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

NCT ID NCT04281537

Completed Knowledge-focused Sponsor: Amicus Therapeutics Source: ClinicalTrials.gov ↗

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.

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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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