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

NCT04681170 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 46

Last updated 2025-08-27

Study results available
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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)

Conditions

  • Homozygous Familial Hypercholesterolaemia (HoFH)

Interventions

DRUG

Lomitapide

2mg,5mg, 10mg and 20mg capsules

Sponsors & Collaborators

  • Amryt Pharma

    lead INDUSTRY

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
5 Years
Max Age
17 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

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

Countries

  • Germany
  • Israel
  • Italy
  • Saudi Arabia
  • Spain
  • Tunisia

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04681170 on ClinicalTrials.gov