Actively Recruiting
A Prospective, Multi-center, Observational Study of Agalsidase Alfa Enzyme Replacement Therapy in Chinese Patients With Fabry Disease
Led by Takeda · Updated on 2026-01-02
200
Participants Needed
18
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Fabry Disease is a rare inherited blood disorder caused by low levels of an enzyme called alpha-galactosidase A. This enzyme normally breaks down fat-like substances, but without enough of it, these substances can build up in blood vessels and harm vital organs such as the heart, kidneys, and brain over time. This study aims to learn more about the treatment effects of agalsidase alfa (Replagal4), an enzyme replacement therapy, in Chinese children and adults with Fabry disease by observing its impact on heart and kidney function, quality of life, and safety in routine clinical use. Participants in this study will receive enzyme replacement therapy with agalsidase alfa as part of their usual clinical care in China. This is a non-interventional, observational study where no additional study-specific visits are scheduled. The treatment follows routine practice settings, and researchers will monitor participants receiving this therapy over time. During the study lasting up to 18 months, participants will be observed for changes in heart function using measures like left ventricular mass index and ejection fraction, and kidney function using estimated glomerular filtration rate and urine analyses. Quality of life assessments and monitoring of adverse events will also be conducted. The study collects data from regular clinical visits without extra procedures, aiming to understand how agalsidase alfa works and its safety in real-world treatment of Fabry disease.
CONDITIONS
Brief 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...
- Participant is 7 years old or older
- Confirmed diagnosis of Fabry disease by investigator
- Never received or received enzyme replacement therapy (agalsidase alfa or beta) within 12 weeks before enrollment
- Will receive enzyme replacement therapy with agalsidase alfa in routine clinical practice
- For ages 18 and older: must sign informed consent; for ages 8 to 17: participant and parent/legal representative must sign informed consent; for under 8 years old: participant assent and parent/legal representative consent required
You will not qualify if you...
- Documented New York Heart Association Class IV heart failure symptoms (except with implanted cardioverter-defibrillator)
- Third-degree atrioventricular block (except with implanted cardioverter-defibrillator)
- Acute myocardial infarction within the last 3 months
- Severe stroke with NIH Stroke Scale score of 21 or higher
- Currently enrolled in another Fabry disease interventional clinical trial
- Other conditions deemed unsuitable for participation by the investigator
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
Duration - Up to 18 months
Participants who undergo routine enzyme replacement therapy with agalsidase alfa are observed in routine clinical practice settings.
Regular visits as part of routine clinical care
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
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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