Study to Evaluate Safety, Tolerability and Efficacy of Inclisiran in Children With Homozyg
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).
| Condition(s) | Familial Hypercholesterolemia - Homozygous |
|---|---|
| Status | Recruiting |
| Phase | Phase 3 |
| Study type | Interventional |
| Summary | 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). |
| Who can participate | Inclusion Criteria: * Male or female participants, 2 to \<12 years of age at screening * HoFH diagnosed by genetic confirmation \- Note: Participants with known null (negative) mutations in both LDLR alleles are not eligible (see also exclusion criteria) * 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 |
| Ages | 2 Years to 11 Years |
| Sex | All |
| Lead sponsor | Novartis Pharmaceuticals |
| Locations | San Francisco, California, United States; San Francisco, California, United States; Washington D.C., District of Columbia, United States; St Louis, Missouri, United States; Vienna, Austria; Beijing, Beijing Municipality, China (+12 more sites) |
| Start date | 2025-02-28 |
| NCT ID | NCT06597006 |
| Official listing | https://clinicaltrials.gov/study/NCT06597006 |