Enzyme replacement therapy studied for rare genetic disorder in china

NCT ID NCT05054387

First seen Oct 31, 2025 · Last updated May 16, 2026 · Updated 27 times

Summary

This study tested a drug called Fabrazyme in 22 Chinese patients with Fabry disease, a rare genetic condition that causes pain and organ damage. Patients received enzyme replacement therapy for 54 weeks to see if it was safe and helped control the disease. The main focus was on side effects, with additional checks on blood markers of the disease.

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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

Conditions

Explore the condition pages connected to this study.