HM15421 for Fabry Disease

This trial explores a new treatment called HM15421 (also known as GC1134A) for individuals with Fabry Disease, a rare genetic disorder affecting the breakdown of a specific type of fat. The trial aims to determine the treatment's safety and effectiveness, as well as its movement through the body. Participants will receive one of three different doses to identify the most effective and tolerable option. Those with Fabry Disease who exhibit clinical symptoms and have not received treatment in the past six months may qualify for this study. As a Phase 1 trial, this research seeks to understand how the treatment works in people, offering participants the opportunity to be among the first to receive it.

The trial does not specify if you need to stop taking your current medications, but you cannot have started or changed the dose of an ACE inhibitor or ARB in the 4 weeks before screening. If you are on FD therapy, you must not have received it in the past 6 months.

Research shows that HM15421, also called GC1134A, is being tested for safety in people with Fabry Disease. This marks the first study of the treatment in humans, so data remains limited. The trial is in its early stages, and researchers are closely monitoring for any side effects.

Unlike the standard treatments for Fabry disease, such as enzyme replacement therapy (ERT) and chaperone therapy, HM15421/GC1134A offers a potentially novel approach. Researchers are excited about these treatments because they explore different dosing regimens—low, mid, and high doses—which may optimize the therapeutic effect and minimize side effects. By experimenting with various doses, HM15421/GC1134A could provide more personalized treatment options, potentially improving patient outcomes and quality of life. This trial aims to refine and enhance how Fabry disease is managed compared to traditional methods.

Research has shown that HM15421, a new treatment being tested in this trial, may help people with Fabry disease. In earlier studies, this treatment significantly reduced the size of the heart's left ventricle, which is important for heart health. Other studies on animals suggested that HM15421 might also improve kidney function and help with blood vessel and nerve problems. These findings indicate that HM15421 could be a promising option for treating Fabry disease by addressing its main symptoms and complications. Participants in this trial will receive different dosages of HM15421 to evaluate its effectiveness and safety.¹³⁵⁶⁷