A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRX) Administered to Adults With Familial Chylomicronemia Syndrome (FCS) Previously Treated With Volanesorsen

Brief Title

A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRX) Administered to Adults With Familial Chylomicronemia Syndrome (FCS) Previously Treated With Volanesorsen

Official Title

An Open-Label Safety Study of AKCEA-APOCIII-LRX Administered Subcutaneously to Patients With Familial Chylomicronemia Syndrome (FCS) Previously Treated With Volanesorsen (ISIS 304801)

Brief Summary

      The purpose of the study is to evaluate the safety, tolerability, pharmacokinetic (PK) and
      pharmacodynamic (PD) effects of olezarsen (formerly known as AKCEA -APOCIII-LRX) in
      participants with FCS previously treated with volanesorsen.
    

Detailed Description

      This is a Phase 3, multi-center, open-label safety study of up to 30 participants with FCS,
      previously treated with volanesorsen. The study consists of 3 periods: 1) Screening Period:
      Week -4 to Week -1 (up to 4 weeks); 2) Treatment Period up to Week 53; and 3) Post-Treatment
      Follow-up Period: Week 54 to Week 66 (13 weeks). Participants enrolled will receive olezarsen
      every 4 weeks during the 53-week Treatment Period.
    

Study Phase

Phase 3

Study Type

Interventional


Primary Outcome

Proportion of Participants With Decrease in Platelet Count by >30% or >50%, or With Platelet Count Value <50,000/square millimeter (mm^3)

Secondary Outcome

 Trough (Pre-Dose) Plasma Concentration of Olezarsen

Condition

Familial Chylomicronemia Syndrome

Intervention

Olezarsen

Study Arms / Comparison Groups

 Olezarsen
Description:  Olezarsen will be administered once every 4 weeks by subcutaneous (SC) injection for up to 49 weeks.

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

30

Start Date

January 2022

Completion Date

March 2025

Primary Completion Date

December 2024

Eligibility Criteria

        Inclusion Criteria

          1. Participants with FCS (clinical or genetic diagnosis) currently on or previously
             treated with volanesorsen (ISIS 304801)

             o Study participants in countries where Waylivra® is commercially approved and
             available for participants should not be deprived of the treatment option with
             Waylivra®. Participation in this study for such participants will only be allowed when
             Waylivra® was discontinued due to AEs

          2. The following concomitant medications will be allowed if dosing regimen is expected to
             remain constant through the end of the study (occasional or intermittent use of
             over-the-counter (OTC) medications will be allowed at Investigator's discretion):

               -  Statins, omega-3 fatty acids (prescription and OTC), fibrates, or other
                  lipid-lowering medications. Participants taking OTC omega-3 fatty acids should
                  make every effort to remain on the same brand through the end of the study

               -  Antidiabetic medications

               -  Oral anticoagulants (e.g., dabigatran, rivaroxaban, or apixaban, and warfarin
                  with regular clinical monitoring)

               -  Tamoxifen, estrogens or progestins

        Exclusion Criteria:

          1. Treatment with another investigational drug (non-oligonucleotide), biological agent,
             or device within 4 weeks of Screening, or 5 half-lives of investigational agent,
             whichever is longer

          2. Concomitant medication/procedure restrictions:

               1. Systemic corticosteroids or anabolic steroids within 6 weeks prior to Screening
                  and during the study unless approved by the Sponsor Medical Monitor

               2. Plasma apheresis within 4 weeks prior to Screening or planned during the study
      

Gender

All

Ages

18 Years - N/A

Accepts Healthy Volunteers

No

Contacts

, 800-679-4747, [email protected]



Administrative Informations


NCT ID

NCT05185843

Organization ID

ISIS 678354-CS7

Secondary IDs

2021-003635-29

Responsible Party

Sponsor

Study Sponsor

Ionis Pharmaceuticals, Inc.


Study Sponsor

, , 


Verification Date

December 2021