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Study to Evaluate Efficacy and Safety of Inclisiran in Children With Heterozygous Familial Hypercholesterolemia
Study to Evaluate Efficacy and Safety of Inclisiran in Children With Heterozygous Familial Hypercholesterolemia

NCT06597019

RecruitingPhase 3

Sponsor: Novartis Pharmaceuticals

Conditions: Familial Hypercholesterolemia - Heterozygous

Interventions: Inclisiran, Placebo

Countries: United States, Argentina, Austria, Belgium, Brazil, China, Czechia, France

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).

Eligibility overview

Sex: ALL

Age: 6 Years to 11 Years

Healthy volunteers: No

Study type: INTERVENTIONAL

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
Locations (66)
  • San Francisco, California, United States
  • San Francisco, California, United States
  • Washington D.C., District of Columbia, United States
  • Washington D.C., District of Columbia, United States
  • Boca Raton, Florida, United States
  • New York, New York, United States
  • Salt Lake City, Utah, United States
  • Salt Lake City, Utah, United States
  • Richmond, Virginia, United States
  • Morgantown, West Virginia, United States
  • Morgantown, West Virginia, United States
  • Buenos Aires, Argentina
  • CABA, Argentina
  • Salzburg, Austria
  • Vienna, Austria
  • Brussels, Belgium
  • Leuven, Belgium
  • Fortaleza, Ceará, Brazil
  • Rio de Janeiro, Rio de Janeiro, Brazil
  • Porto Alegre, Rio Grande do Sul, Brazil
  • São Paulo, São Paulo, Brazil
  • Beijing, Beijing Municipality, China
  • Shanghai, China
  • Prague, Czechia
  • Prague, Czechia
  • Marseille, France
  • Nantes, France
  • Paris, France
  • Freiburg im Breisgau, Baden-Wurttemberg, Germany
  • Frankfurt am Main, Hesse, Germany
  • + 36 more on CT.gov