Back to resultsA Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) Administered to Patients With Familial Chylomicronemia Syndrome (FCS) (BALANCE)
Primary Purpose
Familial Chylomicronemia Syndrome
Status
Active
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Olezarsen
Placebo
Sponsored by
About this trial
This is an interventional treatment trial for Familial Chylomicronemia Syndrome focused on measuring FCS
Eligibility Criteria
Key Inclusion Criteria:
- 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
Key Exclusion Criteria:
- Acute coronary syndrome within 6 months of Screening
- Major surgery within 3 months of Screening
- Have any other conditions, which, in the opinion of the Investigator would make the participant unsuitable for inclusion, or could interfere with participating in or completing the study
Sites / Locations
- Diabetes/Lipid Management & Research Center
- University of California, San Francisco (UCSF) - Medical Center
- Excel Medical Clinical Trials, LLC
- NorthShore University Health System
- Advocate Health and Hospitals Corporation - Lutheran General Hospital
- Ascension St. Vincent Cardiovascular Research Institute
- University of Kansas Medical Center (KUMC)
- University of Michigan- Endocrinology & Metabolism
- New York University (NYU) Langone Medical Center
- Columbia University Medical Center
- Moses H. Cone Memorial Hospital
- University of Pennsylvania
- Vanderbilt University
- University of Texas Southwestern
- Baylor College of Medicine
- York Clinical Research LLC
- West Virginia University Heart and Vascular Institute
- Ecogene-21
- Nathalie Saint-Pierre
- Clinique des Maladies Lipidiques de Quebec Inc.
- Institute de Recherches Cliniques de Montreal
- Hôpital Louis Pradel - HCL
- CHU Dijon - Bocage
- Assistance Publique - Hopitaux de Marseille
- Fejer Megyei Szent Gyorgy Egyetemi Oktato Korhaz
- Ziv Medical Center
- ASST Grande Ospedale Metropolitano Niguarda
- Azienda Ospedaliera Universitaria Federico II
- Azienda Ospedaliera Universitaria Policlinico Paolo Giaccone
- Azienda Ospedaliero Universitaria Policlinico Umberto I
- Academic Medical Center - Department of Vascular Medicine
- Erasmus MC
- Universitair Medisch Centrum Utrecht
- The Lipid Clinic (Oslo University Hospital)
- Centro Hospitalar de Lisboa Ocidental. E.P.E, - Hospital Santa Cruz
- Hospital da Senhora da Oliveira - Guimaraes
- Centro Hospitalar de Lisboa Ocidental, EPE - Hospital Egas Moniz
- Metabolicke centrum MUDr Katariny Raslovej s. r. o.
- Hospital Abente y Lago
- Hospital Clinic Barcelona
- Hospital Universitario 12 de Octubre
- Hospital Universitario Virgen del Rocio
- Fundacio Pere Virgili
- Sahlgrenska University Hospital
- Karolinska University Hospital
- The Royal Free Hospital
- St. Thomas' Hospital
- Manchester University NHS Foundation Trust (MFT)
- Sandwell General Hospital
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Placebo Comparator
Arm Label
Olezarsen
Placebo
Arm Description
Olezarsen will be administered once every 4 weeks by subcutaneous (SC) injection from Week 1 through Week 49.
Olezarsen-matching placebo will be administered once every 4 weeks by SC injection from Week 1 through Week 49.
Outcomes
Primary Outcome Measures
Percent Change from Baseline in Fasting TG at 6 Months (average of Weeks 23, 25, and 27) compared to placebo
Secondary Outcome Measures
Percent Change from Baseline in Fasting TG at 12 months (average of Weeks 51 and 53) compared to placebo
Change from Baseline in the Proportion of Participants who Achieve ≥ 40% Reduction in Fasting TG at 6 Months Compared to Placebo
Percent Change form Baseline in Fasting Apolipoprotein B-48 (apoB-48) at 6 Months Compared to Placebo
Proportion of Participants who Achieve Fasting TG ≤ 750 mg/dL at 6 Months compared to placebo
Adjudicated Acute Pancreatitis Event Rate during the Treatment Period Compared to Placebo, in Participants with ≥ 2 Events of Adjudicated Acute Pancreatitis in 5 Years prior to Enrollment
Adjudicated Acute Pancreatitis Event Rate during the Treatment Period Compared to Placebo
Change from Baseline in the Proportion of Participants who Achieve ≥ 70% Reduction in Fasting TG at 6 Months Compared to Placebo
Proportion of Participants who Achieve Fasting TG ≤ 500 mg/dL at 6 Months Compared to Placebo
Full Information
NCT ID
NCT04568434
First Posted
September 23, 2020
Last Updated
August 3, 2023
Sponsor
Ionis Pharmaceuticals, Inc.
Collaborators
Akcea Therapeutics
1. Study Identification
Unique Protocol Identification Number
NCT04568434
Brief Title
A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) Administered to Patients With Familial Chylomicronemia Syndrome (FCS)
Acronym
BALANCE
Official Title
A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Study of AKCEA-APOCIII-LRx Administered Subcutaneously to Patients With Familial Chylomicronemia Syndrome (FCS)
Study Type
Interventional
2. Study Status
Record Verification Date
August 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
November 16, 2020 (Actual)
Primary Completion Date
July 14, 2023 (Actual)
Study Completion Date
October 2023 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Ionis Pharmaceuticals, Inc.
Collaborators
Akcea Therapeutics
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Product Manufactured in and Exported from the U.S.
Yes
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
The purpose of the study is to evaluate the efficacy of Olezarsen as compared to placebo on the percent change in fasting triglycerides (TG) from baseline.
Detailed Description
This is a multi-center, double-blind, Phase 3 study in up to 60 patients with FCS. Participants will be randomized in a 2:1 ratio to receive Olezarsen or matching placebo in a 53-week treatment period. The length of participation in the study is approximately 74 weeks, which includes an up to 8-week screening period, a 53-week treatment period, and a 13-week post-treatment evaluation period. Following the treatment period, eligible patients may have the option of enrolling in an open label extension study.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Familial Chylomicronemia Syndrome
Keywords
FCS
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantInvestigator
Allocation
Randomized
Enrollment
66 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Olezarsen
Arm Type
Experimental
Arm Description
Olezarsen will be administered once every 4 weeks by subcutaneous (SC) injection from Week 1 through Week 49.
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Olezarsen-matching placebo will be administered once every 4 weeks by SC injection from Week 1 through Week 49.
Intervention Type
Drug
Intervention Name(s)
Olezarsen
Other Intervention Name(s)
ISIS 678354, AKCEA-APOCIII-LRx
Intervention Description
Olezarsen will be administered by SC injection.
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Olezarsen-matching placebo will be administered by SC injection.
Primary Outcome Measure Information:
Title
Percent Change from Baseline in Fasting TG at 6 Months (average of Weeks 23, 25, and 27) compared to placebo
Time Frame
Baseline and Month 6
Secondary Outcome Measure Information:
Title
Percent Change from Baseline in Fasting TG at 12 months (average of Weeks 51 and 53) compared to placebo
Time Frame
Baseline and Month 12
Title
Change from Baseline in the Proportion of Participants who Achieve ≥ 40% Reduction in Fasting TG at 6 Months Compared to Placebo
Time Frame
Baseline and Month 6
Title
Percent Change form Baseline in Fasting Apolipoprotein B-48 (apoB-48) at 6 Months Compared to Placebo
Time Frame
At Month 6
Title
Proportion of Participants who Achieve Fasting TG ≤ 750 mg/dL at 6 Months compared to placebo
Time Frame
At Month 6
Title
Adjudicated Acute Pancreatitis Event Rate during the Treatment Period Compared to Placebo, in Participants with ≥ 2 Events of Adjudicated Acute Pancreatitis in 5 Years prior to Enrollment
Time Frame
Week 1 through Week 53
Title
Adjudicated Acute Pancreatitis Event Rate during the Treatment Period Compared to Placebo
Time Frame
Week 1 through Week 53
Title
Change from Baseline in the Proportion of Participants who Achieve ≥ 70% Reduction in Fasting TG at 6 Months Compared to Placebo
Time Frame
Baseline and Month 6
Title
Proportion of Participants who Achieve Fasting TG ≤ 500 mg/dL at 6 Months Compared to Placebo
Time Frame
At Month 6
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Key Inclusion Criteria:
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
Key Exclusion Criteria:
Acute coronary syndrome within 6 months of Screening
Major surgery within 3 months of Screening
Have any other conditions, which, in the opinion of the Investigator would make the participant unsuitable for inclusion, or could interfere with participating in or completing the study
Facility Information:
Facility Name
Diabetes/Lipid Management & Research Center
City
Huntington Beach
State/Province
California
ZIP/Postal Code
92648
Country
United States
Facility Name
University of California, San Francisco (UCSF) - Medical Center
City
San Francisco
State/Province
California
ZIP/Postal Code
94143
Country
United States
Facility Name
Excel Medical Clinical Trials, LLC
City
Boca Raton
State/Province
Florida
ZIP/Postal Code
33434
Country
United States
Facility Name
NorthShore University Health System
City
Evanston
State/Province
Illinois
ZIP/Postal Code
60201
Country
United States
Facility Name
Advocate Health and Hospitals Corporation - Lutheran General Hospital
City
Park Ridge
State/Province
Illinois
ZIP/Postal Code
60068
Country
United States
Facility Name
Ascension St. Vincent Cardiovascular Research Institute
City
Indianapolis
State/Province
Indiana
ZIP/Postal Code
46290
Country
United States
Facility Name
University of Kansas Medical Center (KUMC)
City
Kansas City
State/Province
Kansas
ZIP/Postal Code
66160
Country
United States
Facility Name
University of Michigan- Endocrinology & Metabolism
City
Ann Arbor
State/Province
Michigan
ZIP/Postal Code
48105
Country
United States
Facility Name
New York University (NYU) Langone Medical Center
City
New York
State/Province
New York
ZIP/Postal Code
10016
Country
United States
Facility Name
Columbia University Medical Center
City
New York
State/Province
New York
ZIP/Postal Code
10032
Country
United States
Facility Name
Moses H. Cone Memorial Hospital
City
Greensboro
State/Province
North Carolina
ZIP/Postal Code
27401
Country
United States
Facility Name
University of Pennsylvania
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Facility Name
Vanderbilt University
City
Nashville
State/Province
Tennessee
ZIP/Postal Code
37232
Country
United States
Facility Name
University of Texas Southwestern
City
Dallas
State/Province
Texas
ZIP/Postal Code
75390
Country
United States
Facility Name
Baylor College of Medicine
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Facility Name
York Clinical Research LLC
City
Norfolk
State/Province
Virginia
ZIP/Postal Code
23510
Country
United States
Facility Name
West Virginia University Heart and Vascular Institute
City
Morgantown
State/Province
West Virginia
ZIP/Postal Code
26506
Country
United States
Facility Name
Ecogene-21
City
Chicoutimi
State/Province
Quebec
ZIP/Postal Code
G7H 7K9
Country
Canada
Facility Name
Nathalie Saint-Pierre
City
Montréal
State/Province
Quebec
ZIP/Postal Code
H2W 1R7
Country
Canada
Facility Name
Clinique des Maladies Lipidiques de Quebec Inc.
City
Québec
State/Province
Quebec
ZIP/Postal Code
G1V 4W2
Country
Canada
Facility Name
Institute de Recherches Cliniques de Montreal
City
Montréal
ZIP/Postal Code
H2W 1R7
Country
Canada
Facility Name
Hôpital Louis Pradel - HCL
City
Bron
ZIP/Postal Code
69677
Country
France
Facility Name
CHU Dijon - Bocage
City
Dijon
ZIP/Postal Code
21000
Country
France
Facility Name
Assistance Publique - Hopitaux de Marseille
City
Marseille
ZIP/Postal Code
13385
Country
France
Facility Name
Fejer Megyei Szent Gyorgy Egyetemi Oktato Korhaz
City
Székesfehérvár
ZIP/Postal Code
8000
Country
Hungary
Facility Name
Ziv Medical Center
City
Safed
ZIP/Postal Code
1311001
Country
Israel
Facility Name
ASST Grande Ospedale Metropolitano Niguarda
City
Milano
ZIP/Postal Code
20162
Country
Italy
Facility Name
Azienda Ospedaliera Universitaria Federico II
City
Napoli
ZIP/Postal Code
80131
Country
Italy
Facility Name
Azienda Ospedaliera Universitaria Policlinico Paolo Giaccone
City
Palermo
ZIP/Postal Code
90127
Country
Italy
Facility Name
Azienda Ospedaliero Universitaria Policlinico Umberto I
City
Roma
ZIP/Postal Code
00161
Country
Italy
Facility Name
Academic Medical Center - Department of Vascular Medicine
City
Amsterdam
ZIP/Postal Code
1105 AZ
Country
Netherlands
Facility Name
Erasmus MC
City
Rotterdam
ZIP/Postal Code
3015 GD
Country
Netherlands
Facility Name
Universitair Medisch Centrum Utrecht
City
Utrecht
ZIP/Postal Code
3584 CX
Country
Netherlands
Facility Name
The Lipid Clinic (Oslo University Hospital)
City
Oslo
ZIP/Postal Code
0372
Country
Norway
Facility Name
Centro Hospitalar de Lisboa Ocidental. E.P.E, - Hospital Santa Cruz
City
Carnaxide
ZIP/Postal Code
2790-134
Country
Portugal
Facility Name
Hospital da Senhora da Oliveira - Guimaraes
City
Creixomil
ZIP/Postal Code
4835-044
Country
Portugal
Facility Name
Centro Hospitalar de Lisboa Ocidental, EPE - Hospital Egas Moniz
City
Lisboa
ZIP/Postal Code
1349-019
Country
Portugal
Facility Name
Metabolicke centrum MUDr Katariny Raslovej s. r. o.
City
Bratislava
ZIP/Postal Code
83301
Country
Slovakia
Facility Name
Hospital Abente y Lago
City
A Coruña
ZIP/Postal Code
15001
Country
Spain
Facility Name
Hospital Clinic Barcelona
City
Barcelona
ZIP/Postal Code
08036
Country
Spain
Facility Name
Hospital Universitario 12 de Octubre
City
Madrid
ZIP/Postal Code
28041
Country
Spain
Facility Name
Hospital Universitario Virgen del Rocio
City
Sevilla
ZIP/Postal Code
41013
Country
Spain
Facility Name
Fundacio Pere Virgili
City
Tarragona
ZIP/Postal Code
43204
Country
Spain
Facility Name
Sahlgrenska University Hospital
City
Göteborg
ZIP/Postal Code
413 45
Country
Sweden
Facility Name
Karolinska University Hospital
City
Solna
ZIP/Postal Code
171 76
Country
Sweden
Facility Name
The Royal Free Hospital
City
London
ZIP/Postal Code
NW3 2QG
Country
United Kingdom
Facility Name
St. Thomas' Hospital
City
London
ZIP/Postal Code
SE1 7EH
Country
United Kingdom
Facility Name
Manchester University NHS Foundation Trust (MFT)
City
Manchester
ZIP/Postal Code
MI39WL
Country
United Kingdom
Facility Name
Sandwell General Hospital
City
West Bromwich
ZIP/Postal Code
B71 4HJ
Country
United Kingdom
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) Administered to Patients With Familial Chylomicronemia Syndrome (FCS)
We'll reach out to this number within 24 hrs