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

Description

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).

Conditions

Familial Hypercholesterolemia - Homozygous

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Study Overview

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Study Details

Study overview

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).

Two Part (Double-blind Inclisiran Versus Placebo [Year 1] Followed by Open-label Inclisiran [Year 2]) Randomized Multicenter Study to Evaluate Safety, Tolerability, and Efficacy of Inclisiran in Children (2 to Less Than 12 Years) With Homozygous Familial Hypercholesterolemia and Elevated LDL-cholesterol

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

Condition
Familial Hypercholesterolemia - Homozygous
Intervention / Treatment

-

Contacts and Locations

San Francisco

UC San Francisco Medical Center, San Francisco, California, United States, 94158

Washington

Childrens National Medical Center, Washington, District of Columbia, United States, 20010

Saint Louis

Washington Univ School Of Medicine, Saint Louis, Missouri, United States, 63110

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility Criteria

  • * Male or female participants, 2 to \<12 years of age at screening
  • * HoFH diagnosed by genetic confirmation
  • * 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.
  • * 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

Ages Eligible for Study

2 Years to 11 Years

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Novartis Pharmaceuticals,

Novartis Pharmaceuticals, STUDY_DIRECTOR, Novartis Pharmaceuticals

Study Record Dates

2029-04-15