Boca Raton, Florida 33434

  • Homozygous

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


NCT ID:

NCT04659863


Primary Contact:

Study Director
Novartis Pharmaceuticals
Novartis Pharmaceuticals


Backup Contact:

N/A


Location Contact:

Boca Raton, Florida 33434
United States



There is no listed contact information for this specific location.

Site Status: N/A


Data Source: ClinicalTrials.gov

Date Processed: January 26, 2023

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