(23026) A prospective, single-arm, Phase 4 study to evaluate the course of serum transthyretin (TTR) level with acoramidis in adult patients with variant or wild-type transthyretin amyloidosis with cardiomyopathy (ATTR-CM) previously treated with tafamidis.

Condition studied

Transthyretin Amyloid Cardiomyopathy (ATTR-CM)

Investigational medicinal product(s)

Eligibility

Primary endpoint

Change in serum TTR level from baseline to month 6 or premature discontinuation of treatment

Endpoint detail

Change in serum TTR level from baseline to week 1, 2, 3, 4 and at month 3., Occurrence of TEAEs and type of TEAEs and serious TEAEs., Change from baseline to month 6 of NT-proBNP, hs-TnT, RBP4., Signs of remodeling from baseline to month 6 in ECHO., Change from baseline to month 6 in 6MWD, Change from baseline to month 6 in kidney and thyroid biomarkers: eGFR, Creatinine, Cystatin C, UACR, and TSH., Change from baseline to month 6 in KCCQ-OS score and in EQ-5D-5L index score.