Don't get left behind! The modernized ClinicalTrials.gov is coming. Check it out now.
Say goodbye to ClinicalTrials.gov!
The new site is coming soon - go to the modernized ClinicalTrials.gov
Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05130450
Recruitment Status : Recruiting
First Posted : November 23, 2021
Last Update Posted : May 3, 2023
Sponsor:
Information provided by (Responsible Party):
Ionis Pharmaceuticals, Inc.

Study Description
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information
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.

Condition or disease Intervention/treatment Phase
Familial Chylomicronemia Syndrome Drug: Olezarsen Phase 3

Detailed Description:
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 53-week treatment period, followed by a 13-week post-treatment follow-up period. The length of participation in this study is approximately 70 weeks, which includes an up to 31-day qualification period, a 53-week treatment period, and a 13-week post-treatment evaluation period.
Study Design
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 60 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-Label Extension Study of AKCEA-APOCIII-LRx Administered Subcutaneously to Patients With Familial Chylomicronemia Syndrome (FCS)
Actual Study Start Date : November 18, 2021
Estimated Primary Completion Date : January 2025
Estimated Study Completion Date : April 2025

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Family Issues

Arms and Interventions
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Arm Intervention/treatment
Experimental: Olezarsen
Olezarsen will be administered once every 4 weeks by subcutaneous (SC) injection from Week 1 through Week 49.
 Drug: Olezarsen
Olezarsen will be administered by SC injection.
Other Names:
  • ISIS 678354
  • AKCEA -APOCIII-LRx


Outcome Measures
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Primary Outcome Measures :
  1. Percent Change From Baseline in Fasting TG at 6 Months (Average of Weeks 23, 25, and 27) Compared to Baseline [ Time Frame: Baseline and 6 months ]

Secondary Outcome Measures :
  1. Percent Change From Baseline in Fasting TG at 12 Months (Average of Weeks 51 and 53) [ Time Frame: Baseline and 12 months ]
  2. Percentage of Participants Who Achieve ≥ 40% Reduction in Fasting TG From Baseline at 6 Months [ Time Frame: At 6 months ]
  3. Percentage of Participants Who Achieve ≥ 40% Reduction in Fasting TG From Baseline at 12 Months [ Time Frame: At 12 months ]
  4. Percent Change From Baseline in Fasting Apolipoprotein 48 (apoB48) at 6 Months [ Time Frame: At 6 months ]
  5. Percent Change From Baseline in Fasting apoB48 at 12 Months [ Time Frame: At 12 months ]
  6. Percentage of Participants Who Achieve Fasting TG ≤ 750 milligrams per deciliter (mg/dL) at 6 Months [ Time Frame: At 6 months ]
  7. Percentage of Participants Who Achieve Fasting TG ≤ 750 mg/dL at 12 months [ Time Frame: At 12 months ]
  8. Adjudicated Acute Pancreatitis Event Rate During the Treatment Period in Participants With ≥ 2 Events of Adjudicated Acute Pancreatitis in 5 Years Prior to Treatment With Olezarsen [ Time Frame: Week 1 through Week 53 ]
  9. Adjudicated Acute Pancreatitis Event Rate During the Treatment Period [ Time Frame: Week 1 through Week 53 ]
  10. Percentage of Participants Who Achieve ≥ 70% Reduction in Fasting TG From Baseline at 6 Months [ Time Frame: At 6 months ]
  11. Percentage of Participants Who Achieve ≥ 70% Reduction in Fasting TG From Baseline at 12 Months [ Time Frame: At 12 months ]
  12. Percentage of Participants Who Achieve Fasting TG ≤ 500 mg/dL at 6 Months [ Time Frame: At 6 months ]
  13. Percentage of Participants Who Achieve Fasting TG ≤ 500 mg/dL at 12 Months [ Time Frame: At 12 months ]

Eligibility Criteria
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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).

Contacts and Locations
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05130450


Contacts
Layout table for location contacts
Contact: Ionis Pharmaceuticals (844) 621-1320 ionisFCS2study@clinicaltrialmedia.com

Locations
Show Show 22 study locations
Sponsors and Collaborators
Ionis Pharmaceuticals, Inc.
More Information
Go to 
Top of Page Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information
Layout table for additonal information
Responsible Party: Ionis Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT05130450    
Other Study ID Numbers: ISIS 678354-CS13
2021-003280-95 ( EudraCT Number )
First Posted: November 23, 2021    Key Record Dates
Last Update Posted: May 3, 2023
Last Verified: May 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: Yes
Keywords provided by Ionis Pharmaceuticals, Inc.:
FCS
Additional relevant MeSH terms:
Layout table for MeSH terms
Hyperlipoproteinemia Type I
Syndrome
Disease
Pathologic Processes
Lipid Metabolism, Inborn Errors
Metabolism, Inborn Errors
 Genetic Diseases, Inborn
Hyperlipoproteinemias
Hyperlipidemias
Dyslipidemias
Lipid Metabolism Disorders
Metabolic Diseases