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New Real-World study tracks fabry Drug's Long-Term impact on heart and kidneys

NCT ID NCT07109375

Disease control Sponsor: Chiesi Italia Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
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
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 27, 2026 · Last updated Jul 09, 2026 · Updated 2 times

Summary

This study follows 75 adults with Fabry disease who are taking or starting pegunigalsidase alfa to see how well it works and how safe it is over time in real-world settings. Researchers will check heart structure and function, kidney health, and exercise capacity. The goal is to understand if this enzyme replacement therapy helps control the disease long-term.

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

About 75 people

The number the study aims to enrol. It can still change while the study runs.

Started

Jun 2026

Expected to finish

Feb 2029

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

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

Who is studied

Adults patients who have a genetically confirmed diagnosis of Fabry disease and are being treated or plan to initiate treatment with pegunigalsidase alfa as per clinical practice.

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: 1. Male and female adults (≥ 18 years). 2. Patients with a clinical diagnosis of Fabry disease confirmed by α-Gal A assay and detection of mutation in α-Gal A gene. 3. Patients either taking or planning to take pegunigalsidase alfa as treatment for Fabry disease. The treatment decision must be made independently from participation in this study. 4. Written informed consent to participate in the study and for the processing of personal data. Exclusion Criteria: 1. History of hypersensitivity reaction to pegunigalsidase alfa. 2. Presence of any medical, emotional, behavioural, or psychological condition that, in the judgment of the physician, could interfere with the ability to participate in the study. 3. Female subjects who are pregnant or planning to become pregnant during the study.

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Conditions

The condition(s) this trial relates to.

Fabry disease lysosomal storage disease

As listed by the trial registrant

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

Fabry disease

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    7 sites. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • A.O. Sant'Anna e San Sebastiano

    NOT_YET_RECRUITING

    Caserta, Italy

  • A.O.U. Policlinico "Giaccone"

    NOT_YET_RECRUITING

    Palermo, Italy

  • AOU Federico II, Dipartimento di Nefrologia

    NOT_YET_RECRUITING

    Naples, Italy

  • AUSL Toscana Sud-Est - Ospedale San Donato

    NOT_YET_RECRUITING

    Arezzo, Italy

  • Azienda Ospedaliera Universitaria Consorziale Policlinico Di Bari

    RECRUITING

    Bari, Italy

  • Irccs Azienda Ospedaliero-Universitaria Di Bologna - Policlinico Di Sant'Orsola

    RECRUITING

    Bologna, Italy

  • U.O.C. Nefrologia ASST Monza - Ospedale San Gerardo

    NOT_YET_RECRUITING

    Monza, Italy

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