Groundbreaking trial tests new hope for rare genetic disorder
NCT ID NCT06858397
Summary
This early-stage study is testing an experimental drug called HM15421/GC1134A in adults with Fabry disease, a rare genetic condition. Researchers want to see if the treatment is safe and if it can reduce harmful substances that build up in patients' bodies. The study will enroll 18 participants who haven't received other Fabry treatments recently.
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.
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Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
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
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Conditions
Explore the condition pages connected to this study.