Safety of Lomitapide in Paediatric Patients With Homozygous Familial Hypercholesterolaemia (HoFH)
Status:
Recruiting
Trial end date:
2023-09-30
Target enrollment:
Participant gender:
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 LLT
(including 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)