Study to evaluate efficacy and safety of inclisiran in children aged 2 to under 12 years of age with homozygous familial hypercholesterolemia
Key facts
- Where
- Ioannina és Thessaloniki, GörögországSee the 2 sites
- Phase
- 3. fázis
Where in Görögország
- University General Hospital of IoanninaIoannina, GörögországTaking people
- Ippokratio General Hospital of ThessalonikiThessaloniki, GörögországTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Novartis Pharma AG
- Trial code
- EU 2024-514595-41-00
- Record updated
- 2026. október 26.
- Original description
Primary objective is to evaluate the effect of inclisiran compared to placebo on reducing LDL-C [percent change] at Day 330 (Year 1) in children (aged 2 to <12 years) with HoFH and elevated LDL-CRead full original descriptionShow less
Two part (double-blind inclisiran versus placebo [Year 1] followed by open-label inclisiran [Year 2]) randomized multicenter study to evaluate safety, tolerability and efficacy of inclisiran in children (2 to less than 12 years) with homozygous familial hypercholesterolemia and elevated LDL-cholesterol (ORION-19)
Primary objective is to evaluate the effect of inclisiran compared to placebo on reducing LDL-C [percent change] at Day 330 (Year 1) in children (aged 2 to <12 years) with HoFH and elevated LDL-C
Inclusion: 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; 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 (≥90 days for evinacumab) 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: Previous treatment (within 90 days of screening) with monoclonal antibodies directed towards PCSK9; Treatment with mipomersen or lomitapide (within 5 months of screening); History of poor response to therapy with any monoclonal antibody directed towards PCSK9 (e.g. <15% reduction in LDL-C); Secondary hypercholesterolemia, e.g. hypothyroidism or nephrotic syndrome; Active liver disease defined as any known current infectious, neoplastic, or metabolic pathology of the liver or unexplained alanine aminotransferase (ALT), aspartate aminotransferase (AST) elevation >3x ULN, or total bilirubin elevation >2x ULN (except patients with Gilbert’s syndrome); Body weight (at the screening and/or randomization (Day 1) visit) <16 kg for participants 6 to <12 years (at screening) or <11 kg for participants 2 to <6 years (at screening); Documented evidence of a null (negative) mutation in both LDLR alleles; Recent and/or planned use of other investigational medicinal products or devices; Heterozygous familial hypercholesterolemia (HeFH); Pregnant or nursing females.