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

Description

This is a pivotal phase III study designed to evaluate safety, tolerability, and efficacy of inclisiran in children (aged 6 to \<12 years) with heterozygous familial hypercholesterolemia (HeFH) and elevated low density lipoprotein cholesterol (LDLC).

Conditions

Familial Hypercholesterolemia - Heterozygous

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Study Overview

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Study Details

Study overview

This is a pivotal phase III study designed to evaluate safety, tolerability, and efficacy of inclisiran in children (aged 6 to \<12 years) with heterozygous familial hypercholesterolemia (HeFH) and elevated low density lipoprotein cholesterol (LDLC).

Two Part (Double-blind Inclisiran Versus Placebo [Year 1] Followed by Open-label Inclisiran [Year 2]) Randomized Multicenter Study to Evaluate Safety, Tolerability and Efficacy of Inclisiran in Children (6 to Less Than 12 Years) With Heterozygous Familial Hypercholesterolemia and Elevated LDL- Cholesterol

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

Condition
Familial Hypercholesterolemia - Heterozygous
Intervention / Treatment

-

Contacts and Locations

Boca Raton

Excel Medical Clinical Trials LLC, Boca Raton, Florida, United States, 33434

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility Criteria

  • * Male or female participants, 6 to \<12 years of age at screening
  • * HeFH diagnosed either by genetic testing or on phenotypic criteria
  • * Fasting LDL-C \>130 mg/dL (3.4 mmol/L) at screening
  • * For participants 8 to \<12 years, on an optimal dose of statin (investigator's discretion) unless statin intolerant, with or without other lipid-lowering therapy (e.g. ezetimibe). For participants \<8 years, the use of background lipid-lowering treatment is based on investigator's discretion.
  • * Participants on lipid-lowering therapies (such as statin and/or e.g. ezetimibe) must be on a stable dose for ≥30 days before screening with no planned medication or dose changes during study participation.
  • * Previous treatment (within 90 days of screening) with monoclonal antibodies directed towards PCSK9
  • * Secondary hypercholesterolemia, e.g. hypothyroidism or nephrotic syndrome
  • * Homozygous familial hypercholesterolemia (HoFH)
  • * Body weight \<16 kg at the screening and/or randomization (Day 1) visit
  • * Active liver disease defined as any known current infectious, neoplastic, or metabolic pathology of the liver or unexplained alanine aminotransferase (ALT), aspartate aminotransferase (AST) elevation \>3x ULN, or total bilirubin elevation \>2x ULN (except patients with Gilbert's syndrome)
  • * Pregnant or nursing females
  • * Recent and/or planned use of other investigational medicinal products or devices

Ages Eligible for Study

6 Years to 11 Years

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Novartis Pharmaceuticals,

Novartis Pharmaceuticals, STUDY_DIRECTOR, Novartis Pharmaceuticals

Study Record Dates

2029-01-14