Study to Assess the Efficacy, Safety and Tolerability of LCQ908 in NAFLD Patients

A Randomized, Multicenter, Double-blind, Placebo-controlled, Parallel-group, 24-week Pilot Study to Assess the Efficacy, Safety and Tolerability of LCQ908 in Patients With Non-alcoholic Fatty Liver Disease

The purpose of this study was to determine whether LCQ908 effectively lowers liver fat, as assessed by MRI and to assess its safety and tolerability profile in subjects with non-alcoholic fatty liver disease (NAFLD).

Inclusion Criteria:

• History of liver steatosis during the preceding 24 months

• History of fasting TGs > 200 mg/dL (confirmed at screening).

• Liver fat ≥ 10% as determined by the central MRI laboratory.

• Subjects on the following medications can be included if these medications are medically necessary, cannot be stopped and the investigator feels their dose will remain stable for the duration of the double-blind treatment period:

  1. Stable dose of anti-diabetic medications (metformin and/or sulfonylureas) for at least 8 weeks prior to screening.
  2. Stable doses of beta-blockers and thiazide diuretics for at least 8 weeks prior to screening.
  3. Stable doses of fibrates, statins, niacin, ezetimibe for at least 8 weeks prior to screening.
  4. Stable dose of vitamin E in patients taking >200 IU/day for at least 6 months prior to screening.

Exclusion Criteria:

• Treatment with omega-3-acid ethyl esters or omega-3-polyunsaturated fatty acid (PUFA)-containing supplements > 200 mg per day within 8 weeks of screening.
• Treatment with antiretrovirals, tamoxifen, methotrexate, cyclophosphamide, isotretinoin, bile acid binding resins or pharmacologic doses of oral glucocorticoids (≥10 mg of prednisone per day or equivalent) within 8 weeks of screening.
• ALT or AST > 250 IU/L at the time of screening.
• History/current evidence of heavy alcohol use or alcoholism (> 21 drinks per week in men and > 14 drinks per week in women) over a 2-year period prior to screening.
• Presence of chronic liver disease, such as chronic hepatitis B and/or C, alcoholic liver disease, hemochromatosis, Wilson's disease, known cirrhosis.
• Platelet count <150,000 at screening.
• BMI >45 Kg/m2.

Other protocol defined inclusion/exclusion criteria may apply