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Study to Evaluate Efficacy an...

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

Last Updated: 2026-01-13

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

141 participants

Study Classification

INTERVENTIONAL

Study Start Date

2021-01-27

Study Completion Date

2024-11-27

Brief Summary

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This was a pivotal phase III study designed to evaluate safety, tolerability, and efficacy of inclisiran in adolescents with heterozygous familial hypercholesterolemia (HeFH) and elevated low density lipoprotein cholesterol (LDL-C).

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Detailed Description

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This was 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 adolescents with heterozygous familial hypercholesterolemia (HeFH) and elevated low density lipoprotein cholesterol (LDL-C) on stable standard of care background lipid-lowering therapy. The primary objective was to demonstrate superiority of inclisiran compared to placebo in reducing LDL-C (percent change) at Day 330.

Conditions

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Familial Hypercholesterolemia - Heterozygous

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Parallel (Year 1) to single-group (Year 2)

Primary Study Purpose

TREATMENT

Blinding Strategy

DOUBLE

Participants Investigators

Masked (Year 1) to No Masking (Year 2)

Study Groups

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Inclisiran

Year 1 - inclisiran sodium 300 mg subcutaneous injection (given at Days 1, 90, and 270) Day 360 only - placebo subcutaneous injection Year 2 - inclisiran sodium 300 mg subcutaneous injection (given at Days 450 and 630)

Group Type EXPERIMENTAL

Inclisiran

Intervention Type DRUG

Inclisiran sodium 300 mg (equivalent to 284 mg inclisiran) in 1.5 mL solution for subcutaneous injection

Placebo

Year 1 - placebo subcutaneous injection (given at Days 1, 90 and 270) Year 2 - inclisiran sodium 300 mg subcutaneous injection (given at Days 360, 450, and 630)

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Sterile normal saline (0.9% sodium chloride in water for subcutaneous injection)

Interventions

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Inclisiran

Inclisiran sodium 300 mg (equivalent to 284 mg inclisiran) in 1.5 mL solution for subcutaneous injection

Intervention Type DRUG

Placebo

Sterile normal saline (0.9% sodium chloride in water for subcutaneous injection)

Intervention Type DRUG

Other Intervention Names

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KJX839 Saline solution

Eligibility Criteria

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Inclusion Criteria

* Heterozygous Familial Hypercholesterolemia (HeFH) diagnosed either by genetic testing or on phenotypic criteria
* Fasting LDL-C \>130 mg/dL (3.4 mmol/L) at screening
* Fasting triglycerides \<400 mg/dL (4.5 mmol/L) at screening
* On maximally tolerated dose of statin (investigator's discretion) with or without other lipid-lowering therapy; stable for ≥ 30 days before screening

Exclusion Criteria

* Homozygous familial hypercholesterolemia (HoFH)
* Active liver disease
* Secondary hypercholesterolemia, e.g. hypothyroidism or nephrotic syndrome
* Previous treatment with monoclonal antibodies directed towards PCSK9 (within 90 days of screening)
* Recent and/or planned use of other investigational medicinal products or devices

Minimum Eligible Age

12 Years

Maximum Eligible Age

17 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Novartis Pharmaceuticals

Role: STUDY_DIRECTOR

Novartis Pharmaceuticals

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United States Argentina Brazil Canada Czechia France Germany Greece Hungary Israel Italy Jordan Lebanon Malaysia Netherlands Norway Poland Russia Slovakia Slovenia South Africa Spain Switzerland Taiwan Turkey (Türkiye) United Kingdom