Study to Evaluate Efficacy and Safety of Inclisiran in Adolescents With Homozygous Familial Hypercholesterolemia (ORION-13)
Familial Hypercholesterolemia - Homozygous
About this trial
This is an interventional treatment trial for Familial Hypercholesterolemia - Homozygous focused on measuring Homozygous familial hypercholesterolemia (HoFH), LDL-cholesterol (LDL-C), adolescents, pediatric, small interfering ribonucleic acid (siRNA)
Eligibility Criteria
Inclusion Criteria:
- Homozygous Familial Hypercholesterolemia (HoFH) diagnosed by genetic confirmation
- Fasting LDL-C >130 mg/dL (3.4 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:
- Documented evidence of a null (negative) mutation in both LDLR alleles
- Heterozygous familial hypercholesterolemia (HeFH)
- Active liver disease
- Secondary hypercholesterolemia, e.g. hypothyroidism or nephrotic syndrome
- Major adverse cardiovascular events within 1 month prior to randomization
- Previous treatment with monoclonal antibodies directed towards PCSK9 (within 90 days of screening)
- Treatment with mipomersen or lomitapide (within 5 months of screening)
- Recent and/or planned use of other investigational medicinal products or devices
Other protocol-defined inclusion/exclusion criteria may apply
Sites / Locations
- Excel Medical Clinical Trials LLC
- Novartis Investigative Site
- Novartis Investigative Site
- University General Hospital of Ioannina
- Metropolitan Hospital
- Hotel Dieu de France Hospital
- American University of Beirut Medical Center
- Novartis Investigative Site
- Novartis Investigative Site
- 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)