The purpose of the study is to evaluate the safety, tolerability, pharmacokinetic (PK) and pharmacodynamic (PD) effects of olezarsen (formerly known as AKCEA -APOCIII-LRX) in participants with FCS previously treated with volanesorsen.
This is a Phase 3, multi-center, open-label safety study in 24 participants with FCS, previously treated with volanesorsen. The study consists of an 8- week screening period, treatment period up to week 209 and a 13-week follow-up period. Participants enrolled will receive olezarsen every 4 weeks during the 209-week Treatment Period. Treatment period was extended to allow participants to receive olezarsen for an additional 52 weeks for a total of a 209-week treatment period until the drug may be commercially available in the patient's country, or until the Sponsor discontinues the olezarsen development program, whichever occurs earlier.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
24
Olezarsen will be administered by SC injection.
Diabetes/Lipid Management & Research Center
Huntington Beach, California, United States
Excel Medical Clinical Trials, LLC
Boca Raton, Florida, United States
University of Michigan, Department of Internal Medicine, Division of Metabolism, Endocrinology and Diabetes (MEND)
Ann Arbor, Michigan, United States
Proportion of Participants With Decrease in Platelet Count by >30% or >50%, or With Platelet Count Value <50,000/cubic millimeter (mm^3)
Time frame: Baseline to Week 209
Proportion of Participants With Clinical Bleeding Events
Time frame: Baseline to Week 209
Proportion of Participants With Decrease in Estimated Glomerular Filtration Rate (eGFR) by ≥30% or ≥50%
Time frame: Baseline to Week 209
Proportion of Participants With Urine Protein/Creatinine Ratio (UPCR) ≥1000 milligram (mg)/gram (g) or with Urine/Albumin Creatinine Ratio (UACR) ≥500 mg/g
Time frame: Baseline to Week 209
Proportion of Participants With Alanine Aminotransferase (ALT) or Aspartate Aminotransferase (AST) ≥5 x Upper Limit of Normal (ULN)
Time frame: Baseline to Week 209
Proportion of Participants With ALT or AST ≥3 x ULN and Total Bilirubin > 2 x ULN
Time frame: Baseline to Week 209
Proportion of Participants With Total Bilirubin ≥2 mg/deciliter (dL)
Time frame: Baseline to Week 209
Trough (Pre-Dose) Plasma Concentration of Olezarsen
Time frame: Up to 209 weeks
Post-Treatment Plasma Concentration of Olezarsen
Time frame: Up to 209 weeks
Change and Percent Change From Baseline in Fasting Triglycerides (TG)
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University of Rochester School of Medicine
Rochester, New York, United States
Centre for Heart Lung Innovation
Vancouver, British Columbia, Canada
ARC Biosystems, Clinical Assessment Unit (CAU)
Vancouver, British Columbia, Canada
St. Boniface General Hospital
Winnipeg, Manitoba, Canada
Ecogene-21
Chicoutimi, Quebec, Canada
Clinique des Maladies Lipidiques de Quebec Inc.
Québec, Quebec, Canada
Centre Hospitalier Universite de Sherbrooke (CHUS)
Sherbrooke, Quebec, Canada
...and 1 more locations
Time frame: Baseline to Week 209
Change and Percent Change From Baseline in Fasting Apolipoprotein C-III (APOC-III)
Time frame: Baseline to Week 209
Change and Percent Change From Baseline in Fasting Very Low-Density Lipoprotein (VLDL)-C
Time frame: Baseline to Week 209
Change and Percent Change From Baseline in Fasting Chylomicron-TG
Time frame: Baseline to Week 209
Change and Percent Change From Baseline in Fasting Total Cholesterol (TC)
Time frame: Baseline to Week 209
Change and Percent Change From Baseline in Fasting Non-High-Density Lipoprotein (non-HDL)-C
Time frame: Baseline to Week 209
Change and Percent Change From Baseline in Fasting Low-Density Lipoprotein (LDL)-C
Time frame: Baseline to Week 209
Change and Percent Change From Baseline in Fasting Apoprotein B (apoB)
Time frame: Baseline to Week 209
Change and Percent Change From Baseline in Fasting Apoprotein B48 (apoB48)
Time frame: Baseline to Week 209
Change and Percent Change From Baseline in Fasting High-Density Lipoprotein (HDL)-C
Time frame: Baseline to Week 209
Change and Percent Change From Baseline in Fasting Apoprotein A-1 (ApoA-1)
Time frame: Baseline to Week 209
Event Rate of Acute Pancreatitis
Time frame: Up to 209 weeks