A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRX) Administered to Adults With Familial Chylomicronemia Syndrome (FCS) Previously Treated With Volanesorsen
- Conditions
- Familial Chylomicronemia Syndrome
- Interventions
- Registration Number
- NCT05185843
- Lead Sponsor
- Ionis Pharmaceuticals, Inc.
- Brief Summary
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.
- Detailed Description
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.
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 24
Not provided
-
Treatment with another investigational drug (non-oligonucleotide), biological agent, or device within 4 weeks of Screening, or 5 half-lives of investigational agent, whichever is longer
-
Concomitant medication/procedure restrictions:
- Systemic corticosteroids or anabolic steroids within 6 weeks prior to Screening and during the study unless approved by the Sponsor Medical Monitor
- Plasma apheresis within 4 weeks prior to Screening or planned during the study
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Olezarsen Olezarsen Olezarsen will be administered once every 4 weeks by subcutaneous (SC) injection for up to 209 weeks.
- Primary Outcome Measures
Name Time Method Proportion of Participants With Decrease in Estimated Glomerular Filtration Rate (eGFR) by ≥30% or ≥50% 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 Baseline to Week 209 Proportion of Participants With Alanine Aminotransferase (ALT) or Aspartate Aminotransferase (AST) ≥5 x Upper Limit of Normal (ULN) Baseline to Week 209 Proportion of Participants With ALT or AST ≥3 x ULN and Total Bilirubin > 2 x ULN Baseline to Week 209 Proportion of Participants With Total Bilirubin ≥2 mg/deciliter (dL) Baseline to Week 209 Proportion of Participants With Clinical Bleeding Events Baseline to Week 209 Proportion of Participants With Decrease in Platelet Count by >30% or >50%, or With Platelet Count Value <50,000/cubic millimeter (mm^3) Baseline to Week 209
- Secondary Outcome Measures
Name Time Method Change and Percent Change From Baseline in Fasting Triglycerides (TG) Baseline to Week 209 Change and Percent Change From Baseline in Fasting Apolipoprotein C-III (APOC-III) Baseline to Week 209 Change and Percent Change From Baseline in Fasting Very Low-Density Lipoprotein (VLDL)-C Baseline to Week 209 Change and Percent Change From Baseline in Fasting Chylomicron-TG Baseline to Week 209 Change and Percent Change From Baseline in Fasting Total Cholesterol (TC) Baseline to Week 209 Change and Percent Change From Baseline in Fasting Non-High-Density Lipoprotein (non-HDL)-C Baseline to Week 209 Change and Percent Change From Baseline in Fasting Low-Density Lipoprotein (LDL)-C Baseline to Week 209 Change and Percent Change From Baseline in Fasting Apoprotein B (apoB) Baseline to Week 209 Change and Percent Change From Baseline in Fasting Apoprotein B48 (apoB48) Baseline to Week 209 Change and Percent Change From Baseline in Fasting High-Density Lipoprotein (HDL)-C Baseline to Week 209 Change and Percent Change From Baseline in Fasting Apoprotein A-1 (ApoA-1) Baseline to Week 209 Event Rate of Acute Pancreatitis Up to 209 weeks Trough (Pre-Dose) Plasma Concentration of Olezarsen Up to 209 weeks Post-Treatment Plasma Concentration of Olezarsen Up to 209 weeks
Trial Locations
- Locations (11)
Ecogene-21
🇨🇦Chicoutimi, Quebec, Canada
Centre Hospitalier Universite de Sherbrooke (CHUS)
🇨🇦Sherbrooke, Quebec, Canada
Diabetes/Lipid Management & Research Center
🇺🇸Huntington Beach, California, United States
ARC Biosystems, Clinical Assessment Unit (CAU)
🇨🇦Vancouver, British Columbia, Canada
Excel Medical Clinical Trials, LLC
🇺🇸Boca Raton, Florida, United States
Karolinska University Hospital Huddinge
🇸🇪Stockholm, Sweden
University of Rochester School of Medicine
🇺🇸Rochester, New York, United States
Clinique des Maladies Lipidiques de Quebec Inc.
🇨🇦Québec, Quebec, Canada
University of Michigan, Department of Internal Medicine, Division of Metabolism, Endocrinology and Diabetes (MEND)
🇺🇸Ann Arbor, Michigan, United States
St. Boniface General Hospital
🇨🇦Winnipeg, Manitoba, Canada
Centre for Heart Lung Innovation
🇨🇦Vancouver, British Columbia, Canada