Study to Evaluate Efficacy and Safety of Inclisiran in Adolescents With Heterozygous Familial Hypercholesterolemia

  • STATUS
    Recruiting
  • End date
    Dec 7, 2023
  • participants needed
    150
  • sponsor
    Novartis Pharmaceuticals
Updated on 12 September 2021
Investigator
Novartis Pharmaceuticals
Primary Contact
Novartis Investigative Site (6.9 mi away) Contact
+55 other location

Summary

This is a pivotal phase III study designed to evaluate safety, tolerability, and efficacy of inclisiran in adolescents with heterozygous familial hypercholesterolemia (HeFH) and elevated low density lipoprotein cholesterol (LDL-C).

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 adolescents with heterozygous familial hypercholesterolemia (HeFH) and elevated low density lipoprotein cholesterol (LDL-C) on stable standard of care background lipid-lowering therapy. The primary objective is to demonstrate superiority of inclisiran compared to placebo in reducing LDL-C (percent change) at Day 330.

Details
Condition Heterozygous Familial Hypercholesterolemia
Treatment Placebo, Inclisiran
Clinical Study IdentifierNCT04652726
SponsorNovartis Pharmaceuticals
Last Modified on12 September 2021

Eligibility

Yes No Not Sure

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
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