New enzyme therapy for fabry disease tested in Real-World study
NCT ID NCT06095713
First seen May 02, 2026 · Last updated Jun 23, 2026 · Updated 7 times
Summary
This study follows 60 adults with Fabry disease across 8 German centers to see how well pegunigalsidase-alfa works in everyday practice. Participants receive the enzyme replacement therapy for 2 years, and researchers track kidney function and other outcomes. The goal is to see if this newer treatment offers better long-term control with fewer side effects than older therapies.
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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.
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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Locations
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Fabry disease center Berlin - Charité - Universitätsmedizin Berlin
Berlin, Germany
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Fabry disease center Cologne, Universitätsklinikum Köln
Cologne, Germany
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Fabry disease center Hamburg, Universitätsklinikum Hamburg
Hamburg, Germany
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Fabry disease center Hannover, Universitätsklinikum Hannover
Hanover, Germany
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Fabry disease center Mainz, Universitätsmedizin Mainz
Mainz, Germany
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Fabry disease center Münster, Universitätsklinikum Münster
Münster, Germany
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Fabry disease center Würzburg, Universitätsklinikum Würzburg
Würzburg, Germany
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Fachinternistische Gemeinschaftspraxis, Müllheim
Müllheim, Germany
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
Pegunigalsidase-alfa (enzyme replacement therapy)
What this could lead to
If successful, this could confirm that pegunigalsidase-alfa is a safe and effective long-term treatment option for Fabry disease, potentially with fewer immune reactions.
What could go wrong
This is an observational study, not a controlled trial, so results may be less definitive. The small size (60 patients) and lack of blinding limit how much we can conclude.
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.