Clinical trial NCT01811472: Study to Assess the Efficacy, Safety and Tolerability of LCQ908 in NAFLD Patients
Official title: 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Non-alcoholic Fatty Liver Disease (NAFLD)
Keywords: Multi-center,
randomized,
double-blind,
NAFLD,
elevated triglycerides
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2013-03-14
Enrollment: 52
Sex: ALL
Minimum age (years): 18
Maximum age (years): 74
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: 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).


Eligibility criteria: 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

Primary outcomes:
measure: Change From Baseline in Percentage of Fat in the Liver as Assessed Using MRI at Week 24
timeFrame: From baseline to week 24
Secondary outcomes:
measure: Change From Baseline in Percentage of Fat in the Liver as Assessed Using MRI at Week 12
timeFrame: From baseline to week 12
measure: Percentage of Responders at Week 12
timeFrame: At week 12
measure: Percentage of Responders at Week 24
timeFrame: From baseline to week 24
measure: Change From Baseline Values for Alanine Aminotransferase (ALT) , Aspartate Aminotransferase (AST) and Gamma-glutamyl Transpeptidase (GGT) to Week 6
timeFrame: From Baseline to week 6
measure: Change From Baseline Values for Alanine Aminotransferase (ALT) , Aspartate Aminotransferase (AST) and Gamma-glutamyl Transpeptidase (GGT) to Week 12
timeFrame: From Baseline to week 12
measure: Change From Baseline Values for Alanine Aminotransferase (ALT) , Aspartate Aminotransferase (AST) and Gamma-glutamyl Transpeptidase (GGT) to Week 24
timeFrame: From Baseline to week 24
measure: Percentage of Patients With Normalized Liver Enzymes
timeFrame: Baseline, week 6, week 12 and week 24
measure: Percent Change From Baseline in Fasting Triglycerides
timeFrame: Baseline, 6, 12 and 24 weeks
measure: Post-prandial Peak Triglycerides Over 0 - 8 Hours
timeFrame: Baseline, 6 and 24 weeks
measure: Change From Baseline in Body Weight
timeFrame: Baseline, 12 and 24 weeks
measure: Change From Baseline in Waist Circumference
timeFrame: Baseline, 12 and 24 weeks
measure: Number of Patients With Adverse Events, Serious Adverse Events (SAEs) and Death as Assessment of Safety and Tolerability
timeFrame: 24 weeks
Other outcomes:
Locations:
city: Mobile
country: United States
facility: Novartis Investigative Site
state: Alabama
city: San Diego
country: United States
facility: Novartis Investigative Site
state: California
city: Gainesville
country: United States
facility: Novartis Investigative Site
state: Florida
city: Miami
country: United States
facility: Novartis Investigative Site
state: Florida
city: Tamarac
country: United States
facility: Novartis Investigative Site
state: Florida
city: Honolulu
country: United States
facility: Novartis Investigative Site
state: Hawaii
city: Louisville
country: United States
facility: Novartis Investigative Site
state: Kentucky
city: Tupelo
country: United States
facility: Novartis Investigative Site
state: Mississippi
city: Houston
country: United States
facility: Novartis Investigative Site
state: Texas
city: Plano
country: United States
facility: Novartis Investigative Site
state: Texas
city: Richmond
country: United States
facility: Novartis Investigative Site
state: Virginia
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References: