NCT05130450

Brief Summary

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
60

participants targeted

Target at below P25 for phase_3

Timeline
22mo left

Started Nov 2021

Longer than P75 for phase_3

Geographic Reach
11 countries

27 active sites

Status
active not recruiting

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

Study Progress72%
Nov 2021Feb 2028

First Submitted

Initial submission to the registry

November 11, 2021

Completed
7 days until next milestone

Study Start

First participant enrolled

November 18, 2021

Completed
5 days until next milestone

First Posted

Study publicly available on registry

November 23, 2021

Completed
6.2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

February 1, 2028

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

February 1, 2028

Last Updated

December 12, 2025

Status Verified

December 1, 2025

Enrollment Period

6.2 years

First QC Date

November 11, 2021

Last Update Submit

December 5, 2025

Conditions

Familial Chylomicronemia Syndrome

Keywords

FCS

Outcome Measures

Primary Outcomes (1)

  • Percent Change From Baseline in Fasting TG at 6 Months (Average of Weeks 23, 25, and 27) Compared to Baseline

    Baseline and 6 months

Secondary Outcomes (18)

  • Percent Change From Baseline in Fasting TG at 12 Months (Average of Weeks 51 and 53) at 24 Months (Average of Week 103 and Week 105) and 36 Months (Average of Week 155 and Week 157)

    Baseline and 36 months

  • Percentage of Participants Who Achieve ≥ 40% Reduction in Fasting TG From Baseline at 6 Months

    At 6 months

  • Percentage of Participants Who Achieve ≥ 40% Reduction in Fasting TG From Baseline at 12, 24, 36 Months

    At 12, 24, 36 months

  • Percent Change in Fasting apoC-III from Baseline at Month 6

    Baseline and 6 months

  • Percent Change in Fasting apoC-III from Month 12, 24, 36

    At 12, 24, 36 months

  • +13 more secondary outcomes

Study Arms (1)

Olezarsen

EXPERIMENTAL

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

Drug: Olezarsen

Interventions

OlezarsenDRUG

Olezarsen will be administered by SC injection.

Also known as: ISIS 678354, AKCEA -APOCIII-LRx
Olezarsen

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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.

You may not qualify if:

  • 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).

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (27)

Diabetes/Lipid Management & Research Center

Huntington Beach, California, 92648, United States

Location

Excel Medical Clinical Trials, LLC

Boca Raton, Florida, 33434, United States

Location

Department of Pharmacy

Park Ridge, Illinois, 60068, United States

Location

University of Kansas Medical Center (KUMC)

Kansas City, Kansas, 66160, United States

Location

CTSI Investigational Pharmacy

New York, New York, 10016, United States

Location

Milstein Hospital

New York, New York, 10032, United States

Location

IDS Central

Philadelphia, Pennsylvania, 19104, United States

Location

Ecogene-21

Chicoutimi, Quebec, G7H 7K9, Canada

Location

Institute de Recherches Cliniques de Montreal

Montreal, Quebec, H2W 1R7, Canada

Location

Nathalie Saint-Pierre

Montreal, Quebec, H2W 1R7, Canada

Location

Clinique des Maladies Lipidiques de Quebec Inc.

Québec, Quebec, G1V 4W2, Canada

Location

Groupement Hospitalier Est- Pharmacie Secteur Essais Clinique

Bron, 69677, France

Location

Hôpital Bicêtre

Le Kremlin-Bicêtre, 94270, France

Location

Pharmacie Hopital de la Conception

Marseille, 05 13385, France

Location

Via Sergio Pansini 5

Naples, 80131, Italy

Location

UOC di Farmacia AOUP PAOLO GIACCONE

Palermo, 90127, Italy

Location

Azienda Ospedaliero Universitaria Policlinico Umberto I

Rome, 00161, Italy

Location

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

Amsterdam, Noor-Holland, 1105 AZ, Netherlands

Location

Oslo Hospital Pharmacy Rikshospitalet

Oslo, 0372, Norway

Location

Hospital da Senhora da Oliveira Guimaraes

Creixomil, 4835-044, Portugal

Location

Dra Margarida Falcao Centro Hospitalar Lisboa Ocidental Hospital Egas Moniz

Lisbon, 1340-019, Portugal

Location

Metabolicke centrum MU

Bratislava, 83101, Slovakia

Location

Hospital Clínic Barcelona C/ Villarroel

Barcelona, 08036, Spain

Location

Hospital Universitario 12 de Octubre

Madrid, 28041, Spain

Location

Hospital Universitario Virgen del Rocío

Seville, 41013, Spain

Location

Apokteket AB

Malmo, 211 24, Sweden

Location

Royal Manchester Children's Hospital

Manchester, M13 9WL, United Kingdom

Location

MeSH Terms

Conditions

Familial hyperchylomicronemia syndrome

Interventions

olezarsen

Study Design

Study Type
interventional
Phase
phase 3
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR
Expanded Access
Yes

Study Record Dates

First Submitted

November 11, 2021

First Posted

November 23, 2021

Study Start

November 18, 2021

Primary Completion (Estimated)

February 1, 2028

Study Completion (Estimated)

February 1, 2028

Last Updated

December 12, 2025

Record last verified: 2025-12

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/.

More information

Locations

IT(3)US(7)PT(2)NL(1)SE(1)CA(4)NO(1)SL(1)GB(1)ES(3)FR(3)