Taiwan study sheds light on rare fabry mutation and treatment effects

NCT ID NCT06052800

Ongoing Knowledge-focused Sponsor: Sanofi Source: ClinicalTrials.gov ↗

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study follows 78 adults in Taiwan who have Fabry disease and carry a specific genetic mutation called GLA IVS4. Researchers are looking at how the disease affects the heart and other organs over time, both in people who have never been treated and those who have received agalsidase beta therapy. The goal is to better understand the disease's natural history and how well the treatment works in this group.

What this could mean

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

Active substance: agalsidase beta
What this could lead to: If successful, this study could provide a clearer picture of how Fabry disease affects people with this specific mutation and how well agalsidase beta works for them.
What could go wrong: This is an observational study, not a controlled trial, so it cannot prove cause and effect. Results may not apply to people with other mutations or outside Taiwan.

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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

Investigational Site Number : 1580001

Taipei, 100, Taiwan
Investigational Site Number : 1580002

Taipei, 112, Taiwan
Investigational Site Number : 1580003

Taipei, 104, Taiwan
Investigational Site Number : 1580004

Taichung, 407219, Taiwan
Investigational Site Number : 1580005

Tainan, 704, Taiwan

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