NCT00943306

Long Term, Follow-on Study of Lomitapide in Patients With Homozygous Familial Hypercholesterolemia

A Phase III, Long Term, Open Label, Follow on Study of Microsomal Triglyceride Transfer Protein (MTP) Inhibitor 'Lomitapide' (LOMITAPIDE) in Patients With Homozygous Familial Hypercholesterolemia

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 19 (actual)
• Sponsor: Aegerion Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This was a long term follow on study to assess the continued long term safety and efficacy of lomitapide in patients with homozygous familial hypercholesterolemia.

Detailed description

This was a phase III open label clinical trial to evaluate the long-term efficacy and safety of lomitapide at the maximum tolerated dose (for each patient) established during the clinical trial 733-005/UP1002. Subjects completing the 78-week treatment period in study 733-005/UP1002 who have not met any of the stopping criteria will be eligible to participate in 733-012. The treatment period continued until a decision was made by the local competent authority regarding marketing authorization. Lomitapide will be given orally once daily. Patient specific doses will be carried forward from 733-005/UP1002, but will not exceed the maximum tolerated dose the patient received during 733-005 /UP1002. The maximum dose for any patient was 80 mg/day. There was no reference therapy in this trial. The effects of the study drug were compared to baseline data (from 733-005/UP1002). Concomitant lipid-lowering therapy including plasmapheresis or LDL apheresis is permitted. Note that hereafter, Week 0 in Study 733-005/UP1002 will be referred to as Baseline and Week 48 in Study AEGR-733-012 will be referred to as Week 126 (of overall treatment).

Conditions

• Familial Hypercholesterolemia

Interventions

Type	Name	Description
DRUG	lomitapide	5-60 mg po every day

Timeline

• Start date: 2009-10-29
• Primary completion: 2012-09-17
• Completion: 2014-12-01
• First posted: 2009-07-22
• Last updated: 2018-06-13
• Results posted: 2018-05-11

Locations

10 sites across 4 countries: United States, Canada, Italy, South Africa