A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRX) Administered to Adults With Familial Chylomicronemia Syndrome (FCS) Previously Treated With Volanesorsen
Olezarsen: Olezarsen will be administered by SC injection.
Study 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.
Eligibility
Sex
ALL
Min age
18 Years
Max age
—
Healthy volunteers
No
Key Inclusion Criteria
1. Participants with FCS (clinical or genetic diagnosis) currently on or previously treated with volanesorsen (ISIS 304801)
o Study participants in countries where Waylivra® is commercially approved and available for participants should not be deprived of the treatment option with Waylivra®. Participation in this study for such participants will only be allowed when Waylivra® was discontinued due to AEs
2. The following concomitant medications will be allowed if dosing regimen is expected to remain constant through the end of the study (occasional or intermittent use of over-the-counter (OTC) medications will be allowed at Investigator's discretion):
* Statins, omega-3 fatty acids (prescription and OTC), fibrates, or other lipid-lowering medications. Participants taking OTC omega-3 fatty acids should make every effort to remain on the same brand through the end of the study
* Antidiabetic medications
* Oral anticoagulants (e.g., dabigatran, rivaroxaban, or apixaban, and warfarin with regular clinical monitoring)
* Tamoxifen, estrogens or progestins
Key Exclusion Criteria:
1. 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
2. Concomitant medication/procedure restrictions:
1. Systemic corticosteroids or anabolic steroids within 6 weeks prior to Screening and during the study unless approved by the Sponsor Medical Monitor
2. Plasma apheresis within 4 weeks prior to Screening or planned during the study
Primary outcome measure(s)
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
Proportion of Participants With Clinical Bleeding Events — Baseline to Week 209
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
Trial sites (11)
Facility
City
Region
Status
Diabetes/Lipid Management & Research Center
Huntington Beach
California
Excel Medical Clinical Trials, LLC
Boca Raton
Florida
University of Michigan, Department of Internal Medicine, Division of Metabolism, Endocrinology and Diabetes (MEND)
Ann Arbor
Michigan
University of Rochester School of Medicine
Rochester
New York
Centre for Heart Lung Innovation
Vancouver
British Columbia
ARC Biosystems, Clinical Assessment Unit (CAU)
Vancouver
British Columbia
St. Boniface General Hospital
Winnipeg
Manitoba
Ecogene-21
Chicoutimi
Quebec
Clinique des Maladies Lipidiques de Quebec Inc.
Québec
Quebec
Centre Hospitalier Universite de Sherbrooke (CHUS)
Sherbrooke
Quebec
Karolinska University Hospital Huddinge
Stockholm
Sweden
More Ionis Pharmaceuticals, Inc. trials in the USA
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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