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A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) Administered to Patients With Familial Chylomicronemia Syndrome (FCS)

NCT ID: NCT04568434

Last Updated: 2025-03-06

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

66 participants

Study Classification

INTERVENTIONAL

Study Start Date

2020-11-18

Study Completion Date

2023-10-17

Brief Summary

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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.

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Detailed Description

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This was a multi-center, double-blind, Phase 3 study in up to 60 patients with FCS. Participants were 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 was approximately 74 weeks, which included 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 had the option to enroll in the Open-label Extension (OLE) Study ISIS 678354-CS13 (NCT05130450).

Conditions

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Familial Chylomicronemia Syndrome

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

DOUBLE

Participants Investigators

Study Groups

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Placebo

Participants received olezarsen-matching placebo, once every 4 weeks by subcutaneous (SC) injection, during Weeks 1 to 49 of the 53-week treatment period.

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Olezarsen-matching placebo was administered by SC injection.

Olezarsen 50 mg

Participants received olezarsen, 50 milligrams (mg), once every 4 weeks by SC injection, during Weeks 1 to 49 of the 53-week treatment period.

Group Type EXPERIMENTAL

Olezarsen

Intervention Type DRUG

Olezarsen was administered by SC injection.

Olezarsen 80 mg

Participants received olezarsen 80 mg, once every 4 weeks by SC injection, during Weeks 1 to 49 of the 53-week treatment period.

Group Type EXPERIMENTAL

Olezarsen

Intervention Type DRUG

Olezarsen was administered by SC injection.

Interventions

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Olezarsen

Olezarsen was administered by SC injection.

Intervention Type DRUG

Placebo

Olezarsen-matching placebo was administered by SC injection.

Intervention Type DRUG

Other Intervention Names

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ISIS 678354 AKCEA-APOCIII-LRx

Eligibility Criteria

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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

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
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Ionis Pharmaceuticals, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Diabetes/Lipid Management & Research Center

Huntington Beach, California, United States

Site Status

University of California, San Francisco (UCSF) - Medical Center

San Francisco, California, United States

Site Status

Excel Medical Clinical Trials, LLC

Boca Raton, Florida, United States

Site Status

NorthShore University Health System

Evanston, Illinois, United States

Site Status

Advocate Health and Hospitals Corporation - Lutheran General Hospital

Park Ridge, Illinois, United States

Site Status

Ascension St. Vincent Cardiovascular Research Institute

Indianapolis, Indiana, United States

Site Status

University of Kansas Medical Center (KUMC)

Kansas City, Kansas, United States

Site Status

University of Michigan- Endocrinology & Metabolism

Ann Arbor, Michigan, United States

Site Status

New York University (NYU) Langone Medical Center

New York, New York, United States

Site Status

Columbia University Medical Center

New York, New York, United States

Site Status

Moses H. Cone Memorial Hospital

Greensboro, North Carolina, United States

Site Status

University of Pennsylvania

Philadelphia, Pennsylvania, United States

Site Status

Vanderbilt University

Nashville, Tennessee, United States

Site Status

University of Texas Southwestern

Dallas, Texas, United States

Site Status

Baylor College of Medicine

Houston, Texas, United States

Site Status

York Clinical Research LLC

Norfolk, Virginia, United States

Site Status

West Virginia University Heart and Vascular Institute

Morgantown, West Virginia, United States

Site Status

Ecogene-21

Chicoutimi, Quebec, Canada

Site Status

Nathalie Saint-Pierre

Montreal, Quebec, Canada

Site Status

Clinique des Maladies Lipidiques de Quebec Inc.

Québec, Quebec, Canada

Site Status

Institute de Recherches Cliniques de Montreal

Montreal, , Canada

Site Status

Hôpital Louis Pradel - HCL

Bron, , France

Site Status

CHU Dijon - Bocage

Dijon, , France

Site Status

Assistance Publique - Hopitaux de Marseille

Marseille, , France

Site Status

ASST Grande Ospedale Metropolitano Niguarda

Milan, , Italy

Site Status

Azienda Ospedaliera Universitaria Federico II

Napoli, , Italy

Site Status

Azienda Ospedaliera Universitaria Policlinico Paolo Giaccone

Palermo, , Italy

Site Status

Azienda Ospedaliero Universitaria Policlinico Umberto I

Roma, , Italy

Site Status

Academic Medical Center - Department of Vascular Medicine

Amsterdam, , Netherlands

Site Status

Erasmus MC

Rotterdam, , Netherlands

Site Status

Universitair Medisch Centrum Utrecht

Utrecht, , Netherlands

Site Status

The Lipid Clinic (Oslo University Hospital)

Oslo, , Norway

Site Status

Centro Hospitalar de Lisboa Ocidental. E.P.E, - Hospital Santa Cruz

Carnaxide, , Portugal

Site Status

Hospital da Senhora da Oliveira - Guimaraes

Creixomil, , Portugal

Site Status

Centro Hospitalar de Lisboa Ocidental, EPE - Hospital Egas Moniz

Lisbon, , Portugal

Site Status

Metabolicke centrum MUDr Katariny Raslovej s. r. o.

Bratislava, , Slovakia

Site Status

Hospital Abente y Lago

A Coruña, , Spain

Site Status

Hospital Clinic Barcelona

Barcelona, , Spain

Site Status

Hospital Universitario 12 de Octubre

Madrid, , Spain

Site Status

Hospital Universitario Virgen del Rocio

Seville, , Spain

Site Status

Fundacio Pere Virgili

Tarragona, , Spain

Site Status

Sahlgrenska University Hospital

Gothenburg, , Sweden

Site Status

Karolinska University Hospital

Solna, , Sweden

Site Status

The Royal Free Hospital

London, , United Kingdom

Site Status

St. Thomas' Hospital

London, , United Kingdom

Site Status

Manchester University NHS Foundation Trust (MFT)

Manchester, , United Kingdom

Site Status

Sandwell General Hospital

West Bromwich, , United Kingdom

Site Status

Countries

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United States Canada France Italy Netherlands Norway Portugal Slovakia Spain Sweden United Kingdom

References

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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.

Reference Type RESULT
PMID: 38587247 (View on PubMed)

Provided Documents

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Document Type: Study Protocol and Statistical Analysis Plan

View Document

Other Identifiers

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2020-002536-67

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

ISIS 678354-CS3

Identifier Type: -

Identifier Source: org_study_id

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