Testing NTLA-2001 against a placebo for transthyretin amyloidosis (ATTR) with cardiomyopathy
Official title: MAGNITUDE: A Phase 3 Study of NTLA-2001 in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM)
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)
- Phase 3
- 2 groups
- Sites in Calgary, Vancouver and 2 more cities
- Recruiting
Interventions
- Biological therapy
NTLA-2001
NTLA-2001 (55mg) by IV infusion
- Medication
Placebo
Normal saline (0.9% NaCl) by IV infusion
Canadian Sites (4)
4 of 4 recruiting
- Recruiting
Alberta Health Services - University of Calgary
Calgary, Alberta
- Recruiting
Vancouver Coastal Health Research Institute
Vancouver, British Columbia
- Recruiting
University Health Network - Toronto General Hospital
Toronto, Ontario
- Recruiting
McGill University Health Centre
Montreal, Quebec
Eligibility Criteria
See who this study is looking for14 criteria
The trial’s own eligibility text, word for word from the registry. Only the trial site can say who takes part.
Inclusion
- +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
- +ScreeningScreeningThe checks done before joining, to see whether a study fits.Read more → NT-proBNP, a blood marker of HF severity, greater than or equal to 600 pg/mL and less than 10,000 pg/mL
Exclusion
- −Has hepatitis B, hepatitis C or human immunodeficiency virus (HIV) infection
- −New York Heart Association (NYHA) Class IV HF
- −Polyneuropathy Disability score of IV (confined to wheelchair or bed)
- −History of active malignancy within 3 years prior to screeningScreeningThe checks done before joining, to see whether a study fits.Read more →
- −RNA silencer therapy (patisiran, inotersen and/or eplontersen) within 12 months prior to dosing. Any prior vutrisiran use is not allowed
- −Initiation of tafamidis or acoramidis within 56 days prior to study dosing
- −Estimated glomerular filtration rate (eGFR) \<30 mL/min/1.73m\^2
- −History of liver disease
- −Uncontrolled blood pressure
- −Unable or unwilling to take vitamin A supplementation for the duration of the study
In plain language
Assembled directly from this trial’s registry record. Every sentence traces to a field below — nothing here is generated or interpreted.
What this study is
This study is testing a treatment for a condition.
Phase 3Phase 3A large study comparing a treatment against the current standard.Read more → — a large study comparing this against the current standard, usually across many hospitals.
From the trial registry
Built from these fields:
- designModule.designInfo.primaryPurpose
- designModule.phases
Who receives what
There are 2 groups in this study.
Group A receives NTLA-2001.
Registry label: A: NTLA-2001
Group B, the placeboPlaceboA dummy treatment with no active medicine, used for comparison.Read more → group, receives Placebo.
Registry label: B: Placebo
From the trial registry
Built from these fields:
- armsInterventionsModule.armGroups[].label
- armsInterventionsModule.armGroups[].type
- armsInterventionsModule.armGroups[].interventionNames
How the study is run
Which group you would be placed in is decided by chance, like a coin flip — not by you and not by your doctor.
You, the study team, the people giving the treatment, and the people analysing the results would all be kept unaware of which group you are in until the study ends.
From the trial registry
Built from these fields:
- designModule.designInfo.allocation
- designModule.designInfo.maskingInfo.masking
Is there a placebo?
One group receives a placeboPlaceboA dummy treatment with no active medicine, used for comparison.Read more → — a dummy treatment with no active medicine in it.
From the trial registry
Built from these fields:
- armsInterventionsModule.armGroups[].type
- armsInterventionsModule.armGroups[].interventionNames
Who the study is looking for
The study lists an age range of 18 years to 90 years.
The study is open to people of any sex.
The study does not accept healthy volunteersHealthy volunteerSomeone without the condition being studied who takes part anyway.Read more →.
These are the criteria the study lists. Only the trial siteTrial siteA hospital or clinic where a study is actually run.Read more → can decide whether someone can take part.
From the trial registry
Built from these fields:
- eligibilityModule.minimumAge
- eligibilityModule.maximumAge
- eligibilityModule.sex
- eligibilityModule.healthyVolunteers
How big and how long
The study aims to enrol about 1,200 people.
The study is currently expected to finish around April 2028.
The main measurement is taken over: Maximum study duration is dependent on event rates and is estimated to be at least 18 months and up to approximately 5 years.
From the trial registry
Built from these fields:
- designModule.enrollmentInfo.count
- statusModule.completionDateStruct.date
- outcomesModule.primaryOutcomes[].timeFrame
What the study measures
Composite outcome of cardiovascular (CV) mortality and CV events — measured over Maximum study duration is dependent on event rates and is estimated to be at least 18 months and up to approximately 5 years.
From the trial registry
Built from these fields:
- outcomesModule.primaryOutcomes[].measure
- outcomesModule.primaryOutcomes[].timeFrame
Source: NCT06128629 on ClinicalTrials.gov. The full registry text is further down this page — this summary never replaces it.
Not medical advice. What do these terms mean?
What is being tested — in plain terms
About NTLA-2001Biological
NTLA-2001 (55mg) by IV infusion
From the trial registry — its own words, unedited.
What a biological is here: A treatment made from or by living systems — such as antibodies, vaccines, or cell-based therapies.
Read the full explanation → · in clinical review
About PlaceboDrug
Normal saline (0.9% NaCl) by IV infusion
From the trial registry — its own words, unedited.
What a drug is here: A medicine — a chemical or small-molecule treatment — given as the thing being studied.
Read the full explanation → · in clinical review
Common questions
Answered from this trial’s registry record. Where the record doesn’t say, these answers say so rather than filling the gap.
Am I eligible for this trial?
Only the trial siteTrial siteA hospital or clinic where a study is actually run.Read more → can decide whether someone can take part. Concord cannot make that determination, and neither can any tool that has not examined you.
What the study lists: an age range of 18 years to 90 years.
The full inclusionInclusion criteriaThe things you must have or be for a study to consider you.Read more → and exclusion criteriaExclusion criteriaThe things that would prevent someone from taking part.Read more → are published on this page, exactly as the study team wrote them.
The useful next step is to bring this trial to your doctor. Answering a few questions first gives them something concrete to review.
From the trial registry
- eligibilityModule.minimumAge
- eligibilityModule.maximumAge
- eligibilityModule.eligibilityCriteria
Is there a placebo?
Yes. This study includes a placeboPlaceboA dummy treatment with no active medicine, used for comparison.Read more → group — a dummy treatment with no active medicine in it.
From the trial registry
- armsInterventionsModule.armGroups[].type
Would I know which treatment I am getting?
You, the study team, the people giving the treatment, and the people analysing the results would all be kept unaware of which group you are in until the study ends.
Which group you would be placed in is decided by chance, like a coin flip — not by you and not by your doctor.
From the trial registry
- designModule.designInfo.maskingInfo.masking
- designModule.designInfo.allocation
Who can join?
The study lists an age range of 18 years to 90 years.
It is open to people of any sex.
It does not accept healthy volunteersHealthy volunteerSomeone without the condition being studied who takes part anyway.Read more →.
Only the trial siteTrial siteA hospital or clinic where a study is actually run.Read more → can confirm whether a particular person can take part.
From the trial registry
- eligibilityModule.minimumAge
- eligibilityModule.maximumAge
- eligibilityModule.sex
- eligibilityModule.healthyVolunteers
How long would this take?
The study's main measurement is taken over: Maximum study duration is dependent on event rates and is estimated to be at least 18 months and up to approximately 5 years.
The study as a whole is currently expected to finish around 2028-04.
How long any one person takes part can differ from the study length. The study team can tell you what the schedule looks like in practice.
From the trial registry
- outcomesModule.primaryOutcomes[].timeFrame
- statusModule.completionDateStruct.date
How many people are taking part?
The study aims to enrol about 1,200 people.
From the trial registry
- designModule.enrollmentInfo.count
Where is this happening?
This study lists 8 locations, including: Calgary, Alberta, Canada; Vancouver, British Columbia, Canada; Toronto, Ontario, Canada; Montreal, Quebec, Canada; Ann Arbor, Michigan, United States; Detroit, Michigan, United States, and 2 more.
Sites can open and close during a study, so confirm with the team before travelling.
From the trial registry
- trial_locations
About This Trial
To evaluate the efficacy and safety of a single dose of NTLA-2001 compared to placebo in participants with ATTR-CM.
Other Sites (5)
University of Michigan
Ann Arbor, Michigan, United States
Barbara Ann Karmanos Cancer Institute
Detroit, Michigan, United States
Mayo Clinic
Rochester, Minnesota, United States
New York University (NYU) School of Medicine - Langone Medical Center
New York, New York, United States
Columbia University Medical Center
New York, New York, United States
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See if this trial could fit youThis page is for informational purposes only and does not constitute medical advice. Clinical trial eligibility can only be determined by the trial site after proper screening. Trial information is sourced from ClinicalTrials.gov and may not reflect the most current status. Always consult your healthcare provider before making decisions about clinical trial participation.