NCT07187440Recruiting
A Study of Agalsidase Alfa Enyzme Replacement Therapy in Chinese Children and Adults With Fabry Disease
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
Source: ClinicalTrials.gov · Last updated 2 Jan 2026
Brief summary (registry, in English)
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.
- Status (registry)
- Recruiting
- Study type
- Observational
- Enrollment
- 200 (estimated)
- First submitted
- 18 Sept 2025
- Start date
- 1 Nov 2025
- Primary completion date
- 30 Nov 2028
- Lead sponsor
- Takeda
- Diseases
- Fabry disease
- NCT number
- NCT07187440
What you commit to
- Study type: Observational
- Planned participants: 200
- From 1 Nov 2025 to the primary completion date, 30 Nov 2028
- 18 sites in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Other illness
- 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
- Other situations that the investigator considers not suitable for participation in this study.
Who the study is looking for
- 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.
Age in the registry: 7 years to - · Sex: All · Does not accept healthy volunteers
Sites (18)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Peking University Third Hospital Coordinator of this site? Confirm its status | Beijing | China | Recruiting |
| Peking University First Hospital Coordinator of this site? Confirm its status | Beijing | China | Not yet recruiting |
| Peking Union Medical College Hospital Coordinator of this site? Confirm its status | Beijing | China | Recruiting |
| West China Hospital of Sichuan University Coordinator of this site? Confirm its status | Chengdu | China | Not yet recruiting |
| Fuzhou University Provincial Affiliated Hospital Coordinator of this site? Confirm its status | Fuzhou | China | Recruiting |
| The Second Affiliated Hospital Zhejiang University School of Medicine Coordinator of this site? Confirm its status | Hangzhou | China | Not yet recruiting |
| The Second Affiliated Hospital of Harbin Medical University Coordinator of this site? Confirm its status | Harbin | China | Not yet recruiting |
| The First Affiliated Hospital of USTC(Anhui Provincial Hospital) Coordinator of this site? Confirm its status | Hefei | China | Not yet recruiting |
| Affiliated Drum Tower Hospital, Medical School of Nanjing University Coordinator of this site? Confirm its status | Nanjing | China | Recruiting |
| The First Affiliated Hospital with Nanjing Medical University, Jiangsu Province Hospital Coordinator of this site? Confirm its status | Nanjing | China | Not yet recruiting |
| Shantou Central Hospital Coordinator of this site? Confirm its status | Shantou | China | Recruiting |
| The University of Hong Kong-Shenzhen Hospital Coordinator of this site? Confirm its status | Shenzhen | China | Not yet recruiting |
| The Second Hospital of Hebei Medical University Coordinator of this site? Confirm its status | Shijiazhuang | China | Recruiting |
| The First Affiliated Hospital of Soochow University Coordinator of this site? Confirm its status | Suzhou | China | Recruiting |
| Second hospital of Shanxi Medical University Coordinator of this site? Confirm its status | Taiyuan | China | Not yet recruiting |
| Tianjin Chest Hospital Coordinator of this site? Confirm its status | Tianjin | China | Not yet recruiting |
| The Second Affiliated Hospital of Xi'an Jiaotong University Coordinator of this site? Confirm its status | Xi'an | China | Recruiting |
| The First Affiliated Hospital of Xi'an Jiaotong University Coordinator of this site? Confirm its status | Xi’an | China | Not yet recruiting |
This page is information, not medical advice. Discuss any trial with your doctor.