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NCT06597006
Study to Evaluate Safety, Tolerability and Efficacy of Inclisiran in Children With Homozygous Familial Hypercholesterolemia
Conditions: Familial Hypercholesterolemia - Homozygous
Sex: All
Ages: 2 Years – 11 Years
Healthy volunteers: No
Phase: PHASE3
Enrollment: 9
Sponsor: Novartis Pharmaceuticals
Location: UC San Francisco Medical Center San Francisco California
Summary
This is a pivotal phase III study designed to evaluate safety, tolerability, and efficacy of inclisiran in children (aged 2 to \
Eligibility Criteria
Inclusion Criteria:
* 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.
Exclusion Criteria:
* Documented evidence of a null (negative) mutation in both LDLR alleles
* Previous treatment (within 90 days of screening) with monoclonal antibodies directed towards PCSK9
* History of poor response to therapy with any monoclonal antibody directed towards PCSK9 (e.g. \
Source: ClinicalTrials.gov (NCT06597006). StuddyBuddy aggregates publicly available trial information.