To evaluate the efficacy and safety of a single dose of NTLA-2001 compared to placebo in participants with ATTR-CM.
This is a multinational, multicenter, double-blind, placebo-controlled study in approximately 1200 participants, who will be randomized to receive a single infusion of either NTLA-2001 or placebo.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
1,200
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
Change in baseline to month 18 in serum TTR
Time frame: Baseline, Month 18
Change from baseline to month 18 in KCCQ-OS score
Time frame: Baseline, Month 18
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University of Arizona
Tucson, Arizona, United States
RECRUITINGCedars-Sinai Medical Center
Los Angeles, California, United States
RECRUITINGWestern Greater Los Angeles VA Medical Center
Los Angeles, California, United States
RECRUITINGStanford University
Palo Alto, California, United States
RECRUITINGUniversity of California, San Diego (UCSD) - Medical Center
San Diego, California, United States
RECRUITINGUniversity of Colorado Denver
Denver, Colorado, United States
RECRUITINGYale University School of Medicine
New Haven, Connecticut, United States
RECRUITINGMedStar Washington Hospital Center - MedStar Heart and Vascular Institute
Washington D.C., District of Columbia, United States
RECRUITINGMayo Clinic - Jacksonville
Jacksonville, Florida, United States
RECRUITINGCleveland Clinic of Florida
Weston, Florida, United States
RECRUITING...and 122 more locations