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A Study of AKCEA-APOCIII-LRx Administered to Patients With Familial Chylomicronemia Syndrome (FCS) (BALANCE)

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: NCT04568434
Recruitment Status : Recruiting
First Posted : September 29, 2020
Last Update Posted : November 18, 2021
Sponsor:
Collaborator:
Akcea Therapeutics
Information provided by (Responsible Party):
Ionis Pharmaceuticals, Inc.

Brief Summary:
The purpose of the study is to evaluate the efficacy of AKCEA-APOCIII-LRx as compared to placebo on the percent change in fasting triglycerides (TG) from baseline.

Condition or disease Intervention/treatment Phase
Familial Chylomicronemia Syndrome Drug: AKCEA-APOCIII-LRx Drug: Placebo Phase 3

Detailed Description:
This is a multi-center, double-blind, Phase 3 study in up to 60 patients with FCS. Participants will be randomized in a 2:1 ratio to receive AKCEA-APOCIII-LRx or matching placebo in a 53-week treatment period. The length of participation in the study is approximately 74 weeks, which includes an up to 8-week screening period, a 53-week treatment period, and a 13-week post-treatment evaluation period. Following the treatment period, eligible patients may have the option of enrolling in an open label extension study.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 60 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Official Title: A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Study of AKCEA-APOCIII-LRx Administered Subcutaneously to Patients With Familial Chylomicronemia Syndrome (FCS)
Actual Study Start Date : November 18, 2020
Estimated Primary Completion Date : March 2023
Estimated Study Completion Date : June 2023


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

Placebo Comparator: Placebo
AKCEA-APOCIII-LRx-matching placebo will be administered once every 4 weeks by SC injection from Week 1 through Week 49.
Drug: Placebo
AKCEA-APOCIII-LRx-matching placebo will be administered by SC injection.




Primary Outcome Measures :
  1. Percent Change from Baseline in Fasting TG at 6 Months (average of Weeks 23, 25, and 27) compared to placebo [ Time Frame: Baseline and Month 6 ]

Secondary Outcome Measures :
  1. Percent Change from Baseline in Fasting TG at 12 months (average of Weeks 51 and 53) compared to placebo [ Time Frame: Baseline and Month 12 ]
  2. Change from Baseline in the Proportion of Participants who Achieve ≥ 40% Reduction in Fasting TG at 6 Months Compared to Placebo [ Time Frame: Baseline and Month 6 ]
  3. Percent Change form Baseline in Fasting Apolipoprotein B-48 (apoB-48) at 6 Months Compared to Placebo [ Time Frame: At Month 6 ]
  4. Proportion of Participants who Achieve Fasting TG ≤ 750 mg/dL at 6 Months compared to placebo [ Time Frame: At Month 6 ]
  5. Adjudicated Acute Pancreatitis Event Rate during the Treatment Period Compared to Placebo, in Participants with ≥ 2 Events of Adjudicated Acute Pancreatitis in 5 Years prior to Enrollment [ Time Frame: Week 1 through Week 53 ]
  6. Adjudicated Acute Pancreatitis Event Rate during the Treatment Period Compared to Placebo [ Time Frame: Week 1 through Week 53 ]
  7. Change from Baseline in the Proportion of Participants who Achieve ≥ 70% Reduction in Fasting TG at 6 Months Compared to Placebo [ Time Frame: Baseline and Month 6 ]
  8. Proportion of Participants who Achieve Fasting TG ≤ 500 mg/dL at 6 Months Compared to Placebo [ Time Frame: At Month 6 ]


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

Key Inclusion Criteria:

  • A diagnosis of genetically confirmed Familial Chylomicronemia Syndrome (type 1 Hyperlipoproteinemia)
  • Fasting TG ≥ 880 mg/dL (10 millimoles per liter (mmol/L)) at Screening
  • History of pancreatitis
  • Stable doses of statins, omega-3 fatty acids, fibrates, or other lipid-lowering medications are allowed

Key Exclusion Criteria:

  • Acute coronary syndrome within 6 months of Screening
  • Major surgery within 3 months of Screening
  • Have any other conditions, which, in the opinion of the Investigator would make the participant unsuitable for inclusion, or could interfere with participating in or completing the study

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): NCT04568434


Contacts
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Contact: Ionis Pharmaceuticals (844) 414-1620 ionisNCT04568434study@clinicaltrialmedia.com

Locations
Show Show 36 study locations
Sponsors and Collaborators
Ionis Pharmaceuticals, Inc.
Akcea Therapeutics
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Responsible Party: Ionis Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT04568434    
Other Study ID Numbers: ISIS 678354-CS3
2020-002536-67 ( EudraCT Number )
First Posted: September 29, 2020    Key Record Dates
Last Update Posted: November 18, 2021
Last Verified: September 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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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:
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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