Study to Evaluate Efficacy and Safety of Inclisiran in Children With Heterozygous Familial Hypercholesterolemia
Trial statusRecruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
Age6-11
SponsorNovartis Pharmaceuticals
About this trial
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).
Eligibility criteria
Qualifiers
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.
Disqualifiers
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
Trial design
Treatments tested in this trial
- Inclisiran
- Placebo
Treatment groups
60 Participants
are divided into 2 treatment groupsSponsors and collaborators
Source ClinicalTrials.gov