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

Launched by NOVARTIS PHARMACEUTICALS · Sep 11, 2024

Keywords

ClinConnect Summary

This clinical trial is studying a medication called inclisiran to see how safe and effective it is for children aged 6 to under 12 years who have a condition known as heterozygous familial hypercholesterolemia (HeFH). This condition causes high levels of a type of cholesterol called low-density lipoprotein cholesterol (LDL-C), which can lead to heart problems later in life. The researchers want to find out if inclisiran can help lower these cholesterol levels in children while being safe to use.

To be eligible for the trial, children must be between 6 and 12 years old and have been diagnosed with HeFH, either through a genetic test or by specific health signs. They should also have a fasting cholesterol level above 130 mg/dL. If they are 8 years or older, they should be taking a standard dose of a cholesterol-lowering medication called a statin, unless they cannot tolerate it. Participants will be monitored closely during the study to ensure their safety and to see how well the medication works. Overall, this trial aims to provide more options for managing high cholesterol in young patients.

Gender

ALL

Eligibility criteria

• Inclusion 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.
• Exclusion Criteria:
• • 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