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

Study Results

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

13 participants

Study Classification

INTERVENTIONAL

Study Start Date

2021-02-16

Study Completion Date

2024-11-18

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 homozygous familial hypercholesterolemia (HoFH) and elevated low density lipoprotein cholesterol (LDL-C).

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Detailed Description

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This was a two-part (double-blind, inclisiran versus placebo \[Year 1\] followed by open-label inclisiran \[Year 2\]) multicenter study in adolescents (aged 12 to \< 18 years) with HoFH and elevated LDL-C (\> 130 mg/dL; 3.4 mmol/L) on stable, individualized, optimal standard of care (SoC) background lipid-lowering therapy (including maximally tolerated statin treatment, at the Investigator's discretion) to evaluate the safety, tolerability, and efficacy of inclisiran in this pediatric patient population.

Following an approximately 4-week screening/run-in period, the study had 2 sequential parts as follows:

Part 1/Year 1: 12 months double-blind, parallel group period, in which participants were randomized in a 2:1 ratio to receive either inclisiran sodium 300 mg subcutaneous (s.c.) or placebo. The primary endpoint was assessed at Day 330.

Part 2/Year 2: 12 months single arm, open-label follow-up period, with all participants receiving inclisiran sodium 300 mg s.c.

Conditions

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Familial Hypercholesterolemia - Homozygous

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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Part 1 - Inclisiran

Inclisiran sodium 300 mg subcutaneous (sc) injection (given at Days 1, 90 and 270)

Group Type EXPERIMENTAL

Inclisiran

Intervention Type DRUG

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

Part 1 - Placebo

Placebo sc injection (given at Day 1, 90 and 270)

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

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

Part 2 - Inclisiran (Total)

Inclisiran sodium 300 mg sc injection (given at Days 450 and 630). In addition, participants assigned to placebo in Part 1 received inclisiran sodium 300 mg sc injection on Day 360, while participants assigned to inclisiran in Part 1 received placebo sc injection on Day 360.

Group Type EXPERIMENTAL

Inclisiran

Intervention Type DRUG

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

Intervention Type DRUG

Other Intervention Names

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KJX839 Saline solution

Eligibility Criteria

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Inclusion Criteria

* Homozygous Familial Hypercholesterolemia (HoFH) diagnosed by genetic confirmation
* Fasting LDL-C \>130 mg/dL (3.4 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
* Male or female participants \>=12 to \<18 years of age at screening

Exclusion Criteria

* Documented evidence of a null (negative) mutation in both LDLR alleles
* Heterozygous familial hypercholesterolemia (HeFH)
* Active liver disease
* Secondary hypercholesterolemia, e.g. hypothyroidism or nephrotic syndrome
* Previous treatment with monoclonal antibodies directed towards PCSK9 (within 90 days of screening)
* Treatment with mipomersen or lomitapide (within 5 months 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

No