Children's National

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

Purpose

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).

Condition

Familial Hypercholesterolemia - Heterozygous

Eligibility

Eligible Ages: Between 6 Years and 11 Years
Eligible Sex: All
Accepts Healthy Volunteers: No

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

Study Design

Phase: Phase 3
Study Type: Interventional
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: Parallel (Year 1) to single-group (Year 2)
Primary Purpose: Treatment
Masking: Double (Participant, Investigator)
Masking Description: Masked (Year 1) to No Masking (Year 2)

Arm Groups

Arm	Description	Assigned Intervention
Experimental Inclisiran	Year 1 - inclisiran sodium subcutaneous injection (given at Days 1, 90, and 270) Day 360 only - placebo subcutaneous injection Year 2 - inclisiran sodium subcutaneous injection (given at Days 450 and 630)	Drug: Inclisiran Inclisiran (inclisiran sodium 300 mg subcutaneous (s.c.) for participants with body weight ≥23 kg and inclisiran sodium 180 mg s.c. for participants with body weight <23 kg. The dose level is based on the participant's body weight on Day 1 (for Part 1) and Day 360 (for Part 2), respectively. Other names: KJX839
Placebo Comparator Placebo	Year 1 - placebo subcutaneous injection (given at Days 1, 90 and 270) Year 2 - inclisiran sodium subcutaneous injection (given at Days 360, 450, and 630)	Drug: Placebo Sterile normal saline (0.9% sodium chloride in water for subcutaneous injection) Other names: saline solution

Recruiting Locations

Children's National and nearby locations

Childrens National Hospital
Washington D.C., District of Columbia 20010

Contact:
Carlos Jesus Carhuas
+1 202 476 5000
ccarhuas@childrensnational.org

More Details

NCT ID: NCT06597019
Status: Recruiting
Sponsor: Novartis Pharmaceuticals

Study Contact

Novartis Pharmaceuticals
1-888-669-6682
novartis.email@novartis.com

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 (aged 6 to <12 years) with heterozygous familial hypercholesterolemia (HeFH) and elevated low density lipoprotein cholesterol (LDL-C) on stable standard of care background lipid-lowering therapy.

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.