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Clinical Trial Details — Status: Active, not recruiting

Administrative data

NCT number NCT05185843
Other study ID # ISIS 678354-CS7
Secondary ID 2021-003635-29
Status Active, not recruiting
Phase Phase 3
First received
Last updated
Start date February 25, 2022
Est. completion date June 2025

Study information

Verified date May 2024
Source Ionis Pharmaceuticals, Inc.
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial 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.


Description:

This is a Phase 3, multi-center, open-label safety study of up to 30 participants with FCS, previously treated with volanesorsen. The study consists of 3 periods: 1) Screening Period: Week -8 to Week -1 (up to 8 weeks); 2) Treatment Period up to Week 157; and 3) Post-Treatment Follow-up Period: Week 158 to Week 170 (12 weeks). Participants enrolled will receive olezarsen every 4 weeks during the 157-week Treatment Period. This study was extended to allow participants to receive olezarsen for an additional 104 weeks following the initial 53-week treatment period.


Recruitment information / eligibility

Status Active, not recruiting
Enrollment 24
Est. completion date June 2025
Est. primary completion date March 2025
Accepts healthy volunteers No
Gender All
Age group 18 Years and older
Eligibility 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 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

Study Design


Related Conditions & MeSH terms


Intervention

Drug:
Olezarsen
Olezarsen will be administered by SC injection.

Locations

Country Name City State
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 ARC Biosystems, Clinical Assessment Unit (CAU) Vancouver British Columbia
Canada Centre for Heart Lung Innovation Vancouver British Columbia
Canada St. Boniface General Hospital Winnipeg Manitoba
Sweden Karolinska University Hospital Huddinge Stockholm
United States University of Michigan, Department of Internal Medicine, Division of Metabolism, Endocrinology and Diabetes (MEND) Ann Arbor Michigan
United States Excel Medical Clinical Trials, LLC Boca Raton Florida
United States Diabetes/Lipid Management & Research Center Huntington Beach California
United States University of Rochester School of Medicine Rochester New York

Sponsors (1)

Lead Sponsor Collaborator
Ionis Pharmaceuticals, Inc.

Countries where clinical trial is conducted

United States,  Canada,  Sweden, 

Outcome

Type Measure Description Time frame Safety issue
Primary 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 157
Primary Proportion of Participants With Major or Clinically Relevant Non-major Bleeding Events Baseline to Week 157
Primary Proportion of Participants With Decrease in Estimated Glomerular Filtration Rate (eGFR) by >30% or >50% Baseline to Week 157
Primary 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 157
Primary Proportion of Participants With Alanine Aminotransferase (ALT) or Aspartate Aminotransferase (AST) >5 x Upper Limit of Normal (ULN) Baseline to Week 157
Primary Proportion of Participants With ALT or AST >3 x ULN and Total Bilirubin > 2 x ULN Baseline to Week 157
Primary Proportion of Participants With Total Bilirubin >2 mg/deciliter (dL) Baseline to Week 157
Secondary Trough (Pre-Dose) Plasma Concentration of Olezarsen Up to 157 weeks
Secondary Post-Treatment Plasma Concentration of Olezarsen Up to 170 weeks
Secondary Change and Percent Change From Baseline in Fasting Triglycerides (TG) Baseline to Week 157
Secondary Change and Percent Change From Baseline in Fasting Apolipoprotein C-III (APOC-III) Baseline to Week 157
Secondary Change and Percent Change From Baseline in Fasting Very Low-Density Lipoprotein (VLDL)-C Baseline to Week 157
Secondary Change and Percent Change From Baseline in Fasting Chylomicron-TG Baseline to Week 157
Secondary Change and Percent Change From Baseline in Fasting Total Cholesterol (TC) Baseline to Week 157
Secondary Change and Percent Change From Baseline in Fasting Non-High-Density Lipoprotein (non-HDL)-C Baseline to Week 157
Secondary Change and Percent Change From Baseline in Fasting Low-Density Lipoprotein (LDL)-C Baseline to Week 157
Secondary Change and Percent Change From Baseline in Fasting Apoprotein B (apoB) Baseline to Week 157
Secondary Change and Percent Change From Baseline in Fasting Apoprotein B48 (apoB48) Baseline to Week 157
Secondary Change and Percent Change From Baseline in Fasting High-Density Lipoprotein (HDL)-C Baseline to Week 157
Secondary Change and Percent Change From Baseline in Fasting Apoprotein A-1 (ApoA-1) Baseline to Week 157
Secondary Event Rate of Acute Pancreatitis Up to 157 weeks
See also
  Status Clinical Trial Phase
Completed NCT04223908 - InFocus France Epidemiological Study of Health Burden in Major Hypertriglyceridemia
Active, not recruiting NCT05130450 - A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) in Participants With Familial Chylomicronemia Syndrome (FCS) Phase 3
Completed NCT04568434 - A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) Administered to Patients With Familial Chylomicronemia Syndrome (FCS) Phase 3
Completed NCT03912181 - Medical Complications in Familial and Multifactorial Chylomicronaemia Syndromes
Not yet recruiting NCT05902598 - A Phase 3 Study of VSA001 in Chinese Adults With Familial Chylomicronemia Syndrome Phase 3
Completed NCT02658175 - The Approach Open Label Study: A Study of Volanesorsen (Formerly IONIS-APOCIIIRx) in Participants With Familial Chylomicronemia Syndrome Phase 3
Completed NCT03360747 - Phase 2 Study of AKCEA-ANGPTL3-LRx (ISIS 703802) in Participants With Familial Chylomicronemia Syndrome (FCS) Phase 2
Completed NCT02211209 - The APPROACH Study: A Study of Volanesorsen (Formerly IONIS-APOCIIIRx) in Patients With Familial Chylomicronemia Syndrome Phase 3
Available NCT06360237 - Olezarsen Early Access Program for Patients With Familial Chylomicronemia Syndrome (FCS)