NCT04681170

Efficacy and Safety of Lomitapide in Paediatric Patients With Homozygous Familial Hypercholesterolaemia (HoFH)

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

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 46 (actual)
• Sponsor: Amryt Pharma · Industry
• Sex: All
• Age: 5 Years – 17 Years
• Healthy volunteers: Not accepted

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)

Detailed description

Lomitapide has been approved for use in adult patients with HoFH in the European Union (EU) and European Economic Area (EEA), United States of America (USA), Israel, Argentina, Canada, Colombia, and Japan. This study is designed to determine if lomitapide is effective and can be safely administered to paediatric patients with HoFH. If the efficacy and safety so far observed in adults is confirmed in paediatric patients, the potential exists to significantly lower low-density lipoprotein cholesterol (LDL-C) levels in paediatric patients with HoFH. Furthermore, the lower LDL-C levels may reduce atherosclerosis progression and would be expected to benefit these paediatric patients with HoFH. A single arm, non comparator design has been selected due to the rarity of the disease and because the evaluation of safety variables such as growth and sexual maturation requires longer term observation than would be feasible in the context of a placebo controlled study. To mitigate the disadvantages of a single arm design, the study includes a Run in Period of at least 6 weeks during which current lipid lowering therapy (LLT) (including lipoprotein apheresis (LA), when applicable) will be stabilised to establish baseline levels allowing each patient to serve as his/her own control. Patients will also remain on stable LLT (including LA, when applicable) during the Efficacy Phase of the study through Week 24±3 days.

Conditions

• Homozygous Familial Hypercholesterolaemia (HoFH)

Interventions

Type	Name	Description
DRUG	Lomitapide	2mg,5mg, 10mg and 20mg capsules

Timeline

• Start date: 2020-12-14
• Primary completion: 2022-10-16
• Completion: 2024-06-06
• First posted: 2020-12-23
• Last updated: 2025-08-27
• Results posted: 2025-08-27

Locations

12 sites across 6 countries: Germany, Israel, Italy, Saudi Arabia, Spain, Tunisia