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

> **NCT04681170** · PHASE3 · COMPLETED · sponsor: **Amryt Pharma** · enrollment: 46 (actual)

## Conditions studied

- Homozygous Familial Hypercholesterolaemia (HoFH)

## Interventions

- **DRUG:** Lomitapide

## Key facts

- **NCT ID:** NCT04681170
- **Lead sponsor:** Amryt Pharma
- **Sponsor class:** INDUSTRY
- **Phase:** PHASE3
- **Study type:** INTERVENTIONAL
- **Status:** COMPLETED
- **Start date:** 2020-12-14
- **Primary completion:** 2022-10-16
- **Final completion:** 2024-06-06
- **Target enrollment:** 46 (ACTUAL)
- **Last updated:** 2025-08-27


## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT04681170

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT04681170, "Efficacy and Safety of Lomitapide in Paediatric Patients With Homozygous Familial Hypercholesterolaemia (HoFH)". Retrieved via AI Analytics 2026-06-25 from https://api.ai-analytics.org/clinical/NCT04681170. Licensed CC0.

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