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A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) in Participants With Familial Chylomicronemia Syndrome (FCS)

NCT ID: NCT05130450

Last Updated: 2025-12-12

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE3

Total Enrollment

60 participants

Study Classification

INTERVENTIONAL

Study Start Date

2021-11-18

Study Completion Date

2028-02-29

Brief Summary

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The purpose of this study is to evaluate the effect of olezarsen (formerly known as AKCEA-APOCIII-LRx) on the percent change in fasting triglycerides (TG) from baseline.

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Detailed Description

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This is a multi-center, open-label extension (OLE) study of up to 60 participants with FCS rolling-over from Study ISIS 678354-CS3 (NCT04568434). Participants will receive olezarsen during a 157-week treatment period, followed by a 13-week post-treatment follow-up period. The length of participation in this study is approximately 201 weeks, which includes an up to 31-day qualification period, a 157-week treatment period, and a 13-week post-treatment evaluation period.

Treatment has been extended to obtain additional safety assessments and efficacy data and to provide patients with continued access to ISIS 678354 until the drug may be available commercially.

Conditions

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Familial Chylomicronemia Syndrome

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Olezarsen

Olezarsen will be administered once every 4 weeks by subcutaneous (SC) injection from Week 1 through Week 153.

Group Type EXPERIMENTAL

Olezarsen

Intervention Type DRUG

Olezarsen will be administered by SC injection.

Interventions

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Olezarsen

Olezarsen will be 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

• Satisfactory completion of treatment with olezarsen in the index study (ISIS 678354-CS3, last dose as scheduled at Week 49) with an acceptable safety profile, per Investigator judgement.

Exclusion Criteria

• Have any new condition or worsening of existing condition which in the opinion of the Investigator would make the participant unsuitable for enrollment, or could interfere with the patient participating in or completing the study, including need for treatment with medications disallowed in the index study (ISIS 678354-CS3).
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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Oslo Hospital Pharmacy Rikshospitalet

Oslo, , Norway

Site Status

Diabetes/Lipid Management & Research Center

Huntington Beach, California, United States

Site Status

Excel Medical Clinical Trials, LLC

Boca Raton, Florida, United States

Site Status

Department of Pharmacy

Park Ridge, Illinois, United States

Site Status

University of Kansas Medical Center (KUMC)

Kansas City, Kansas, United States

Site Status

CTSI Investigational Pharmacy

New York, New York, United States

Site Status

Milstein Hospital

New York, New York, United States

Site Status

IDS Central

Philadelphia, Pennsylvania, United States

Site Status

Ecogene-21

Chicoutimi, Quebec, Canada

Site Status

Institute de Recherches Cliniques de Montreal

Montreal, Quebec, Canada

Site Status

Nathalie Saint-Pierre

Montreal, Quebec, Canada

Site Status

Clinique des Maladies Lipidiques de Quebec Inc.

Québec, Quebec, Canada

Site Status

Groupement Hospitalier Est- Pharmacie Secteur Essais Clinique

Bron, , France

Site Status

Hôpital Bicêtre

Le Kremlin-Bicêtre, , France

Site Status

Pharmacie Hopital de la Conception

Marseille, , France

Site Status

Via Sergio Pansini 5

Napoli, , Italy

Site Status

UOC di Farmacia AOUP PAOLO GIACCONE

Palermo, , Italy

Site Status

Azienda Ospedaliero Universitaria Policlinico Umberto I

Rome, , Italy

Site Status

Academisch Medisch Centrum Goederenontvangst Apotheek t.a.v. Kenniscentrum

Amsterdam, Noor-Holland, Netherlands

Site Status

Hospital da Senhora da Oliveira Guimaraes

Creixomil, , Portugal

Site Status

Dra Margarida Falcao Centro Hospitalar Lisboa Ocidental Hospital Egas Moniz

Lisbon, , Portugal

Site Status

Metabolicke centrum MU

Bratislava, , Slovakia

Site Status

Hospital Clínic Barcelona C/ Villarroel

Barcelona, , Spain

Site Status

Hospital Universitario 12 de Octubre

Madrid, , Spain

Site Status

Hospital Universitario Virgen del Rocío

Seville, , Spain

Site Status

Apokteket AB

Malmo, , Sweden

Site Status

Royal Manchester Children's Hospital

Manchester, , United Kingdom

Site Status

Countries

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

Other Identifiers

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2021-003280-95

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

ISIS 678354-CS13

Identifier Type: -

Identifier Source: org_study_id

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