Phase IIIa Study to Evaluate Safety and Effects of YOLT-201 Infusion in Adults Aged 18 to 80 with Transthyretin Amyloidosis Polyneuropathy or Cardiomyopathy

What this Trial Is About

Researchers are evaluating the safety, tolerability, pharmacokinetics, and pharmacodynamics of YOLT-201 in adults aged 18 to 80 with hereditary transthyretin amyloidosis affecting nerves polyneuropathy or the heart cardiomyopathy. This study focuses on participants with confirmed genetic mutations causing these conditions to better understand how YOLT-201 behaves and is tolerated in these groups. The study involves giving participants a single infusion of YOLT-201 and monitoring them over time. This is an open-label trial, so all participants receive the study drug without a placebo group. The study includes an initial dose-escalation phase followed by a dose-expansion phase to assess different dosing levels and effects. Participants will be closely monitored for safety and tolerability through week 104, with regular assessments including laboratory tests and clinical evaluations. Researchers will track how the drug is processed in the body and its biological effects. Participants agree to avoid certain treatments and follow contraceptive guidelines during the study. The total duration includes screening, treatment, and long-term follow-up to understand the drugs impact and safety profile.

A Phase I/IIa，Open-label, Single Ascending Dose and Dose-expansion Clinical Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of YOLT-201 in Patients With Transthyretin Amyloidosis Polyneuropathy (ATTR-PN) or Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM)