At a glance
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Phase III, Single Arm, Open Label, International, Multi Centre Study to Evaluate the Efficacy and Safety of Lomitapide in Paediatric Patients With Homozygous Familial Hypercholesterolaemia (HoFH) on Stable Lipid Lowering Therapy
In Brief
A Phase 3 clinical trial evaluating Lomitapide for Homozygous Familial Hypercholesterolaemia (HoFH). Completed, enrolled 46 participants across 12 sites in 6 countries.
Detailed Summary
This is a single arm, open label, multi centre phase III study to evaluate the efficacy and long term safety of lomitapide in paediatric patients with HoFH receiving stable lipid lowering therapy (LLT) (including lipoprotein apheresis (LA), when applicable) comprising of the following phases: * Screening Period (starting at Week 12, i.e. ≤12 weeks prior to Baseline for up to 6 weeks) * Stratified Enrolment and Start of Run in Period (starting at minimum at Week 6, i.e., 6 weeks prior to Baseline for a minimum of 6 weeks): * Efficacy Phase (starting at Baseline, i.e. Day \[D\] 0 for 24 weeks±3 days * Safety Phase (starting at Week 24±3 days for 80±1 weeks)
Study Details
Timeline
Interventions
2mg,5mg, 10mg and 20mg capsules