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New enzyme therapy for fabry disease tested in Real-World setting

NCT ID NCT06095713

Disease control Sponsor: Universität Münster Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Participants

60 people

The number who actually took part.

Started

Oct 2023

Expected to finish

Sep 2027

An estimate. End dates often move.

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Who is studied

Patient with Fabry Disease

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Males and females, ≥18 years, diagnosed with Fabry disease. * ERT naïve (patients with signs of organ involvement (kidney, heart and/or CNS signs) to be considered for ERT following the European Consensus Guidelines on ERT (Biegstraaten et al. 2015) or patients with neuropathic pain not controlled with pain medication or patients with GI symptoms not relieved with standard medication or ERT switch patients (under ERT for ≥12 months). * Subjects taking ACE inhibitors, ARBs, or renin inhibitors on a stable dose for at least 4 weeks before screening. * Subjects taking analgesics/antidepressants on a stable dose for at least 4 weeks before screening. * Female patients must have a negative pregnancy test and use a medically accepted form of contraception throughout the study. Exclusion Criteria: * Patient is unwilling to give informed consent. * Patient is unable to comply with the clinical protocol. * Patients on dialysis. * Patient has a clinically significant organ disease (e.g., cancer in the past 5 years) that in the opinion of the investigator would preclude participation in the trial. * Patients with a history of organ transplantation.

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Conditions

The condition(s) this trial relates to.

Fabry disease

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Fabry disease

Contacts and locations

Locations

  • Fabry disease center Berlin - Charité - Universitätsmedizin Berlin

    Berlin, Germany

  • Fabry disease center Cologne, Universitätsklinikum Köln

    Cologne, Germany

  • Fabry disease center Hamburg, Universitätsklinikum Hamburg

    Hamburg, Germany

  • Fabry disease center Hannover, Universitätsklinikum Hannover

    Hanover, Germany

  • Fabry disease center Mainz, Universitätsmedizin Mainz

    Mainz, Germany

  • Fabry disease center Münster, Universitätsklinikum Münster

    Münster, Germany

  • Fabry disease center Würzburg, Universitätsklinikum Würzburg

    Würzburg, Germany

  • Fachinternistische Gemeinschaftspraxis, Müllheim

    Müllheim, Germany

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