ClinicaliQ Trial Snapshot
- Study to Evaluate Safety, Tolerability and Efficacy of Inclisiran in Children With Homozygous Familial Hypercholesterolemia — Recruiting • Phase III • Cardiology / Cardiovascular • NCT06597006.
- Treatment tested: Inclisiran, a PCSK9 inhibitor administered via subcutaneous injection, being evaluated for its ability to lower LDL cholesterol in paediatric patients with homozygous familial hypercholesterolemia (HoFH)—a rare genetic condition causing severely elevated cholesterol levels from birth.
- Patient eligibility: Children aged 2 years and older with confirmed homozygous familial hypercholesterolemia, including those already on background lipid-lowering therapy, representing a traditionally difficult-to-treat paediatric population with extremely high cardiovascular risk.
Use This Page For
- Quick orientation before opening the registry record.
- Checking recruitment status, phase and sponsor at a glance.
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What This Trial Is Studying
This is a pivotal phase III study designed to evaluate safety, tolerability, and efficacy of inclisiran in children (aged 2 to
Eligibility Snapshot
- : * Male or female participants, 2 to 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.
Use the source registry for the full inclusion and exclusion criteria before discussing referral or enrolment.