Clinical Trial
Study Details
Status
RECRUITING
Study ID #
NCT06128629
Study Start
Primary Completion
Study Completion
Enrollment
765
Study Type
Interventional
Phase
Phase 3
Interventions
Placebo

Normal saline (0.9% NaCl) by IV infusion

Primary Outcomes
Measure
Composite outcome of cardiovascular (CV) mortality and CV events
Timeframe
Maximum study duration is dependent on event rates and is estimated to be at least 18 months and up to 48 months
Secondary Outcomes
Measure
Change in baseline to month 18 in serum TTR
Timeframe
Baseline, Month 18
Measure
Change from baseline to month 18 in KCCQ-OS score
Timeframe
Baseline, Month 18
Summary

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

Description

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.

Conditions
Transthyretin Amyloidosis (ATTR) With Cardiomyopathy
Study Contact
Name
Trial Manager at Intellia
Role
Contact
Phone
1-857-285-6200
Email
medicalinformation@intelliatx.com
Facility

MedStar Washington Hospital Center - MedStar Heart and Vascular Institute
Washington, DC 20010
United States

Facility

University of Kentucky Gill Heart Institute
Lexington, KY 40536
United States

Facility

Columbia University Medical Center
New York, NY 10032
United States

Facility

The Canberra Hospital
Garran
Australia

Facility

Fiona Stanley Hospital
Perth
Australia

Facility

Westmead Hospital
Westmead
Australia

Facility

New Zealand Clinical Research
Auckland
New Zealand

Facility

New Zealand Clinical Research
Christchurch
New Zealand

Facility

Richmond Pharmacology
London
United Kingdom

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, 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

Eligibility Minimum Age
18 Years
Eligibility Maximum Age
90 Years
Sexes Eligible for Study
ALL
Eligibility Std Ages
Adult
Older Adult
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