The purpose of this study is to determine whether LCQ908 is effective and safe in lowering triglycerides in subjects with Familial Chylomicronemia Syndrome (FCS) (Hyperlipoproteinemia \[HLP\] type I). Data from this study will be used to support a registration submission of LCQ908 20 mg and 40 mg as treatment of chylomicronemia in subjects with FCS (HLP Type 1).
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Enrollment
45
Novartis Investigative Site
Seatlle, Washington, United States
Novartis Investigative Site
Chicoutimi, Quebec, Canada
Novartis Investigative Site
Ste-Foy, Quebec, Canada
Percent Change in Fasting Triglycerides From Baseline to 12 Weeks
Blood samples were collected for a fasting lipid panel, including triglycerides. If the 12-week value was missing, the measurement value at 12 weeks or the last available post-baseline measurement value during the double-blind treatment period was analyzed. Baseline is defined as the average of fasting triglyceride values taken at day -3 and day 1. Adjusted geometric means are calculated by back-transforming the adjusted means from the model and expressing as a percentage change from baseline.
Time frame: Baseline to 12 weeks
Percentage of Patients Responding to Investigational Treatment by Achieving Fasting Triglycerides (TG) of at Least 40% From Baseline or Final Fasting TG < 8.4 mmol/L (750 mg/dL)
Percentage calculated as (m/n)\*100 where m = number of patients who respond; n = the number of patients with non-missing fasting triglyceride.
Time frame: Baseline, 12 weeks, 24 weeks, 52 weeks
Percentage of Patients Responding to Investigational Treatment by Achieving Final Fasting Triglycerides < 8.4 mmol/L (750 mg/dL)
Percentage calculated as (m/n)\*100 where m = number of patients who respond; n = the number of patients with non-missing fasting triglyceride.
Time frame: 12 weeks, 24 weeks, 52 weeks
Percentage of Patients Responding to Investigational Treatment by Achieving Fasting Triglycerides (TG) of at Least 40% From Baseline
Percentage calculated as (m/n)\*100 where m = number of patients who respond; n = the number of patients with non-missing fasting triglyceride.
Time frame: Baseline, 12 weeks, 24 weeks, 52 weeks
Percentage of Patients Achieving Fasting Triglycerides (TG) Target Thresholds
Percentage of patients reaching target values of \<1000 mg/dL or target values of \< 2000 mg/dL for fasting triglycerides is reported. Pecentage calculated as (m/n)\*100; where 'm' The number of patients who reach target values for fasting triglyceride, 'n' the number of patients with non-missing fasting triglyceride.
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Novartis Investigative Site
Ouest-Montreal, Canada
Novartis Investigative Site
Bron, France
Novartis Investigative Site
Nantes, France
Novartis Investigative Site
Paris, France
Novartis Investigative Site
Cologne, Germany
Novartis Investigative Site
Hamburg, Germany
Novartis Investigative Site
Meibergdreef 9, Netherlands
...and 4 more locations
Time frame: 12 weeks, 24 weeks, 52 weeks
Percent Change From Baseline in Fasting Triglycerides
Time frame: Baseline, 24 weeks, 52 weeks
Percent Change From Baseline for Postprandial Triglycerides Following the Standardized Meal Tolerance Test at Week 12
Post prandial peak triglycerides - maximum triglyceride value over 0-24 hours Post prandial triglycerides AUC0-24 - area under the time curve for triglycerides over 0-24 Adjusted geometric means are calculated by back-transforming the adjusted means from the model and expressed as a percentage change from baseline. hours
Time frame: 0-24 hours at Baseline, Week 12
Pharmacokinetics of LCQ908 - Trough Concentration (Cmin) and Observed Maximum Blood Concentration (Cmax)
Lowest observed blood concentration (Cmin) and observed maximum blood concentration (Cmax) following drug administration derived from non-compartmental analysis using scheduled sampling time for the whole dataset.
Time frame: 0, 1, 2, 3, 4, 6, and 24 hours at Week 12
Pharmacokinetics of LCQ908- Area Under the Plasma Concentration Time Curve AUC (0-24hour)
The area under the concentration-time curve from time zero to 24 hours after drug administration was calculated by using linear trapezoidal rule.
Time frame: 0, 1, 2, 3, 4, 6, and 24 hours at Week 12
Pharmacokinetics of LCQ908- Time to Reach Maximum Concentration Following Drug Administration Tmax (Hours)
Time frame: 0, 1, 2, 3, 4, 6, and 24 hours at Week 12
Pharmacokinetics of LCQ908- Average Observed Blood Concentration (Cavg)
Average observed blood concentration measured by (AUC0-24)/24.
Time frame: 0, 1, 2, 3, 4, 6, and 24 hours at Week 12
Number of Patients Reported With Any Adverse Event, Serious Adverse Event and Death
Time frame: 52 weeks