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

ConditionFamilial Hypercholesterolemia - Heterozygous
Trial statusRecruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
Age6-11
SponsorNovartis Pharmaceuticals

About this trial

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).

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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.

Disqualifiers

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

Trial design

Design model

Parallel

Treatments tested in this trial

  • Inclisiran

    Drug

    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.

  • Placebo

    Drug

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

Treatment groups

60 Participants
are divided into 2 treatment groups
Group A: InclisiranExperimental treatment 1 intervention
Group B: PlaceboPlacebo comparator 1 intervention

Trial outcomes

Primary outcomes

1

Percentage change in LDL-C from baseline to Day 330 (Year 1)

Demonstrate superiority of inclisiran compared to placebo in reducing LDL-C \[percent change\] at Day 330

Time frame
Baseline and Day 330

Secondary outcomes

1

Time-adjusted percent change in LDL-C from baseline after Day 90 and up to Day 330 (Year 1)

Demonstrate superiority of inclisiran compared to placebo in reducing LDL-C \[time-adjusted percent change\] over Year 1

Time frame
Baseline, after Day 90 up to Day 330
2

Absolute change in LDL-C from baseline to Day 330 (Year 1)

Demonstrate superiority of inclisiran compared to placebo in reducing LDL-C \[absolute change\] at Day 330 (Year 1)

Time frame
Baseline and Day 330
3

Percent change in PCSK9 from baseline to Day 330 (Year 1)

Demonstrate superiority of inclisiran compared to placebo in reducing PCSK9, total cholesterol, Apo B, and non-HDL-C \[percent change\] at Day 330 (Year 1)

Time frame
Baseline and Day 330
4

Percent change in total cholesterol, non-HDL-C from baseline to Day 330 (Year 1)

Demonstrate superiority of inclisiran compared to placebo in reducing PCSK9, total cholesterol, Apo B, and non-HDL-C \[percent change\] at Day 330 (Year 1)

Time frame
Baseline and Day 330

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Novartis Pharmaceuticals

Lead sponsor

Source ClinicalTrials.gov
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