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.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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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
Locations
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Centro Medico IPAM
RECRUITINGRosario, Santa Fe Province, S2000, Argentina
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Children's Hospital Medical Center
RECRUITINGCincinnati, Ohio, 45229-3039, United States
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David Geffen School of Medicine UCLA, UCLA Health
RECRUITINGLos Angeles, California, 90095, United States
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Hospital Italiano de Buenos Aires
RECRUITINGBuenos Aires, C1199ABB, Argentina
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Lysosomal and Rare Disorders Research and Treatment Center
RECRUITINGFairfax, Virginia, 22030, United States
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Pusan National University Children's Hospital in Yangsan
RECRUITINGYangsan, Gyeongsangnam-do, 50612, South Korea
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University of Kansas School of Medicine
RECRUITINGKansas City, Kansas, 66160-8500, United States
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University of Minnesota
RECRUITINGMinneapolis, Minnesota, 55455, United States
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University of Pittsburgh Medical Center Children's Hoispital of Pittsburgh
RECRUITINGPittsburgh, Pennsylvania, 15224, United States
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Yonsei University, College of Medicine
RECRUITINGSeoul, 03722, South Korea