New hope for kids with rare genetic disease: experimental enzyme therapy trial opens
NCT ID NCT06328608
Summary
This study is testing an enzyme replacement therapy called PRX-102 in children and adolescents aged 2 to 17 who have Fabry disease, a rare genetic disorder. The main goals are to find the safest and most effective dose for young patients and to see how well the treatment works at managing symptoms like pain, heart problems, and kidney issues. About 22 participants will receive the drug through an IV every two weeks, and researchers will monitor their health closely over time.
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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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