Status:
RECRUITING
A Study of Agalsidase Alfa Enyzme Replacement Therapy in Chinese Children and Adults With Fabry Disease
Lead Sponsor:
Takeda
Conditions:
Fabry Disease
Eligibility:
All Genders
7+ years
Brief Summary
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 subst...
Eligibility Criteria
Inclusion
- 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 alfa or agalsidase beta) within 12 weeks at most prior to enrolment.
- Participant who will receive ERT with agalsidase alfa in routine clinical practice settings.
- For \>= 18 years old, participant should sign the informed consent form (ICF); for 8-17 years old, participant and his (her) parents/ legally authorized representative (LAR) should both sign the ICF; for \< 8 years old, participant will give assent and his (her) parents/legally authorized representative should sign the ICF accordingly.
- Exclusion Criteria
- Participant will be excluded if they have documented New York Heart Association (NYHA) functional Class IV heart failure symptoms (Implantable cardioverter-defibrillator \[ICD\] implanted excluded), third-degree atrioventricular block (ICD implanted excluded), acute myocardial infarction within the last 3 months and severe stroke (NIH Stroke Scale \[NIHSS\] \>= to 21).
- Participant has enrolled in Fabry disease interventional clinical trial currently.
- Other situations that the investigator considers not suitable for participation in this study.
Exclusion
Key Trial Info
Start Date :
November 1 2025
Trial Type :
OBSERVATIONAL
Allocation :
ESTIMATED
End Date :
November 30 2028
Estimated Enrollment :
200 Patients enrolled
Trial Details
Trial ID
NCT07187440
Start Date
November 1 2025
End Date
November 30 2028
Last Update
January 2 2026
Active Locations (18)
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1
The First Affiliated Hospital Of USTC (AnHui Provincial Hospital)
Hefei, Anhui, China, 230001
2
Peking University First Hospital
Beijing, Beijing Municipality, China, 100034
3
Peking University Third Hospital
Beijing, Beijing Municipality, China, 100191
4
Peking Union Medical College Hospital
Beijing, Beijing Municipality, China, 100730