Boca Raton,
Florida
33434
Purpose:
This is a pivotal phase III study designed to evaluate safety, tolerability, and efficacy of
inclisiran in adolescents with homozygous familial hypercholesterolemia (HoFH) and elevated
low density lipoprotein cholesterol (LDL-C).
Study summary:
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 adolescents with homozygous familial hypercholesterolemia (HoFH) and elevated
low density lipoprotein cholesterol (LDL-C) on stable standard of care background
lipid-lowering therapy. The primary objective is to evaluate the effect of inclisiran
compared to placebo in reducing LDL-C (percent change) at Day 330.
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