Safety and Early Effectiveness of ASC618 Gene Therapy for Severe and Moderately Severe Hemophilia A in Adult Males

What this Trial Is About

Researchers are evaluating a gene therapy called ASC618 for adult men with severe or moderately severe Hemophilia A, a condition where the blood does not clot properly due to low levels of factor VIII. The study aims to assess the safety and early effectiveness of this therapy, which uses a liver-targeted viral vector to deliver a modified human factor VIII gene. This approach addresses challenges with current treatments that require frequent intravenous injections due to their short half-life. Participants will receive a single intravenous infusion of ASC618. This gene therapy uses an AAV vector to deliver a codon-optimized human factor VIII gene designed to increase factor VIII activity in the liver. The study is conducted as a phase 12 trial, focusing on the treatments safety and preliminary effects. During the 12 months following infusion, participants will be monitored for adverse events and serious adverse events. Researchers will measure changes in factor VIII activity levels, annual factor VIII consumption, and annual bleeding rates. The study involves regular evaluations to track these outcomes and ensure participant safety throughout the follow-up period.

ASC618 Gene Therapy in Hemophilia A Patients