MAGNITUDE: A Phase 3 Study of NTLA-2001 in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM)
To evaluate the efficacy and safety of a single dose of NTLA-2001 compared to placebo in participants with ATTR-CM.
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.
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 600 pg/mL and less than 10,000 pg/mL
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 or acoramidis within 56 days 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
Eligibility
Age eligible for study : NA
Healthy volunteers accepted : No
Gender eligible for study: ALL
Things to know
Study dates
Study start:
Primary completion:
Study completion finish:
Study type
TREATMENT
Phase
- PHASE3
Study ID
NCT06128629
Intervention or treatment
BIOLOGICAL: NTLA-2001
DRUG: Placebo
Conditions
- Transthyretin Amyloidosis (ATTR) With Cardiomyopathy
You may be eligible to participate in this trial based on your search. Check eligibility by answering some questions.
Research sites nearby
Closest Location:
The Canberra Hospital
Select from list below to view details:
The Canberra Hospital
Garran, Australian Capital Territory, Australia
Westmead Hospital
Westmead, New South Wales, Australia
Cairns Hospital
Cairns, Queensland, Australia
Eastern Health - Box Hill Hospital
Box Hill, Victoria, Australia
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Who is running the clinical trial?
Intellia Therapeutics
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/Arm | Intervention/Treatment |
|---|---|
EXPERIMENTAL: NTLA-2001
| Biological: NTLA-2001 |
PLACEBO_COMPARATOR: Placebo
| Drug: Placebo |
What is the study measuring?
Primary Outcome
| Measure | Description | Time Frame |
|---|---|---|
| Composite outcome of cardiovascular (CV) mortality and CV events | Not Specified | Maximum study duration is dependent on event rates and is estimated to be at least 18 months and up to 48 months |
Secondary Outcome
| Measure | Description | Time Frame |
|---|---|---|
| Change in baseline to month 18 in serum TTR | Not Specified | Baseline, Month 18 |
| Change from baseline to month 18 in KCCQ-OS score | Not Specified | Baseline, Month 18 |
References
Clinical Trials Gov: MAGNITUDE: A Phase 3 Study of NTLA-2001 in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM)Other trials to consider
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