homozygous familial hypercholesterolemia (HoFH) and elevated low density lipoprotein cholesterol (LDLC).
Detailed Description
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 2 to \<12 years) with homozygous familial hypercholesterolemia (HoFH) and elevated low density lipoprotein cholesterol (LDL-C) on stable standard of care background lipid-lowering therapy.
Eligibility Criteria
Age Range: 2 years to 11 years
Interventions
Inclisiran
Placebo
Conditions
Locations
UC San Francisco Medical Center
San Francisco, California 94143-0348
United States
Childrens National Hospital
Washington D.C., District of Columbia 20010
United States
Washington Univ School Of Medicine
St Louis, Missouri 63110
United States
Primary Childrens Medical Center
Salt Lake City, Utah 84113
United States
Novartis Investigative Site
Vienna, 1090
Austria
Novartis Investigative Site
Beijing, Beijing Municipality 100013
China
Novartis Investigative Site
Frankfurt am Main, Hesse 60590
Germany
Novartis Investigative Site
Ioannina, 455 00
Greece
Novartis Investigative Site
Thessaloniki, 546 42
Greece
Novartis Investigative Site
Kota Bharu, Kelantan 16150
Malaysia
Novartis Investigative Site
Amsterdam, North Holland 1105 AZ
Netherlands
Novartis Investigative Site
Bloemfontein, Free State 9301
South Africa
Novartis Investigative Site
Johannesburg, Gauteng 2193
South Africa
Novartis Investigative Site
Taichung, 407219
Taiwan
Novartis Investigative Site
Taipei, 111045
Taiwan
Novartis Investigative Site
Adana, Saricam 01330
Turkey (Türkiye)
Novartis Investigative Site
Ankara, Yenimahalle 06500
Turkey (Türkiye)
Novartis Investigative Site
Izmir, 35100
Turkey (Türkiye)
Novartis Investigative Site
Southampton, SO16 6YD
United Kingdom