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Study to Evaluate Safety, Tolerability and Efficacy of Inclisiran in Children With Homozygous Familial Hypercholesterolemia

NCT06597006

RecruitingPhase 3

Sponsor: Novartis Pharmaceuticals

Conditions: Familial Hypercholesterolemia - Homozygous

Interventions: Inclisiran, Placebo

Countries: United States, Austria, China, Germany, Greece, Malaysia, Netherlands, South Africa

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).

Eligibility overview

Sex: ALL

Age: 2 Years to 11 Years

Healthy volunteers: No

Study type: INTERVENTIONAL

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

Locations (19)

San Francisco, California, United States
Washington D.C., District of Columbia, United States
St Louis, Missouri, United States
Salt Lake City, Utah, United States
Vienna, Austria
Beijing, Beijing Municipality, China
Frankfurt am Main, Hesse, Germany
Ioannina, Greece
Thessaloniki, Greece
Kota Bharu, Kelantan, Malaysia
Amsterdam, North Holland, Netherlands
Bloemfontein, Free State, South Africa
Johannesburg, Gauteng, South Africa
Taichung, Taiwan
Taipei, Taiwan
Adana, Saricam, Turkey (Türkiye)
Ankara, Yenimahalle, Turkey (Türkiye)
Izmir, Turkey (Türkiye)
Southampton, United Kingdom