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

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ClinicalTrials.gov Identifier: NCT04568434
Recruitment Status : Recruiting
First Posted : September 29, 2020
Last Update Posted : May 14, 2021
Sponsor:
Collaborator:
Akcea Therapeutics
Information provided by (Responsible Party):
Ionis Pharmaceuticals, Inc.

Tracking Information
First Submitted Date  ICMJE September 23, 2020
First Posted Date  ICMJE September 29, 2020
Last Update Posted Date May 14, 2021
Actual Study Start Date  ICMJE November 18, 2020
Estimated Primary Completion Date March 2023   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: September 23, 2020)
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 ]
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: September 23, 2020)
  • 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 ]
  • 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 ]
  • Percent Change form Baseline in Fasting Apolipoprotein B-48 (apoB-48) at 6 Months Compared to Placebo [ Time Frame: At Month 6 ]
  • Proportion of Participants who Achieve Fasting TG ≤ 750 mg/dL at 6 Months compared to placebo [ Time Frame: At Month 6 ]
  • 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 ]
  • Adjudicated Acute Pancreatitis Event Rate during the Treatment Period Compared to Placebo [ Time Frame: Week 1 through Week 53 ]
  • 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 ]
  • Proportion of Participants who Achieve Fasting TG ≤ 500 mg/dL at 6 Months Compared to Placebo [ Time Frame: At Month 6 ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Study of AKCEA-APOCIII-LRx Administered to Patients With Familial Chylomicronemia Syndrome (FCS)
Official Title  ICMJE A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Study of AKCEA-APOCIII-LRx Administered Subcutaneously to Patients With Familial Chylomicronemia Syndrome (FCS)
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.
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.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Condition  ICMJE Familial Chylomicronemia Syndrome
Intervention  ICMJE
  • Drug: AKCEA-APOCIII-LRx
    AKCEA-APOCIII-LRx will be administered by SC injection.
    Other Name: ISIS 678354
  • Drug: Placebo
    AKCEA-APOCIII-LRx-matching placebo will be administered by SC injection.
Study Arms  ICMJE
  • Experimental: AKCEA-APOCIII-LRx
    AKCEA-APOCIII-LRx will be administered once every 4 weeks by subcutaneous (SC) injection from Week 1 through Week 49.
    Intervention: Drug: AKCEA-APOCIII-LRx
  • Placebo Comparator: Placebo
    AKCEA-APOCIII-LRx-matching placebo will be administered once every 4 weeks by SC injection from Week 1 through Week 49.
    Intervention: Drug: Placebo
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: September 23, 2020)
60
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE June 2023
Estimated Primary Completion Date March 2023   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Ionis Pharmaceuticals 800-679-4747 patients@ionisph.com
Listed Location Countries  ICMJE Canada,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04568434
Other Study ID Numbers  ICMJE ISIS 678354-CS3
2020-002536-67 ( EudraCT Number )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
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
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Ionis Pharmaceuticals, Inc.
Study Sponsor  ICMJE Ionis Pharmaceuticals, Inc.
Collaborators  ICMJE Akcea Therapeutics
Investigators  ICMJE Not Provided
PRS Account Ionis Pharmaceuticals, Inc.
Verification Date May 2021

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP