Study to Evaluate Safety, Tolerability and Efficacy of Inclisiran in Children With Homozygous Familial Hypercholesterolemia

Trial statusRecruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
Age2-11
SponsorNovartis Pharmaceuticals

About this trial

This is a pivotal phase III study designed to evaluate safety, tolerability, and efficacy of inclisiran in children (aged 2 to \<12 years) with homozygous familial hypercholesterolemia (HoFH) and elevated low density lipoprotein cholesterol (LDLC).

Eligibility criteria

Qualifiers

Male or female participants, 2 to <12 years of age at screening

HoFH diagnosed by genetic confirmation

Disqualifiers

Fasting LDL-C >130 mg/dL (3.4 mmol/L) at screening

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 e.g. statins, ezetimibe) must be on a stable dose for ≥30 days before screening with no planned medication or dose changes during study participation

Participants on a documented regimen of LDL-apheresis for ≥ 3 months before screening will be allowed to continue the apheresis during the study, if needed. The apheresis schedule/settings/duration must be stable prior to screening, are not allowed to change during the double-blind period of the trial and must permit that an apheresis coincides with each study visit.

Trial design

Treatments tested in this trial

  • Inclisiran
  • Placebo

Treatment groups

9 Participants
are divided into 2 treatment groups

Locations

18
Austria
Novartis Investigative Site1090, Vienna Austria
China
Novartis Investigative Site100013, BeijingBeijing Municipality, China
Germany
Novartis Investigative Site60590, Frankfurt am MainHesse, Germany
Greece
Novartis Investigative Site455 00, Ioannina Greece

Sponsors and collaborators

Novartis Pharmaceuticals

Lead sponsor