Open Label Study to Evaluate Safety and Effectiveness of HM15421GC1134A in Adults With Fabry Disease

What this Trial Is About

Researchers are conducting a Phase 12 first-in-human study to evaluate the safety, tolerability, pharmacokinetics, and efficacy of the drug HM15421 in patients diagnosed with Fabry Disease FD. This study aims to understand how the drug behaves in the body and its effects on this condition. It is sponsored by GC Biopharma Corp and focuses on patients with clinical symptoms and genetic markers of FD. Participants will receive different doses of HM15421 administered subcutaneously, divided into three groups low, mid, and high dose cohorts. The study is open-label and non-randomized, meaning all participants know they are receiving the study drug. The treatment and evaluation period lasts up to 48 weeks, during which safety and efficacy outcomes will be closely monitored. During the study, participants will undergo regular assessments including laboratory tests to measure drug levels and kidney function, urine and plasma biomarkers related to FD, and monitoring for any adverse events. The primary outcome is the incidence and nature of adverse events over 48 weeks. Researchers will also measure drug concentrations in the blood and evaluate kidney changes using specialized scoring systems. The total participation time can extend up to 48 weeks with continuous safety and efficacy monitoring.

A proof-of Concept Study to Assess Safety and Tolerability of HM15421/GC1134A in Patients With Fabry Disease