Study to Evaluate Safety, Tolerability and Efficacy of Inclisiran in Children With Homozygous Familial Hypercholesterolemia
Trial statusRecruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
Age2-11
SponsorNovartis Pharmaceuticals
About this trial
This is a pivotal phase III study designed to evaluate safety, tolerability, and efficacy of inclisiran in children (aged 2 to \<12 years) with homozygous familial hypercholesterolemia (HoFH) and elevated low density lipoprotein cholesterol (LDLC).
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Male or female participants, 2 to <12 years of age at screening
HoFH diagnosed by genetic confirmation
Disqualifiers
Fasting LDL-C >130 mg/dL (3.4 mmol/L) at screening
On an optimal dose of statin (investigator's discretion), unless statin intolerant, with or without other lipid-lowering therapy (e.g. ezetimibe)
Participants on lipid-lowering therapies (such as e.g. statins, ezetimibe) must be on a stable dose for ≥30 days before screening with no planned medication or dose changes during study participation
Participants on a documented regimen of LDL-apheresis for ≥ 3 months before screening will be allowed to continue the apheresis during the study, if needed. The apheresis schedule/settings/duration must be stable prior to screening, are not allowed to change during the double-blind period of the trial and must permit that an apheresis coincides with each study visit.
Trial design
Design model
Parallel
Treatments tested in this trial
Inclisiran
DrugInclisiran (inclisiran sodium 300 mg subcutaneous (s.c.) for participants with body weight ≥23 kg, inclisiran sodium 180 mg s.c. for participants with body weight \<23 kg to ≥16 kg, or inclisiran sodium 100 mg s.c. for participants with body weight \<16 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
DrugSterile normal saline (0.9% sodium chloride in water for subcutaneous injection)
Treatment groups
9 Participants
are divided into 2 treatment groupsGroup 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)
Evaluate the effect of inclisiran compared to placebo on reducing LDL-C \[percent change\] at Day 330
Time frame
Baseline and Day 330Secondary outcomes
1
Time-adjusted percent change in LDL-C from baseline after Day 90 and up to Day 330 (Year 1)
Evaluate the effect of inclisiran compared to placebo on reducing LDL-C \[time-adjusted percent change\] over Year 1
Time frame
Baseline, after Day 90 up to Day 3302
Percent change in LDL-C, total cholesterol, non-HDL-C, triglycerides, HDL-C, VLDL-C from baseline to each assessment time up to Day 720 (Year 2)
Evaluate the effect of inclisiran, compared to placebo (for Year 1) and long-term (up to Day 720), on lowering LDL-C, other lipoprotein and lipid parameters, and PCSK9 over time
Time frame
Baseline, up to Day 7203
Percent change in PCSK9 from baseline to each assessment time up to Day 720 (Year 2)
Evaluate the effect of inclisiran, compared to placebo (for Year 1) and long-term (up to Day 720), on lowering LDL-C, other lipoprotein and lipid parameters, and PCSK9 over time
Time frame
Baseline, up to Day 7204
Percent change in Apo B, Apo A1 from baseline to each assessment time up to Day 720 (Year 2)
Evaluate the effect of inclisiran, compared to placebo (for Year 1) and long-term (up to Day 720), on lowering LDL-C, other lipoprotein and lipid parameters, and PCSK9 over time
Time frame
Baseline, up to Day 720Sponsors and contacts
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Source ClinicalTrials.gov