Niewydolność serca: badanie kliniczne fazy III

Kryteria włączenia kto może wziąć udział (6)

1. Male or female.
2. Age 18 years or above at the time of signing the informed consent.
3. Have an established diagnosis of ATTR-CM, (ATTRwt or ATTRv), with cardiac amyloid infiltration, increased left ventricular (LV) wall thickness, and HF. Note: Target ATTRv recruitment is approximately 15% of the study population. a. Cardiac amyloid infiltration demonstrated by: i. Cardiac biopsy positive for TTR amyloid, OR, ii. Grade 2 or 3 cardiac uptake at PYP/DPD/HMDP nuclear medicine imaging with single-photon emission computed tomography (SPECT) or (SPECT/CT) (preferably) combined with an extracardiac biopsy positive for TTR amyloid, OR, iii. Grade 2 or 3 cardiac uptake at PYP/DPD/HMDP nuclear medicine imaging with SPECT or SPECT/CT (preferably) combined with normal serum free light chain ratio, and negative SPIE and UPIE) (or mass spectrometry based methods including mass fixation) Notes: o Bone tracer nuclear medicine imaging with SPECT or SPECT/CT (preferably) will be conducted using 99m-technetium (Tc)-labelled pyrophosphate (99mTc-PYP)/99mTc-labelled 3,3-diphosphono-1,2-propanodicarboxylic acid (99mTc DPD)/99mTc-labeled hydroxymethylene diphosphonate (99mTc-HMDP). o The eGFR adjusted acceptable serum free light chain ratio o Patients with Grade 2 or 3 cardiac uptake at PYP/DPD/HDMP nuclear imaging with SPECT or SPECT/CT (preferably) and evidence of MGUS (based on serum and urine protein electrophoresis and serum free light chains) will require endomyocardial biopsy with typing using mass spectrometry or immunohistochemistry to confirm presence of TTR protein in tissue. • Timing of serum free light chain ratio, SPIE, UPIE and mass spectrometry- based methods including mass fixation should be within 12 months of SPECT or SPECT/CT nuclear imaging. b. Increased LV wall thickness, as assessed by centralised review of echocardiography, showing interventricular septal wall thickness ≥12 mm. c. Chronic HF (New York Heart Classification [NYHA] I-IV) with: 1. At least 1 documented hospitalisation for HF, OR 2. History of HF manifested by signs or symptoms of volume overload or elevated intracardiac pressures (e.g., elevated jugular venous pressure, shortness of breath, signs of pulmonary congestion on x-ray or auscultation, or peripheral oedema that required or requires ongoing treatment with a diuretic).
4. Expected to be on stable CV medical therapy (defined as no greater than 50% dose adjustment and no categorical changes of medications), with the exception of diuretics, 4 weeks prior to the randomisation visit.
5. NT-proBNP concentration ≥"CCI" pg/mL at screening. Note: Participants with NT-proBNP levels between "CCI" and "CCI" pg/mL may be enrolled until a cap of 35% of the total study population is reached.
6. Completed >50 meters on the 6MWT at screening.