Olezarsen Early Access Program for Patients With Familial Chylomicronemia Syndrome (FCS)

December 23, 2024 updated by: Ionis Pharmaceuticals, Inc.

Olezarsen (ISIS 678354) Early Access Program for Patients With Familial Chylomicronemia Syndrome (FCS)

The purpose of the Expanded Access Program is to provide pre-approval access of olezarsen to eligible patients with Familial Chylomicronemia Syndrome (FCS).

Study Overview

Status

Approved for marketing

Conditions

Intervention / Treatment

Detailed Description

The Expanded Access Program (EAP) is intended to provide pre-approval access to olezarsen for eligible patients with FCS who have limited or no available treatment options. This program is open in the United States and operates under the individual patient (also referred to as single patient) IND expanded access route in which the patient's treating physician serves as the sponsor. Expanded access requests must be from the patient's treating physicians and submitted according to the instructions at https://www.ionispharma.com/patients/expanded-access-policy/

Study Type

Expanded Access

Expanded Access Type

  • Individual Patients

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • California
      • Carlsbad, California, United States, 92010
        • Expanded Access Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

N/A

Description

Inclusion Criteria:

  • Has a diagnosis of FCS as determined by the sponsoring physician. Ionis will review each application to determine eligibility based on documentation of validated genetic or clinical diagnosis.

    o Documented loss of function mutations (homozygous, compound / double heterozygous) in genes such as LPL, GPIHBP1, APOA5, APOC2 or LMF1) or clinically validated diagnosis of FCS.

  • Resides in and is a resident of the United States.
  • Willing to follow a diet comprising ≤20 g fat per day.

Exclusion Criteria:

  • Has any new or worsening of existing conditions which, in the opinion of the physician, would make the patient unsuitable for treatment with olezarsen.
  • Olezarsen naïve patients with baseline platelet count <100x109/L at qualification.
  • Estimated GFR (eGFR) <30 mL/min/1.73 m2.
  • Secondary factors are the cause of triglyceride elevations.
  • Is currently hospitalized in an acute emergency setting.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Ionis Pharmaceuticals, Inc.

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

First Submitted

April 6, 2024

First Submitted That Met QC Criteria

April 6, 2024

First Posted (Actual)

April 11, 2024

Study Record Updates

Last Update Posted (Actual)

December 27, 2024

Last Update Submitted That Met QC Criteria

December 23, 2024

Last Verified

December 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ISIS 678354

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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