Status:
RECRUITING
Study to Evaluate Efficacy and Safety of Inclisiran in Children With Heterozygous Familial Hypercholesterolemia
Lead Sponsor:
Novartis Pharmaceuticals
Conditions:
Familial Hypercholesterolemia - Heterozygous
Eligibility:
All Genders
6-11 years
Phase:
PHASE3
Brief Summary
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 el...
Detailed Description
This is a two-part (1 year double-blind inclisiran versus placebo / 1 year open-label inclisiran) multicenter study designed to evaluate safety, tolerability, and efficacy of inclisiran in children (a...
Eligibility Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
December 9 2024
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
April 15 2029
Estimated Enrollment :
51 Patients enrolled
Trial Details
Trial ID
NCT06597019
Start Date
December 9 2024
End Date
April 15 2029
Last Update
November 21 2025
Active Locations (66)
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1
UC San Francisco Medical Center
San Francisco, California, United States, 94143
2
UC San Francisco Medical Center
San Francisco, California, United States, 94143
3
Children's National Hospital
Washington D.C., District of Columbia, United States, 20010
4
Childrens National Hospital
Washington D.C., District of Columbia, United States, 20010