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

Phase 3 Completed 46 enrolled Amryt Pharma

Non-randomizedParallel-groupOpen-labelTreatment

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)

Timeline

Start: 2020-12-14
Primary completion: 2022-10-16
Completion: 2024-06-06

Outcome

Met primary endpoint

registry analysis (other test); All Age Groups; p <0.0001; Mean Difference (Net) -53.910 (95% CI -61.8590 to -45.9612); t-test, 1 sided NCT04681170 ↗

paper Mean change from baseline in LDL cholesterol at week 24 was -53·5% (95% CI -61·6 to -45·4, p<0·0001). PMID 39426393 ↗

Publications

Masana L, Zambon A, Schmitt CP, Taylan C, Driemeyer J, Cohen H, Buonuomo PS, Alashwal A, Al-Dubayee M, Kholaif N, Diaz-Diaz JL, Maatouk F, Martinez-Hervas S, Mangal B, Lowe S, Cunningham T. Lomitapide for the treatment of paediatric patients with homozygous familial hypercholesterolaemia (APH-19): results from the efficacy phase of an open-label, multicentre, phase 3 study. Lancet Diabetes Endocrinol. 2024 Dec;12(12):880-889. doi: 10.1016/S2213-8587(24)00233-X. Epub 2024 Oct 16.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	lomitapide	Small molecule	20 mg	Oral
Subject	lomitapide	Small molecule	40 mg	Oral
Subject	lomitapide	Small molecule	60 mg	Oral