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MAGNITUDE: a Phase 3 Study of NTLA-2001 in Participants with Transthyretin Amyloidosis with Cardiomyopathy (ATTR-CM)

PHASE3RECRUITING

To evaluate the efficacy and safety of a single dose of NTLA-2001 compared to placebo in participants with ATTR-CM.

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Study details:

This is a multinational, multicenter, double-blind, placebo-controlled study in approximately 765 participants, who will be randomized to receive a single infusion of either NTLA-2001 or placebo.

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Eligibility criteria

Researchers look for people who fit a certain description, called eligibility criteria. See if you qualify.

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
  • Unable or unwilling to take vitamin A supplementation for the duration of the study
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    Eligibility

    Age eligible for study : 18 and older

    Healthy volunteers accepted : No

    Gender eligible for study: All

    Things to know

    Study dates

    Study start: 2023-12-13

    Primary completion: 2027-12-01

    Study completion finish: 2028-04-01

    study type

    Study type

    TREATMENT

    phase

    Phase

      PHASE3

    trial

    Trial ID

    NCT06128629

    Intervention or treatment

    BIOLOGICAL: NTLA-2001

    DRUG: Placebo

    Conditions

    • Transthyretin Amyloidosis (ATTR) with Cardiomyopathy
    Image related to Transthyretin Amyloidosis (ATTR) with Cardiomyopathy
    • Condition: Transthyretin Amyloidosis (ATTR) with Cardiomyopathy

    • BIOLOGICAL: NTLA-2001 and other drugs

    • Adelaide, Not Specified, Australia and more

    • Sponsor: Intellia Therapeutics

    Find a site

    Closest Location:

    Royal Adelaide Hospital

    Research sites nearby

    Select from list below to view details:

    • Royal Adelaide Hospital

      Adelaide, Not Specified, Australia

    • The Canberra Hospital

      Garran, Australian Capital Territory, Australia

    • Westmead Hospital

      Westmead, New South Wales, Australia

    • Cairns Hospital

      Cairns, Queensland, Australia

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    Study Plan

    This section provides details of the study plan, including how the study is designed and what the study is measuring.

    How is the study designed?

    Participant Group/ArmIntervention/Treatment
    EXPERIMENTAL: NTLA-2001
    • Single intravenous (IV) infusion of NTLA-2001
    BIOLOGICAL: NTLA-2001
    • NTLA-2001 (55mg) by IV infusion
    PLACEBO_COMPARATOR: Placebo
    • Single IV infusion of normal saline
    DRUG: Placebo
    • Normal saline (0.9% NaCl) by IV infusion

    What is the study measuring?

    Primary outcome

    Primary Outcome MeasurePrimary Outcome DescriptionPrimary Outcome Time Frame
    Composite outcome of cardiovascular (CV) mortality and CV eventsNot SpecifiedMaximum study duration is dependent on event rates and is estimated to be at least 18 months and up to 48 months

    Secondary outcome

    Secondary Outcome MeasureSecondary Outcome DescriptionSecondary Outcome Time Frame
    Change in baseline to month 18 in serum TTRNot SpecifiedBaseline, Month 18
    Change from baseline to month 18 in KCCQ-OS scoreNot SpecifiedBaseline, Month 18

    Frequently Asked Questions

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    References

    Clinical Trials Gov: MAGNITUDE: a Phase 3 Study of NTLA-2001 in Participants with Transthyretin Amyloidosis with Cardiomyopathy (ATTR-CM)

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