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  • MAGNITUDE: A Phase 3, Multinational, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of NTLA-2001 in Participants with Transthyretin Amyloidosis with Cardiomyopathy (ATTR-CM)

    Cancer Categories
    • Hematologic (Blood Cancers)
    Karmanos Trial ID
    • 2023-084
    NCT ID
    • NCT06128629
    Age Group
    • Adult
    Scope
    • National
    PhaseClick for Clinical Trial Phase DefinitionPhase III
    Includes trials conducted after preliminary evidence suggesting effectiveness of the drug has been obtained, and are intended to gather additional information to evaluate the overall benefit-risk relationship of the drug
    • Phase III
    Principal Investigator
    • Jeffrey
      Zonder, M.D.

      Oncology - Hematology, Oncology - Medical View Profile

    Objective:

    Primary Objective:

    • To evaluate the efficacy of NTLA-2001, as measured by the composite risk of CV-related mortality and CV events (urgent HF visits and hospitalizations due to HF, arrhythmia, or stroke), compared to placebo

    Secondary Objectives:

    • To evaluate the effect of NTLA-2001 on serum TTR compared to placebo
    • To evaluate the impact of NTLA-2001 on participant-reported CM-related symptoms and quality of life compared to placebo
  • Eligibility

    Eligibility

    Inclusion Criteria:
    • Documented diagnosis of ATTR amyloidosis with cardiomyopathy
    • Medical history of heart failure (HF)
    • Symptoms of HF are optimally managed and clinically stable within 28 days prior to administration of study intervention
    • Screening NT-proBNP, a blood marker of HF severity, greater than or equal to 1000 pg/mL (or greater than or equal to 2000 pg/mL if participant has known atrial fibrillation)
    Exclusion Criteria:
    • New York Heart Association (NYHA) Class IV HF
    • Polyneuropathy Disability score of IV (confined to wheelchair or bed)
    • Has hepatitis B, hepatitis C or human immunodeficiency virus (HIV) infection
    • History of active malignancy within 3 years prior to screening
    • RNA silencer therapy (patisiran, inotersen and/or eplontersen) within 12 months prior to dosing. Any prior vutrisiran use is not allowed
    • Initiation of tafamidis within 6 months prior to study dosing
    • Estimated glomerular filtration rate (eGFR) <30 mL/min/1.73m^2
    • Liver failure
    • Uncontrolled blood pressure