Pivotal Trial
This treatment is in the last trial phase before FDA approval.
Prior Safety Data
This treatment has already been tested in at least one earlier human trial.
Ready to participate?
Review the details below, then apply to join this clinical trial.
At a Glance
Age
18 – 90
Sex
Any
Trial phase
Phase 3
Study type
Interventional
Purpose
Treatment
Participants needed
1,200 (estimated)
Sponsor
Intellia Therapeutics · Industry
Who this trial is looking for
This trial is looking for people with a specific heart problem related to a condition called ATTR amyloidosis. Participants will receive either a new treatment called NTLA-2001 or a placebo to compare their effects.
Are You a Good Fit for This Trial?
Rules you outUncontrolled blood pressureUnable to take vitamin A supplements during the study
You may be able to join if
I have been diagnosed with ATTR amyloidosis with cardiomyopathy
I have a history of heart failure
My heart failure symptoms have been managed and stable for 28 days before the study
My NT-proBNP level is between 600 and 10,000 pg/mL
You may not be able to join if
I have New York Heart Association Class IV heart failure
I have a disability score that requires me to be in a wheelchair or bed
I have hepatitis B, hepatitis C, or HIV infection
I have had an active cancer in the last 3 years
I have used RNA silencer therapy in the past 12 months
I started tafamidis or acoramidis less than 56 days ago
My kidney function is very low (eGFR < 30 mL/min/1.73m^2)
I have a history of liver disease
Summarized in plain language from this trial's official eligibility criteria.
The full criteria are further down this page — only the research team can
confirm whether you qualify.
Think this trial could be right for you?
Answer three quick questions written from this study's own rules to see if you may qualify. No account needed to find out.
To evaluate the efficacy and safety of a single dose of NTLA-2001 compared to placebo in participants with ATTR-CM.
Trial Locations
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Status
Eligibility Criteria
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, …
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
* History of liver disease
* Uncontrolled blood pressure
* Unable or unwilling to take vitamin A supplementation for the duration of the study
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