Efficacy and Safety of Intravenous YOLT-201 for Transthyretin Amyloidosis Cardiomyopathy
Status:
Recruiting
Trial end date:
2025-10-16
Target enrollment:
Participant gender:
Summary
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.