New hope for fabry patients: experimental drug enters human trials
NCT ID NCT06858397
First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This early-stage trial is testing an experimental drug called HM15421/GC1134A in 18 adults with Fabry disease, a rare genetic disorder. The study aims to check the drug's safety, how the body processes it, and whether it can help control the disease. Participants receive the drug as an injection under the skin.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
HM15421/GC1134A (an experimental drug given as an injection under the skin)
What this could lead to
If successful, this could point toward a new treatment option for Fabry disease, potentially helping to manage symptoms and slow disease progression.
What could go wrong
This is an early, first-in-human study with only 18 participants, so safety and effectiveness are not yet proven. The drug may cause side effects or fail to show benefit.
Disclaimer
Read more
Show less
This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for FABRY DISESASE are added.
By submitting, you agree to our Terms of use
Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Show contact details
Enter your email to view the contact information for this study.
By submitting, you agree to our Terms of use
Study contacts
-
Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
-
Centro Medico IPAM
RECRUITINGRosario, Santa Fe Province, S2000, Argentina
Contact
-
Children's Hospital Medical Center
RECRUITINGCincinnati, Ohio, 45229-3039, United States
Contact
-
David Geffen School of Medicine UCLA, UCLA Health
RECRUITINGLos Angeles, California, 90095, United States
Contact
-
Hospital Italiano de Buenos Aires
RECRUITINGBuenos Aires, C1199ABB, Argentina
Contact
-
Lysosomal and Rare Disorders Research and Treatment Center
RECRUITINGFairfax, Virginia, 22030, United States
Contact
-
Pusan National University Children's Hospital in Yangsan
RECRUITINGYangsan, Gyeongsangnam-do, 50612, South Korea
Contact
-
University of Kansas School of Medicine
RECRUITINGKansas City, Kansas, 66160-8500, United States
Contact
-
University of Minnesota
RECRUITINGMinneapolis, Minnesota, 55455, United States
Contact
-
University of Pittsburgh Medical Center Children's Hoispital of Pittsburgh
RECRUITINGPittsburgh, Pennsylvania, 15224, United States
Contact
-
Yonsei University, College of Medicine
RECRUITINGSeoul, 03722, South Korea
Contact