Real-Life Clinical Efficacy of Acoramidis in Participants With ATTR-CM and Association With Cardiac Biomarkers

Korábbi kezelés

• Likelihood of receiving a heart transplant within 1 year from the time screening begins

Más betegség

• Hypersensitivity to acoramidis, its metabolites, or additives in the formulation has been confirmed.
• In the opinion of the responsible (sub)physician for the post-marketing clinical trial, has a history of drug abuse, alcoholism, or psychiatric disorder that would preclude compliance with this Post-Marketing Clinical Study Protocol

Laborértékek

• Switch participants: prior treatment with gene silencing agents (pachysilane sodium, butrisilane sodium) as treatment for ATTR-CM (including when specifically scheduled to start treatment with a gene silencing agent)
• Has a clinically significant medical condition, an abnormal laboratory test result, or a condition that may jeopardize the safety of the study participant, increase the risk of participation in the post-marketing clinical trial, or affect the study

Egyéb

• Have confirmed diagnosis of AL amyloidosis
• Pregnant or lactating women
• Participating in an interventional study other than this study, including a clinical trial

• Treatment history of ATTR-CM is one of the following:
• Naive participants: newly diagnosed with ATTR-CM and no prior treatment with drugs for ATTR-CM
• Switch participants: Participants who are using tafamidis, a TTR stabilizer, as treatment for ATTR-CM and who, in the judgment of the post-marketing clinical trial investigator (co-principal investigator), can be expected to benefit from switching to acoramidis.
• Naive participants must meet the following requirements:
  • History of hospitalization for heart failure or heart failure symptoms requiring treatment, including diuretics
  • Echocardiographic end-diastolic ventricular septal thickness greater than 12 millimeters (mm)
  • Confirmed diagnosis of ATTR-CM (wild type or mutant) by one of the following diagnostic methods
  • Tissue biopsy shows amyloid deposition and TTR precursor protein is identified by immunohistochemistry or mass spectrometry.
  • Bone scintigraphy showing strong accumulation ** (Perugini score ≥ 2) consistent with myocardium and no M protein, negating the possibility of AL amyloidosis