New hope for kids with fabry: experimental drug PRX-102 under study
NCT ID NCT06328608
First seen Nov 01, 2025 · Last updated May 15, 2026 · Updated 31 times
Summary
This study is testing an experimental drug called PRX-102 in children and teens aged 2 to 18 with Fabry disease, a rare genetic condition. The goal is to see if the drug is safe and helps control the disease by replacing a missing enzyme. About 22 participants will receive the drug and be monitored for side effects, growth, and disease symptoms.
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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.
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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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Centre Hospitalier Universitaire (CHU) de Bordeaux - Groupe Hospitalier Pellegrin
RECRUITINGBordeaux, 33076, France
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Cincinnati Children's Hospital Medical Center
NOT_YET_RECRUITINGCincinnati, Ohio, 45229, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Emory Genetics Clinical Trials Center
RECRUITINGAtlanta, Georgia, 30322, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Great Ormond Street Hospital for Children NHS Foundation Trust
NOT_YET_RECRUITINGLondon, United Kingdom
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Haukeland Universitetssjukehus
RECRUITINGBergen, 5021, Norway
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Hopital Arnaud de Villeneuve
RECRUITINGMontpellier, France
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Hospital Clinico Universitario De Santiago De Compostela
RECRUITINGSantiago de Compostela, Spain
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Lysosomal and Rare Disorders Research and Treatment Center Inc
NOT_YET_RECRUITINGFairfax, Virginia, 22030, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Phoenix Children's
RECRUITINGPhoenix, Arizona, 85016, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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UK für Kinder- und Jugendheilkunde der PMU Salzburg
RECRUITINGSalzburg, Austria
Contact Phone: •••-•••-•••• Email: •••••@•••••
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University of Iowa
RECRUITINGIowa City, Iowa, 52242, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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University of Utah
RECRUITINGSalt Lake City, Utah, 84108, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Conditions
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