New hope for kids with rare genetic disease
NCT ID NCT06328608
Summary
This study is testing a drug called PRX-102 to see if it is safe and effective for treating children and adolescents with Fabry disease, a rare genetic disorder. The drug is an enzyme replacement therapy, given as an infusion into a vein every two weeks, and is intended to help manage the disease. The trial will enroll about 22 participants, aged 2 to 17, to find the right dose and see how well it works on symptoms like pain and organ function.
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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Genom att skicka in godkänner du våra Användarvillkor
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
Explore the condition pages connected to this study.