New enzyme therapy for fabry disease tested in Real-World setting
NCT ID NCT06095713
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study follows 60 adults with Fabry disease who are receiving pegunigalsidase-alfa, a newer enzyme replacement therapy. Researchers want to see how well it works in everyday medical practice, especially for kidney function. Participants will be treated for 2 years at specialized Fabry centers in 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 (an enzyme replacement therapy)
- What this could lead to
- If successful, this could show that pegunigalsidase-alfa is a better long-term option for managing Fabry disease, especially for kidney health.
- What could go wrong
- This is an observational study, not a controlled trial, so results are less definitive. It's also small (60 people) and only lasts 2 years, which may not capture long-term effects or rare side effects.
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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.
Contacts and locations
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
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can early enzyme therapy save kidneys in fabry disease?
- Can continued lucerastat access help fabry patients?
- Can a single gene infusion rewrite the story of fabry disease?
- Fabry disease sperm study halted early
- Heart study reveals hidden link between blood vessels and muscle blockage
- Taiwan study sheds light on rare fabry mutation and treatment effects