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

ConditionTransthyretin Amyloidosis (ATTR) With Cardiomyopathy
Trial statusRecruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
Age18-90
SponsorIntellia Therapeutics

About this trial

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

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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 600 pg/mL and less than 10,000 pg/mL

Disqualifiers

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

Trial design

Design model

Parallel

Treatments tested in this trial

  • NTLA-2001

    Biological/Vaccine

    NTLA-2001 (55mg) by IV infusion

  • Placebo

    Drug

    Normal saline (0.9% NaCl) by IV infusion

Treatment groups

1,200 Participants
are divided into 2 treatment groups
Group A: NTLA-2001Experimental treatment 1 intervention
Group B: PlaceboPlacebo comparator 1 intervention

Trial outcomes

Primary outcomes

1

Composite outcome of cardiovascular (CV) mortality and CV events

Time frame
Maximum study duration is dependent on event rates and is estimated to be at least 18 months and up to approximately 5 years

Secondary outcomes

1

Change in baseline to month 18 in serum TTR

Time frame
Baseline, Month 18
2

Change from baseline to month 18 in KCCQ-OS score

Time frame
Baseline, Month 18

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Intellia Therapeutics

Lead sponsor

Regeneron Pharmaceuticals

Collaborator

Source ClinicalTrials.gov
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