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.