A Phase 3 Global Double-Blind Randomized Placebo-Controlled Study to Evaluate the Efficacy and Safety of ION-682884 in Patients with Transthyretin-Mediated Amyloid Cardiomyopathy

Are you eligible to participate in this study?

You may be eligible for this study if you meet the following criteria:

  • Conditions:
    Transthyretin Amyloid Cardiomyopathy
  • Age: Between 18 years - 90 years
  • Gender: Male or Female
  • Other Inclusion Criteria:
    1. Participants able to provide written informed consent
    2. Participants who are not pregnant or breastfeeding
    3. Participants must agree to use acceptable contraceptives as per the protocol
    4. Those who are willing to be genetically tested for mutations in the TTR gene
    5. Participants who have medical history of Heart failure secondary to hereditary or wild-type ATTR-CM
    6. New York Heart Association (NYHA) class I-III
    7. If on medical treatment for Heart Failure on stable dosage regimen for 2 weeks prior to randomization
    8. Willingness to adhere to vitamin A supplementation per protocol

You may not be eligible for this study if the following are true:

    1. Acute coronary syndrome, unstable angina, stroke, TIA, coronary revascularization, cardiac device implantation, cardiac valve repair, or major surgery within 3 months of Screening
    2. Those with hospitalization or urgent visit to ER/ED for worsening of HF
    3. Participants with uncontrolled hypertension
    4. Uncontrolled clinically significant cardiac arrhythmia
    5. Severe uncorrected cardiac valvular disease
    6. Cardiomyopathy not primarily caused by ATTR-CM
    7. Screening laboratory results as the protocol
    8. Active infection requiring systemic antiviral or antimicrobial therapy
    9. Known history of or positive test for human immunodeficiency virus (HIV), hepatitis C or hepatitis B
    10. History of bleeding, diathesis or coagulopathy
    11. If receiving oral anticoagulants, the dose must have been stable for 4 weeks prior to the first dose
    12. Those with malignancy within 5 years
    13. Prior liver or heart transplant, and/or Left Ventricular Assist Device (LVAD) or anticipated liver transplant or LVAD within 1 year after randomization
    14. Participants with known Light chain/Primary Amyloidosis or known leptomeningeal amyloidosis or known history of multiple myeloma
    15. Participants with anticipated survival < 2 years
    16. Treatment with another investigational drug and/or biological agent within 1 month of Screening, or 5 half-lives of investigational agent
    17. Current or previous treatment with TegsediTM (inotersen) or OnpattroTM (patisiran) or other oligonucleotide or RNA therapeutic (including siRNA)
    18. Current treatment with diflunisal, doxycycline, and/or calcium-channel blocker. Patients receiving any of these agents must respect a wash-out period of 14 days before randomization

If you are registered as a volunteer, please log in to contact the study team/express interest in this study.