Last updated 2 months ago
Study to Evaluate Efficacy and Safety of Inclisiran in Children With Heterozygous Familial Hypercholesterolemia
60 patients around the world
Available in Argentina, United States
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 children (aged 6 to <12 years) with heterozygous familial hypercholesterolemia (HeFH) and elevated low density lipoprotein cholesterol (LDL-C) on stable standard of care background lipid-lowering therapy.
Novartis Pharmaceuticals
60Patients around the world
This study is for people with
Hypercholesterolemia
Familial hypercholesterolemia
Requirements for the patient
To 11 Years
All Gender
Medical requirements
- 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).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.
StudyORION-20
SponsorNovartis Pharmaceuticals
Study typeInterventional
Conditions
Familial hypercholesterolemia
Requirements
To 11 YearsAll Gender
Unique study IDclinicaltrials.gov
NCT06597019
