This is a Phase 3 single-arm, open-label, international, multi-center clinical trial to evaluate the efficacy and safety of lomitapide in pediatric patients with HoFH who are receiving stable lipid-lowering therapy, including LDL apheresis. The study is comprised of a 12-week Run-in Period, a primary 24-week Efficacy Phase, followed by an 80-week Safety Phase.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Percent change in LDL-C
Time frame: Baseline, Week 24
Percent Change in TC
Time frame: Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52, 56, 68, 80, 92, 104
Percent change in non-HDL-C
Time frame: Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52, 56, 68, 80, 92, 104
Percent change in HDL-C
Time frame: Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52, 56, 68, 80, 92, 104
Percent change in TG
Time frame: Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52, 56, 68, 80, 92, 104
Percent change in VLDL-C
Time frame: Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52, 56, 68, 80, 92, 104
Percent change in Lp(a)
Time frame: Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52, 56, 68, 80, 92, 104
Percent change in apo B
Time frame: Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52, 56, 68, 80, 92, 104
Percent change in apo A-1
Time frame: Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52, 56, 68, 80, 92, 104
Percent change in LDL-C
Time frame: Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52, 56, 68, 80, 92, 104
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Changes in lipid-lowering therapy
Time frame: Week 24 through Week 104
Changes in LDL apheresis
Time frame: Week 24 through Week 104
Percent of patients achieving goal (LDL-C of <100 mg/dL [2.6 mmol/L] for patients without documented cardiovascular disease [CVD] at Baseline
Time frame: Week 24 and through Week 108
Percent of patients achieving goal LDL-C of <70 mg/dL [1.8 mmol/L]) for patients with documented CVD at Baseline.
Time frame: Week 24 and through Week 108
Changes in laboratory parameters (including hepatic and renal function)
Time frame: Baseline through Year 2
Reported Adverse Events
Time frame: Baseline through Year 2
Electrocardiogram (ECG) changes
Time frame: Baseline through Year 2
Pulmonary function tests (PFTs)
Time frame: Baseline through Year 2
Bone health/age (x-ray of the wrist)
Time frame: Baseline through Year 2
Height Measurement
Time frame: Baseline through Year 2
Weight Measurement
Time frame: Baseline through Year 2
Body Mass Measurement
Time frame: Baseline through Year 2
Tanner Staging
Time frame: Baseline through Year 2
Percent change in hepatic fat
Time frame: Baseline through Year 2
Blood Pressure
Time frame: Baseline through Year 2
Heart Rate
Time frame: Baseline through Year 2
Temperature
Time frame: Baseline through Year 2
Respiration (breaths/min)
Time frame: Baseline through Year 2