Study to Evaluate Efficacy and Safety of Inclisiran in Adolescents With Heterozygous Familial Hypercholesterolemia (ORION-16)
Familial Hypercholesterolemia - Heterozygous
About this trial
This is an interventional treatment trial for Familial Hypercholesterolemia - Heterozygous focused on measuring Heterozygous familial hypercholesterolemia (HeFH), LDL-cholesterol (LDL-C), adolescents, pediatric, small interfering ribonucleic acid (siRNA)
Eligibility Criteria
Inclusion Criteria:
- Heterozygous Familial Hypercholesterolemia (HeFH) diagnosed either by genetic testing or on phenotypic criteria
- Fasting LDL-C >130 mg/dL (3.4 mmol/L) at screening
- Fasting triglycerides <400 mg/dL (4.5 mmol/L) at screening
- On maximally tolerated dose of statin (investigator's discretion) with or without other lipid-lowering therapy; stable for ≥ 30 days before screening
- Estimated glomerular filtration rate (eGFR) >30 mL/min/1.73 m2 at screening
Exclusion Criteria:
- Homozygous familial hypercholesterolemia (HoFH)
- Active liver disease
- Secondary hypercholesterolemia, e.g. hypothyroidism or nephrotic syndrome
- Major adverse cardiovascular events within 3 months prior to randomization
- Previous treatment with monoclonal antibodies directed towards PCSK9 (within 90 days of screening)
- Recent and/or planned use of other investigational medicinal products or devices
Other protocol-defined inclusion/exclusion criteria may apply
Sites / Locations
- Tucson Medical Center
- Icahn School of Medicine at Mount Sinai
- Wake Forest U of Health Sciences
- Cincinnati Children's Hospital Medical Center
- Childrens Hospital Pittsburgh of UPMC
- Primary Children's Medical Center
- Novartis Investigative Site
- Unidade de pesquisa clinica - Hospital Universitario Walter Cantidio
- Nucleo de Pesquisa Clinica do Rio Grande do Sul
- Setor de Lípides, Aterosclerose e Biologia
- Heart Institute (InCOr) HCMFUSP
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Universitaetsmedizin Mannheim
- KKIM UK Frankfurt/Main
- Universitaetsklinikum Freiburg
- University General Hospital of Ioannina
- Hippokrateion General Hospital of Athens Greece
- Metropolitan Hospital
- Novartis Investigative Site
- Lipid Research
- Lipids Center Sheba Medical Center, Israel
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Hotel Dieu de France Hospital
- UiTM Sungai Buloh
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Institute of the complex problems of cardiovascular disease
- Novartis Investigative Site
- Institute of Internal Prev. Med.
- Novartis Investigative Site
- University Medical Centre Ljubljana, Div. of Pediatric Dept. of Endocrinology, Diabetes and Metabolic Diseases
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Hospital Reina Sofia
- Hospital Virgen de la Vcitoria
- Hospital Central de Asturias
- Novartis Investigative Site
- Hospital Abente y Lago
- Novartis Investigative Site
- Far Eastern Memorial Hospital
- Taipei Veterans General Hospital
- Novartis Investigative Site
- Novartis Investigative Site
- Gazi University Medical Faculty
- Novartis Investigative Site
- Novartis Investigative Site
Arms of the Study
Arm 1
Arm 2
Experimental
Placebo Comparator
Inclisiran
Placebo
Year 1 - inclisiran sodium 300 mg subcutaneous injection (given at Days 1, 90, and 270) Day 360 only - placebo subcutaneous injection Year 2 - inclisiran sodium 300 mg subcutaneous injection (given at Days 450 and 630)
Year 1 - placebo subcutaneous injection (given at Days 1, 90 and 270) Year 2 - inclisiran sodium 300 mg subcutaneous injection (given at Days 360, 450, and 630)