Last updated 13 days ago
Study to Evaluate Efficacy and Safety of Inclisiran in Children With Heterozygous Familial Hypercholesterolemia
51 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
51Patients 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
