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RecruitingPhase 3

Study to Evaluate Efficacy and Safety of Inclisiran in Children With Heterozygous 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 (6 to Less Than 12 Years) With Heterozygous 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
6 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 well inclisiran works and how safe it is in children ages 6 to 11 with high LDL, or “bad” cholesterol. It is for children with heterozygous familial hypercholesterolemia (HeFH), a condition passed down in families that causes high cholesterol.

This is a pivotal phase III study designed to evaluate safety, tolerability, and efficacy of inclisiran in children (aged 6 to \<12 years) with heterozygous familial hypercholesterolemia (HeFH) 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

  • Be 6 to 11 years old at the screening visit.
  • Have HeFH confirmed by a gene test or signs of the condition.
  • Have LDL cholesterol above 130 mg/dL (3.4 mmol/L) on a blood test taken after fasting (not eating).
  • Children ages 8 to 11 must take a statin at a dose the study doctor thinks is best, unless they cannot tolerate statins. Statins lower cholesterol. Other cholesterol drugs may also be used.
  • For children under 8, the study doctor decides whether cholesterol medicine is needed.
  • If taking cholesterol medicine, have no dose changes for at least 30 days before screening and no planned medicine or dose changes during the study.

Who can't join

  • Have used PCSK9 antibody medicines, a type of cholesterol treatment, within 90 days before screening.
  • Have high cholesterol caused by another condition, such as an underactive thyroid or a kidney condition called nephrotic syndrome.
  • Have homozygous familial hypercholesterolemia (HoFH), a different inherited form of high cholesterol.
  • Weigh less than 16 kg at screening or the Day 1 visit.
  • Have current liver disease, unexplained liver enzyme blood tests (ALT or AST) above 3 times the normal limit, or bilirubin above 2 times the normal limit. The bilirubin rule has an exception for Gilbert’s syndrome.
  • Are pregnant or breastfeeding.
  • Have recently used, or plan to use, other medicines or devices being tested in research.

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

Official eligibility criteria

Inclusion Criteria: * Male or female participants, 6 to \<12 years of age at screening * HeFH diagnosed either by genetic testing or on phenotypic criteria * Fasting LDL-C \>130 mg/dL (3.4 mmol/L) at screening * For participants 8 to \<12 years, on an optimal dose of statin (investigator's discretion) unless statin intolerant, with or without other lipid-lowering therapy (e.g. ezetimibe). For participants \<8 years, the use of background lipid-lowering treatment is based on investigator's discretion. * Participants on lipid-lowering therapies (such as statin and/or e.g. ezetimibe) must be on a stable dose for ≥30 days before screening with no planned medication or dose changes during study participation. Exclusion Criteria: * Previous treatment (within 90 days of screening) with monoclonal antibodies directed towards PCSK9 * Secondary hypercholesterolemia, e.g. hypothyroidism or nephrotic syndrome * Homozygous familial hypercholesterolemia (HoFH) * Body weight \<16 kg at the screening and/or randomization (Day 1) visit * 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 (11)

CA

DC

FL

NY

UT

VA

WV