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

Aegerion Pharmaceuticals

Status and phase: Completed; Phase 3

Conditions: Familial Hypercholesterolemia

Treatments: Drug: lomitapide

Study type: Interventional

Funder types: Industry

Identifiers

NCT00943306

AEGR-733-012

Details and patient eligibility

About

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.

Full 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).

Enrollment: 19 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

1. Completed UP1002 or 733-005.
2. Willing and able to provide consent and comply with the requirements of the study protocol.

Exclusion criteria

1. Met any of the stopping rules for study discontinuation at the final visit of study UP1002 or 733-005.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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19 participants in 1 patient group

lomitapide

Experimental group

Description:

Maximum tolerated dose of lomitapide in addition to existing lipid lowering therapy including plasmapheresis or lipid apheresis.

Treatment:

Drug: lomitapide