Follows Chinese children and adults with Fabry disease on agalsidase alfa treatment
Observes routine agalsidase alfa treatment in people with Fabry disease in China.
Plain-language title generated from the registry record
Registry title: A Study of Agalsidase Alfa Enyzme Replacement Therapy in Chinese Children and Adults With Fabry Disease
Key facts
- Where
- Beijing, Chengdu, Fuzhou, Hangzhou, Harbin, Hefei, Nanjing, Shantou, Shenzhen, Shijiazhuang, Suzhou, Taiyuan, Tianjin, Xi'an, and Xi’an, ChinaSee the 18 sites
Where in China
- Peking Union Medical College HospitalBeijing, ChinaTaking people
- Peking University First HospitalBeijing, ChinaNot yet recruiting
- Peking University Third HospitalBeijing, ChinaTaking people
- West China Hospital of Sichuan UniversityChengdu, ChinaNot yet recruiting
- Fuzhou University Provincial Affiliated HospitalFuzhou, ChinaTaking people
- The Second Affiliated Hospital Zhejiang University School of MedicineHangzhou, ChinaNot yet recruiting
- The Second Affiliated Hospital of Harbin Medical UniversityHarbin, ChinaNot yet recruiting
- The First Affiliated Hospital of USTC(Anhui Provincial Hospital)Hefei, ChinaNot yet recruiting
- Affiliated Drum Tower Hospital, Medical School of Nanjing UniversityNanjing, ChinaTaking people
- The First Affiliated Hospital with Nanjing Medical University, Jiangsu Province HospitalNanjing, ChinaNot yet recruiting
- Shantou Central HospitalShantou, ChinaTaking people
- The University of Hong Kong-Shenzhen HospitalShenzhen, ChinaNot yet recruiting
- The Second Hospital of Hebei Medical UniversityShijiazhuang, ChinaTaking people
- The First Affiliated Hospital of Soochow UniversitySuzhou, ChinaTaking people
- Second hospital of Shanxi Medical UniversityTaiyuan, ChinaNot yet recruiting
- Tianjin Chest HospitalTianjin, ChinaNot yet recruiting
- The Second Affiliated Hospital of Xi'an Jiaotong UniversityXi'an, ChinaTaking people
- The First Affiliated Hospital of Xi'an Jiaotong UniversityXi’an, ChinaNot yet recruiting
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Takeda
- Trial code
- NCT07187440
- Record updated
- 2 January 2026
- Original description
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.Read full original descriptionShow less
A Prospective, Multi-center, Observational Study to Evaluate the Clinical Outcomes of Agalsidase Alfa Enzyme Replacement Therapy Among Chinese Patients With Fabry Disease in Real-world Clinical Practice
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.
Inclusion: 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: 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.