A Study of Agalsidase Alfa Enyzme Replacement Therapy in Chinese Children and Adults With Fabry Disease
Recruiting
Observational Study
Fabry Disease
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
No Study Drug
Researchers observe your health over time — no experimental treatment is given.
At a Glance
- Age
- 7 and older
- Sex
- Any
- Study type
- Observational
- Participants needed
- 200 (estimated)
- Sponsor
- Takeda · Industry
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About This Trial
Fabry Disease is a rare blood disorder that some people are born with. People with Fabry disease have low levels of an enzyme called alpha-galactosidase A. This enzyme helps to cut down fat-like substances. Without alpha-galactosidase A, large forms of these substances build up and clot in blood vessels. Over time, this can affect vital organs (especially the heart, kidneys, and brain) causing ser…
Trial Locations
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Eligibility Criteria
Inclusion Criteria
Participants who meet all of the following criteria are eligible for this study:
* Participant is greater than or equal to (\>= 7) years old.
* Participant with confirmed diagnosis of Fabry disease (by investigator).
* Participant never received or has received ERT (agalsidase a…
Contacts