NCT06597006RecruitingPhase 3
Study to Evaluate Safety, Tolerability and Efficacy of Inclisiran in Children With Homozygous Familial Hypercholesterolemia
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
Source: ClinicalTrials.gov · Last updated 24 Jul 2026
Brief summary (registry, in English)
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).
- Status (registry)
- Recruiting
- Phase (registry)
- Phase 3
- Study type
- Interventional
- Enrollment
- 9 (estimated)
- First submitted
- 11 Sept 2024
- Start date
- Feb 2025
- Primary completion date
- Mar 2028
- Lead sponsor
- Novartis Pharmaceuticals
- NCT number
- NCT06597006
- EudraCT number
- 2024-514595-41
What you commit to
- Study type: Interventional · Phase 3
- Planned participants: 9
- From Feb 2025 to the primary completion date, Mar 2028
- 19 sites in 11 countries
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Previous treatment (within 90 days of screening) with monoclonal antibodies directed towards PCSK9
- Treatment with mipomersen or lomitapide (within 5 months of screening)
Other illness
- 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
Lab values
- 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)
Age
- 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)
Other
- Documented evidence of a null (negative) mutation in both LDLR alleles
- Heterozygous familial hypercholesterolemia (HeFH)
- Pregnant or nursing females
- Recent and/or planned use of other investigational medicinal products or devices
Who the study is looking for
- 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 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.
Age in the registry: 2 years to 11 years · Sex: All · Does not accept healthy volunteers
Sites (19)
Sites in registry order. Pick your city on the disease page to sort them by distance.
This page is information, not medical advice. Discuss any trial with your doctor.