Lipids & metabolic liver disease
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
Authors' conclusion
Lomitapide provided a significant, clinically meaningful LDL cholesterol reduction and has the potential to be an efficient, LDL receptor-independent option for paediatric patients with HoFH.
Study context
- Population
- Patients aged 5–17 years with homozygous familial hypercholesterolaemia (HoFH) (2014 European Atherosclerosis Society Consensus Panel criteria) on stable lipid-lowering therapy, 12 centres in Germany, Israel, Italy, Saudi Arabia, Spain and Tunisia; median age 10.7 years, 56% female
- Participants
- 43 patients
- Outcome
- Percentage change from baseline in LDL-C
- Follow-up
- Week 24
Methodological detailsopen-label, multicentre, phase 3, 6-week run-in, 24-week efficacy phase, 80-week safety phase, Enrolment Dec 20, 2020 to Oct 16, 2022
- Open-label
- Multicentre
- Phase 3
- 6-week run-in, 24-week efficacy phase, 80-week safety phase
- Enrolment Dec 20, 2020 to Oct 16, 2022
Main finding
As reported in the abstract
Mean % change from baseline in LDL-C
Mean change from baseline in LDL-C
−53.5%
95% CI −61.6 to −45.4p<0.0001
Secondary outcomes
Mean % change in non-HDL cholesterol · week 24
- Mean % reduction
- −53.9% (95% CI −61.7 to −46.1, p<0.0001)
Mean % change in total cholesterol · week 24
- Mean % reduction
- −50.0% (95% CI −57.6 to −42.4, p<0.0001)
Mean % change in VLDL cholesterol · week 24
- Mean % reduction (−59.1 to −41.2, p<0.0001)
- −50.2%
Mean % change in apolipoprotein B · week 24
- Mean % reduction (−60.3 to −44.5, p<0.0001)
- −52.4%
Mean % change in TG · week 24
- Mean % change (−58.8 to −41.0, p<0.0001)
- −49.9%
Mean % change in lipoprotein(a) · week 24
- 21 patients with measurements in mg/dL (−32.9 to 10.3)
- −11.3%
- 22 patients with measurements in nmol/L (−38.2 to −9.0)
- −23.6%
- Combined
- P = 0.0070
Safety
- Adverse events of special interest
- five (12%) patients (gastrointestinal in two, hepatic in three)
- Serious treatment-emergent adverse event: increase in hepatic enzymes (two dose interruptions, two dose reductions, repeated dose escalation)
- 1
- Adverse events mostly mild, gastrointestinal and hepatic
Original publication
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. Published 16 October 2024. doi:10.
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