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How much time does fabry treatment really take? new study measures the burden

NCT ID NCT04281537

Knowledge-focused Sponsor: Amicus Therapeutics 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
Running, but no longer taking on new participants.
Completed This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 completed study looked at how much time healthcare professionals spend preparing and giving enzyme replacement therapy (ERT) to adults with Fabry disease. It also measured the impact on patients and their caregivers, including time, costs, and quality of life. The goal was to describe the real-world experience of ERT treatment across multiple countries.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
agalsidase alfa and agalsidase beta (enzyme replacement therapy)
What this could lead to
If successful, this study could help clinics plan better and reduce the burden of treatment for Fabry disease patients and their caregivers.
What could go wrong
This is an observational study, not a treatment trial. It describes current practices but does not test a new therapy or change outcomes.

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

82 people

The number who actually took part.

Started

Mar 2020

Finished

May 2022

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

Patients with FD who are receiving ERT and their caregivers will be identified and recruited from approximately 12 specialist centres in four countries (Taiwan, Turkey, Brazil and Japan).

Ages

18 years and older

Sex

Anyone

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.

Patient Inclusion Criteria: * Patients with a documented diagnosis of Fabry Disease (FD) * Patients who have received ≥4 doses of ERT (with agalsidase alfa or agalsidase beta) for the treatment of FD. * Patients who present to the participating hospital(s) or treatment centre(s) for administration of a dose of ERT (as part of their routine treatment) during the data collection period. Caregiver Inclusion Criteria: -Self-identifies as a caregiver of a patient with FD for whom written informed consent has been obtained for inclusion in the study. Patient Exclusion Criteria: * Patients who are unable or unwilling to give consent for study participation. * Patients whose ERT preparation and administration takes place exclusively in the home setting with no healthcare professional (HCP) involvement in preparation of the infusion. * For the time and motion evaluation: Patients whose ERT is administered by a HCP who does not consent to be observed. Caregiver Exclusion Criteria: \- Caregiver (and/or the patient with FD whom they support or care for) is unable or unwilling to give consent for study participation.

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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

  • Amicus Therapeutics, Inc.

    Philadelphia, Pennsylvania, 19104, United States

  • Dokuz Eylul University Medical Faculty

    Izmir, Turkey (Türkiye)

  • Ege University Hospital

    Izmir, Turkey (Türkiye)

  • Gazi University Hospital

    Ankara, Turkey (Türkiye)

  • Instituto de Genética e Erros Inatos do Metabolismo (IGEIM)

    São Paulo, Brazil

  • Keio University Hospital

    Tokyo, Japan

  • National Taiwan University

    Taipei, Taiwan

  • Taipei Veterans General Hospital

    Taipei, Taiwan

  • Yokohama Municipal Citizen's Hospital

    Yokohama, Japan

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