Status:
RECRUITING
Study to Evaluate Safety, Tolerability and Efficacy of Inclisiran in Children With Homozygous Familial Hypercholesterolemia
Lead Sponsor:
Novartis Pharmaceuticals
Conditions:
Familial Hypercholesterolemia - Homozygous
Eligibility:
All Genders
2-11 years
Phase:
PHASE3
Brief Summary
This is a pivotal phase III study designed to evaluate safety, tolerability, and efficacy of inclisiran in children (aged 2 to \<12 years) with homozygous familial hypercholesterolemia (HoFH) and elev...
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, 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
- 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
Key Trial Info
Start Date :
February 28 2025
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
April 15 2029
Estimated Enrollment :
9 Patients enrolled
Trial Details
Trial ID
NCT06597006
Start Date
February 28 2025
End Date
April 15 2029
Last Update
December 12 2025
Active Locations (17)
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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
Childrens National Hospital
Washington D.C., District of Columbia, United States, 20010
4
Washington Univ School Of Medicine
St Louis, Missouri, United States, 63110