Evaluating Inclisiran for Children with Familial Hypercholesterolemia

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This pivotal phase III study aims to assess the safety, tolerability, and efficacy of inclisiran in children aged 6 to less than 12 years who have heterozygous familial hypercholesterolemia (HeFH) and elevated LDL cholesterol levels. The study consists of two parts: a one-year double-blind phase comparing inclisiran to a placebo, followed by a one-year open-label phase where all participants receive inclisiran. Participants must be on stable background lipid-lowering therapy, and the study will monitor their LDL-C levels throughout the trial.

Why it matters

Who can join: the full rules

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

Where it's running

• UC San Francisco Medical Center
  San Francisco, California, United States Recruiting
• UC San Francisco Medical Center
  San Francisco, California, United States Recruiting
• Childrens National Hospital
  Washington D.C., District of Columbia, United States Recruiting
• Excel Medical Clinical Trials LLC
  Boca Raton, Florida, United States Recruiting
• Icahn School of Med at Mt Sinai
  New York, New York, United States Recruiting
• Primary Childrens Medical Center
  Salt Lake City, Utah, United States Recruiting

+16 more sites. See ClinicalTrials.gov for the full list.

Study contacts

• Study coordinator: Novartis Pharmaceuticals
• Email: novartis.email@novartis.com
• Phone: 1-888-669-6682

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.