A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) Administered to Patients With Familial Chylomicronemia Syndrome (FCS)
BALANCE
A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Study of AKCEA-APOCIII-LRx Administered Subcutaneously to Patients With Familial Chylomicronemia Syndrome (FCS)
2 other identifiers
interventional
66
11 countries
47
Brief Summary
The purpose of the study was to evaluate the efficacy of olezarsen as compared to placebo on the percent change in fasting triglycerides (TG) from baseline.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_3
Started Nov 2020
Typical duration for phase_3
47 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
September 23, 2020
CompletedFirst Posted
Study publicly available on registry
September 29, 2020
CompletedStudy Start
First participant enrolled
November 18, 2020
CompletedPrimary Completion
Last participant's last visit for primary outcome
July 14, 2023
CompletedStudy Completion
Last participant's last visit for all outcomes
October 17, 2023
CompletedResults Posted
Study results publicly available
March 6, 2025
CompletedMarch 6, 2025
February 1, 2025
2.7 years
September 23, 2020
February 13, 2025
February 13, 2025
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Percent Change From Baseline in Fasting TG at Month 6
Baseline, Month 6
Secondary Outcomes (14)
Percent Change From Baseline in Fasting TG at Month 12
Baseline, Month 12
Percent Change From Baseline in Fasting Apolipoprotein C-III (apoC-III) at Months 6 and 12
Baseline, Months 6 and 12
Percentage of Participants With ≥ 40% Reduction in Fasting TG at Month 6
Month 6
Percent Change From Baseline in Fasting Apolipoprotein B-48 (apoB-48) at Months 6 and 12
Baseline, Months 6 and 12
Percent Change From Baseline in Fasting Non-High-Density Lipoprotein Cholesterol (Non-HDL-C) at Months 6 and 12
Baseline, Months 6 and 12
- +9 more secondary outcomes
Study Arms (3)
Placebo
PLACEBO COMPARATORParticipants received olezarsen-matching placebo, once every 4 weeks by subcutaneous (SC) injection, during Weeks 1 to 49 of the 53-week treatment period.
Olezarsen 50 mg
EXPERIMENTALParticipants received olezarsen, 50 milligrams (mg), once every 4 weeks by SC injection, during Weeks 1 to 49 of the 53-week treatment period.
Olezarsen 80 mg
EXPERIMENTALParticipants received olezarsen 80 mg, once every 4 weeks by SC injection, during Weeks 1 to 49 of the 53-week treatment period.
Interventions
Eligibility Criteria
You may qualify if:
- A diagnosis of genetically confirmed Familial Chylomicronemia Syndrome (type 1 Hyperlipoproteinemia)
- Fasting TG ≥ 880 mg/dL (10 millimoles per liter (mmol/L) at Screening
- History of pancreatitis. Patients without a documented history of pancreatitis are also eligible but their enrollment will be capped at 35%
- Stable doses of statins, omega-3 fatty acids, fibrates, or other lipid-lowering medications are allowed
You may not qualify if:
- Acute coronary syndrome within 6 months of Screening
- Major surgery within 3 months of Screening
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (47)
Diabetes/Lipid Management & Research Center
Huntington Beach, California, 92648, United States
University of California, San Francisco (UCSF) - Medical Center
San Francisco, California, 94143, United States
Excel Medical Clinical Trials, LLC
Boca Raton, Florida, 33434, United States
NorthShore University Health System
Evanston, Illinois, 60201, United States
Advocate Health and Hospitals Corporation - Lutheran General Hospital
Park Ridge, Illinois, 60068, United States
Ascension St. Vincent Cardiovascular Research Institute
Indianapolis, Indiana, 46290, United States
University of Kansas Medical Center (KUMC)
Kansas City, Kansas, 66160, United States
University of Michigan- Endocrinology & Metabolism
Ann Arbor, Michigan, 48105, United States
New York University (NYU) Langone Medical Center
New York, New York, 10016, United States
Columbia University Medical Center
New York, New York, 10032, United States
Moses H. Cone Memorial Hospital
Greensboro, North Carolina, 27401, United States
University of Pennsylvania
Philadelphia, Pennsylvania, 19104, United States
Vanderbilt University
Nashville, Tennessee, 37232, United States
University of Texas Southwestern
Dallas, Texas, 75390, United States
Baylor College of Medicine
Houston, Texas, 77030, United States
York Clinical Research LLC
Norfolk, Virginia, 23510, United States
West Virginia University Heart and Vascular Institute
Morgantown, West Virginia, 26506, United States
Ecogene-21
Chicoutimi, Quebec, G7H 7K9, Canada
Nathalie Saint-Pierre
Montreal, Quebec, H2W 1R7, Canada
Clinique des Maladies Lipidiques de Quebec Inc.
Québec, Quebec, G1V 4W2, Canada
Institute de Recherches Cliniques de Montreal
Montreal, H2W 1R7, Canada
Hôpital Louis Pradel - HCL
Bron, 69677, France
CHU Dijon - Bocage
Dijon, 21000, France
Assistance Publique - Hopitaux de Marseille
Marseille, 13385, France
ASST Grande Ospedale Metropolitano Niguarda
Milan, 20162, Italy
Azienda Ospedaliera Universitaria Federico II
Napoli, 80131, Italy
Azienda Ospedaliera Universitaria Policlinico Paolo Giaccone
Palermo, 90127, Italy
Azienda Ospedaliero Universitaria Policlinico Umberto I
Roma, 00161, Italy
Academic Medical Center - Department of Vascular Medicine
Amsterdam, 1105 AZ, Netherlands
Erasmus MC
Rotterdam, 3015 GD, Netherlands
Universitair Medisch Centrum Utrecht
Utrecht, 3584 CX, Netherlands
The Lipid Clinic (Oslo University Hospital)
Oslo, 0372, Norway
Centro Hospitalar de Lisboa Ocidental. E.P.E, - Hospital Santa Cruz
Carnaxide, 2790-134, Portugal
Hospital da Senhora da Oliveira - Guimaraes
Creixomil, 4835-044, Portugal
Centro Hospitalar de Lisboa Ocidental, EPE - Hospital Egas Moniz
Lisbon, 1349-019, Portugal
Metabolicke centrum MUDr Katariny Raslovej s. r. o.
Bratislava, 83301, Slovakia
Hospital Abente y Lago
A Coruña, 15001, Spain
Hospital Clinic Barcelona
Barcelona, 08036, Spain
Hospital Universitario 12 de Octubre
Madrid, 28041, Spain
Hospital Universitario Virgen del Rocio
Seville, 41013, Spain
Fundacio Pere Virgili
Tarragona, 43204, Spain
Sahlgrenska University Hospital
Gothenburg, 413 45, Sweden
Karolinska University Hospital
Solna, 171 76, Sweden
The Royal Free Hospital
London, NW3 2QG, United Kingdom
St. Thomas' Hospital
London, SE1 7EH, United Kingdom
Manchester University NHS Foundation Trust (MFT)
Manchester, MI39WL, United Kingdom
Sandwell General Hospital
West Bromwich, B71 4HJ, United Kingdom
Related Publications (2)
Stroes ESG, Alexander VJ, Karwatowska-Prokopczuk E, Hegele RA, Arca M, Ballantyne CM, Soran H, Prohaska TA, Xia S, Ginsberg HN, Witztum JL, Tsimikas S; Balance Investigators. Olezarsen, Acute Pancreatitis, and Familial Chylomicronemia Syndrome. N Engl J Med. 2024 May 16;390(19):1781-1792. doi: 10.1056/NEJMoa2400201. Epub 2024 Apr 7.
PMID: 38587247RESULTSikora Kessler A, Vera-Llonch M, Karwatowska-Prokopczuk E, Alexander VJ, Ann Q, Mejia Herrera C, Paparrodopoulos S, Xia S, Stroes ESG, Baum SJ, Tsimikas S; Balance Investigators. Olezarsen reduces all-cause health services utilization and improves the treatment experience of patients with familial chylomicronemia syndrome. J Clin Lipidol. 2025 Dec 19:S1933-2874(25)00547-1. doi: 10.1016/j.jacl.2025.12.018. Online ahead of print.
PMID: 41545246DERIVED
MeSH Terms
Conditions
Interventions
Results Point of Contact
- Title
- Ionis Pharmaceuticals, Inc.
- Organization
- Ionis Pharmaceuticals, Inc.
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- OTHER
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- RANDOMIZED
- Masking
- DOUBLE
- Who Masked
- PARTICIPANT, INVESTIGATOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
- Expanded Access
- Yes
Study Record Dates
First Submitted
September 23, 2020
First Posted
September 29, 2020
Study Start
November 18, 2020
Primary Completion
July 14, 2023
Study Completion
October 17, 2023
Last Updated
March 6, 2025
Results First Posted
March 6, 2025
Record last verified: 2025-02
Data Sharing
- IPD Sharing
- Will share
Ionis may share anonymized individual participant data, aggregated clinical data, and other types of data that support the results in this study. Data requests from qualified researchers will be considered once all three of the following criteria are met: (1) 12 months from marketing approval of the study drug in both the United States and European Union; (2) 18 months from conclusion of the study; and (3) 6 months from publication of study article. Access would be via a secure environment and is contingent upon approval of a research proposal and entry into an appropriate data use agreement. Requests to access data can be submitted via the website https://vivli.org/ourmember/ionis/.