Efficacy and Safety of Lomitapide in Japanese Patients With HoFH on Concurrent Lipid-Lowering Therapy

This is a Phase 3, single-arm, open-label, multicenter clinical trial to evaluate both the efficacy and long-term safety of lomitapide in Japanese patients with HoFH receiving maximally-tolerated, stable lipid-lowering therapy. This study is comprised of a run-in period, a primary 26-week Efficacy Phase, and a 30-week Safety Phase.

Inclusion criteria

• Japanese male and female patients aged ≥ 18 years of age who are receiving maximally tolerated, stable, lipid-lowering therapy
• Diagnosis of functional HoFH
• Body weight ≥ 40 kg and < 136 kg
• Negative pregnancy test at screening

Exclusion criteria

• Uncontrolled hypertension
• History of chronic renal insufficiency
• History of biopsy proven cirrhosis or abnormal liver function tests (LFTs) at screening
• Any major surgical procedure occurring < 3 months prior to the screening visit
• Cardiac insufficiency
• Previous organ transplantation
• History of a non-skin malignancy within the previous 3 years
• Patients who are not able to limit their alcohol intake
• Participation in an investigational drug study within 6 weeks prior to the screening visit
• Known significant gastrointestinal bowel disease
• Nursing mothers
• Serious or unstable medical or psychological conditions
• Requirement for certain prohibited medications known to be potentially hepatotoxic
• Use of strong or moderate inhibitors of CYP3A4
• Use of simvastatin at doses >10 mg per day
• Documented diagnosis of any liver disease