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RecruitingPhase 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

Who can join

As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.

Age
2 Years to 11 Years
Sex
All sexes
Healthy volunteers
Not accepted
Compensation
Not stated

Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.

What to expect

No one can promise a trial will help you personally — that is what the research is trying to find out.

What participation involves

Not stated in the study description — ask the study team about visits and procedures.

Compensation
Not stated
  • Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
  • Participants are monitored closely by the research team throughout the study.
  • Some participants may receive access to an investigational treatment, which is still being studied.
  • Your participation helps researchers learn whether this approach could help future patients.

About this study

In plain language

This study tests how safe inclisiran is, how well children tolerate it, and how well it lowers LDL (“bad”) cholesterol. It is for children ages 2 to under 12 with high LDL cholesterol and HoFH, an inherited condition that causes very high cholesterol.

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).

Compensation is read from the study's own description and may be missing — always confirm with the study team.

Eligibility

Who can join

  • Age 2 to under 12 at screening.
  • HoFH confirmed by a genetic test.
  • LDL cholesterol above 130 mg/dL (3.4 mmol/L) after fasting at screening.
  • Taking the best statin dose chosen by the study doctor, unless unable to tolerate statins. Other cholesterol medicines may also be used.
  • Cholesterol medicine doses must be unchanged for at least 30 days before screening, with no changes planned during the study.
  • Children using LDL apheresis (a treatment that removes LDL from blood) may continue if needed. They must have a documented treatment plan for at least 3 months before screening. The schedule, settings, and length must be stable before screening and stay unchanged during the blinded study period. Treatment must line up with each study visit.

Who can't join

  • Known gene changes that leave both copies of the LDLR gene unable to work.
  • Use of PCSK9 antibody medicines (a type of cholesterol medicine) within 90 days before screening, or a past poor response to them, such as less than a 15% drop in LDL.
  • Use of mipomersen or lomitapide within 5 months before screening.
  • High cholesterol caused by another condition, such as an underactive thyroid or nephrotic syndrome (a kidney disorder), or HeFH, a different inherited form of high cholesterol.
  • Weight below 16 kg for ages 6 to under 12, or below 11 kg for ages 2 to under 6, at screening or the first-day visit.
  • Current liver disease, or unexplained liver blood test results: ALT or AST above 3 times the upper normal limit, or total bilirubin above 2 times that limit. The bilirubin rule has an exception for Gilbert’s syndrome.
  • Pregnant or breastfeeding.
  • Recent or planned use of other experimental medicines or devices.

Simplified for readability. The official criteria below are what the study team uses.

Official eligibility criteria

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 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. \<15% reduction in LDL-C) * Treatment with mipomersen or lomitapide (within 5 months of screening) * Secondary hypercholesterolemia, e.g. hypothyroidism or nephrotic syndrome * Heterozygous familial hypercholesterolemia (HeFH) * 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) * 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) * Pregnant or nursing females * Recent and/or planned use of other investigational medicinal products or devices

Study sites (4)

CA

DC

MO

UT