Actively Recruiting
A Study of Agalsidase Alfa Enyzme Replacement Therapy in Chinese Children and Adults With Fabry Disease
Led by Takeda · Updated on 2026-01-02
200
Participants Needed
18
Research Sites
160 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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 serious health problems with advancing age. Agalsidase alfa (Replagal®) is a human enzyme made in the laboratory and may provide higher levels of alpha-galactosidase A. Replagal® works the same way as natural alpha-galactosidase A does. The main aim of this study is to learn more about the treatment with Replagal® in Chinese children and adults with Fabry disease. The study aims to assess the heart and kidney function in people with Fabry disease who are routinely treated with Replagal®. Other aims are to learn about the change in heart and kidney function, impact on quality of life, how the treatment with Replagal® works for people with Fabry Disease, and how safe the treatment with Replagal® is in routine real-world settings. Participants will receive with Replagal® per the routine treatment settings in China. No study-specific visits to the clinical are scheduled.
CONDITIONS
Official Title
A Study of Agalsidase Alfa Enyzme Replacement Therapy in Chinese Children and Adults With Fabry Disease
Who Can Participate
Eligibility Criteria
You may qualify if you...
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.
You will not qualify if you...
History of severe allergic reactions to study medication Currently pregnant or breastfeeding Recent participation in another clinical trial within the last 30 days Presence of uncontrolled medical conditions that could affect safety
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 18 locations
1
The First Affiliated Hospital Of USTC (AnHui Provincial Hospital)
Hefei, Anhui, China, 230001
Not Yet Recruiting
2
Peking University First Hospital
Beijing, Beijing Municipality, China, 100034
Not Yet Recruiting
3
Peking University Third Hospital
Beijing, Beijing Municipality, China, 100191
Actively Recruiting
4
Peking Union Medical College Hospital
Beijing, Beijing Municipality, China, 100730
Actively Recruiting
5
Fuzhou University Provincial Affiliated Hospital
Fuzhou, Fujian, China, 350001
Actively Recruiting
6
The Second Affiliated Hospital ZheJiang University School Of Medicine
Hangzhou, Guangdong, China, 310009
Not Yet Recruiting
7
ShanTou Central Hospital
Shantou, Guangdong, China, 515031
Actively Recruiting
8
The University Of Hong Kong-ShenZhen Hospital
Shenzhen, Guangdong, China, 518053
Not Yet Recruiting
9
The Second Hospital of Hebei Medical University
Shijiazhuang, Hebei, China, '050000
Actively Recruiting
10
The Second Affiliated Hospital of Harbin Medical University
Harbin, Heilongjiang, China, 150086
Not Yet Recruiting
11
Affiliated Drum Tower Hospital, Medical School of Nanjing University
Nanjing, Jiangsu, China, 210008
Actively Recruiting
12
The First Affiliated Hospital With NanJing Medical University(JiangSu Province Hospital)
Nanjing, Jiangsu, China, 210029
Not Yet Recruiting
13
The First Affiliated Hospital Of Soochow University
Suzhou, Jiangsu, China, 215006
Actively Recruiting
14
The Second Affiliated Hospital of Xi 'an Jiaotong University
Xi'an, Shaanxi, China, 710004
Actively Recruiting
15
The First Affiliated Hospital of Xi 'an Jiaotong University
Xi'an, Shaanxi, China, 710061
Not Yet Recruiting
16
Second Hospital Of ShanXi Medical University
Taiyuan, Shanxi, China, '030001
Not Yet Recruiting
17
West China Hospital of Sichuan University
Chengdu, Sichuan, China, 610041
Not Yet Recruiting
18
TianJin Chest Hospital
Tianjin, Tianjin Municipality, China, 300222
Not Yet Recruiting
Research Team
T
Takeda Contact
CONTACT
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
1
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