Promising enzyme therapy for kids with rare fabry disease enters key trial
NCT ID NCT06328608
First seen Nov 01, 2025 · Last updated Jun 22, 2026 · Updated 40 times
Summary
This study tests a drug called PRX-102 (pegunigalsidase alfa) in 22 children and teens aged 2 to 18 with Fabry disease, a rare genetic disorder that causes pain and organ damage. The drug is an enzyme replacement therapy given by infusion every two weeks. Researchers will monitor safety, side effects, and how well it controls disease symptoms like pain and kidney function.
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This is a summary of
the original study
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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: •••••@•••••
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
PRX-102 (pegunigalsidase alfa), a lab-made enzyme that replaces the missing or faulty enzyme in Fabry disease
What this could lead to
If successful, this could provide a treatment option for children with Fabry disease to help manage symptoms and slow disease progression.
What could go wrong
This is an early-to-mid stage trial with only 22 participants, so results may not apply to all patients. Side effects like infusion reactions are possible.
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.