This is a single center, open-label study to evaluate the efficacy of AKCEA-ANGPTL3-LRx for reduction of triglyceride (TG) levels in participants with FCS.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
3
AKCEA-ANGPTL3-LRx at dose 20 mg, administered via SC injection QW.
Investigative Site
Montreal, Quebec, Canada
Absolute Change From Baseline to Month 3 in Fasting Triglycerides (TG)
Baseline was defined as the average of Day 1 predose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment. Month 3 was defined as the average of Week 13 and Week 14 fasting assessments.
Time frame: Baseline to Month 3
Percent Change From Baseline to Month 3 in Fasting Triglycerides (TG)
Baseline was defined as the average of Day 1 predose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment. Month 3 was defined as the average of Week 13 and Week 14 fasting assessments.
Time frame: Baseline to Month 3
Absolute Change From Baseline to Month 3 in Fasting Angiopoietin-Like 3 (ANGPTL3)
Baseline was defined as the average of Day 1 predose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment. Month 3 was defined as the average of Week 13 and Week 14 fasting assessments.
Time frame: Baseline to Month 3
Percent Change From Baseline to Month 3 in Fasting Angiopoietin-like 3 (ANGPTL3)
Baseline was defined as the average of Day 1 predose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment. Month 3 was defined as the average of Week 13 and Week 14 fasting assessments.
Time frame: Baseline to Month 3
Fasting Lipid and Lipoprotein Measurements at Month 3
Fasting lipid and lipoprotein measurements included non-HDL-C, ApoB, HDL-C, ApoA-1, VLDL-C and LDL-C. Month 3 was defined as the average of Week 13 and Week 14 fasting assessments.
Time frame: Month 3
Absolute Change From Baseline to Month 3 in Other Fasting Lipid Parameters
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Other fasting lipid measurements included total cholesterol (TC), non-HDL-C, ApoB, HDL-C, ApoA-1, VLDL-C, and LDL-C. Baseline was defined as average of Day 1 predose fasting assessment and last fasting measurement prior to Day 1 pre-dose fasting assessment. Month 3 was defined as the average of Week 13 and Week 14 fasting assessments.
Time frame: Baseline to Month 3
Percent (%) Change From Baseline to Month 3 in Other Fasting Lipid Parameters
Other fasting lipid measurements included TC, non-HDL-C, ApoB, HDL-C, ApoA-1, VLDL-C, and LDL-C. Baseline was defined as average of Day 1 predose fasting assessment and last fasting measurement prior to Day 1 pre-dose fasting assessment. Month 3 was defined as the average of Week 13 and Week 14 fasting assessments.
Time frame: Baseline to Month 3
Change From Baseline to Day 92 in Maximum Postprandial Triglycerides (TG)
Participants consumed standardized pre-cooked meals (lunches and dinners and instructions for breakfasts and snacks) for 2 days prior to the postprandial assessments. Change from Baseline to Day 92 in maximum postprandial TG was assessed.
Time frame: Baseline to Day 92
Number of Participants Who Experienced Abdominal Pain During the Treatment Period
Time frame: Days 1, 29, 57 and 92
Number of Participants With Treatment Emergent Adverse Events (TEAEs)
An adverse event (AE) was defined as any unfavorable and unintended sign (including a clinically-significant abnormal laboratory finding, for example), symptom, or disease temporally associated with the study or use of investigational drug product, whether or not the AE is considered related to the investigational drug product. A TEAE was defined as any AE starting on or after the first dose of the study drug.
Time frame: From time of informed consent to end of follow-up period (Up to Week 26)