Fabrazyme shows promise for fabry disease in chinese study

NCT ID NCT05054387

Completed Disease control Sponsor: Genzyme, a Sanofi Company Source: ClinicalTrials.gov ↗

First seen Jun 24, 2026 · Last updated Jun 26, 2026 · Updated 1 time

Summary

This study tested the safety and effectiveness of Fabrazyme (agalsidase beta) in 22 Chinese patients with Fabry disease, a rare genetic disorder. Participants received enzyme replacement therapy through IV infusions over 54 weeks. The study monitored side effects and changes in disease markers like lyso-GL3 and GL3 levels.

What this could mean

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

Active substance: Fabrazyme (agalsidase beta)
What this could lead to: If successful, this could confirm that Fabrazyme is safe and effective for Chinese patients with Fabry disease, providing a treatment option to manage the condition.
What could go wrong: This is a small, single-arm study with only 22 participants, so results may not apply to all patients. As an enzyme replacement therapy, it requires ongoing infusions and does not cure the disease.

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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 :1560001

Shanghai, 200025, China
Investigational Site Number :1560002

Beijing, 100730, China
Investigational Site Number :1560003

Beijing, 100034, China
Investigational Site Number :1560004

Shanghai, 201102, China
Investigational Site Number :1560005

Wuhan, 016040, China
Investigational Site Number :1560006

Taiyuan, 030001, China

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