Transthyroxin Amyloidosis Cardiomyopathy Clinical Trial
Official title:
Efficacy and Safety of Intravenous YOLT-201 for Transthyretin Amyloidosis Cardiomyopathy
This study is a single-arm, open-label, dose-escalation trial aimed at determining the optimal biologically active dose (OBD) of YOLT-201 and providing safety and efficacy evaluation. The OBD is the dose at which serum transthyretin (TTR) protein baseline reduction is ≥60% but not exceeding 95% after 28 days of dosing. The OBD dose should not exceed the maximum tolerated dose (MTD), defined as the highest dose at which no more than one subject experiences dose-limiting toxicity (DLT) within each cohort.
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