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
1 other identifier
observational
200
1 country
18
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 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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for all trials
Started Nov 2025
Typical duration for all trials
18 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
September 18, 2025
CompletedFirst Posted
Study publicly available on registry
September 23, 2025
CompletedStudy Start
First participant enrolled
November 1, 2025
CompletedPrimary Completion
Last participant's last visit for primary outcome
November 30, 2028
ExpectedStudy Completion
Last participant's last visit for all outcomes
November 30, 2028
January 2, 2026
December 1, 2025
3.1 years
September 18, 2025
December 29, 2025
Conditions
Keywords
Outcome Measures
Primary Outcomes (2)
Annualized Rate of Change in Left Ventricular Mass Index (LVMI)
The annualized rate of change in LVMI will be estimated using linear mixed effects model.
Up to 18 months
Annualized Rate of Change in Estimated Glomerular Filtration Rate (eGFR)
Up to 18 months
Secondary Outcomes (14)
Annualized Rate of Change in Left Ventricular Posterior Wall Dimensions (LVPWD)
Up to 18 months
Annualized Rate of Change in Ejection Fraction (EF)
Up to 18 months
Change From Baseline Over Time in LVMI
Baseline, up to 18 months
Change From Baseline Over Time in LVPWD
Baseline, up to 18 months
Change From Baseline Over Time in EF
Baseline, up to 18 months
- +9 more secondary outcomes
Study Arms (1)
All Participants
Participants with Fabry disease will receive enzyme replacement therapy (ERT) with agalsidase alfa under routine clinical practice settings.
Interventions
Eligibility Criteria
Participants with Fabry disease who receive ERT with agalsidase alfa in routine clinical practice settings in China.
You may qualify if:
- 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.
You may not qualify if:
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Takedalead
Study Sites (18)
The First Affiliated Hospital Of USTC (AnHui Provincial Hospital)
Hefei, Anhui, 230001, China
Peking University First Hospital
Beijing, Beijing Municipality, 100034, China
Peking University Third Hospital
Beijing, Beijing Municipality, 100191, China
Peking Union Medical College Hospital
Beijing, Beijing Municipality, 100730, China
Fuzhou University Provincial Affiliated Hospital
Fuzhou, Fujian, 350001, China
The Second Affiliated Hospital ZheJiang University School Of Medicine
Hangzhou, Guangdong, 310009, China
ShanTou Central Hospital
Shantou, Guangdong, 515031, China
The University Of Hong Kong-ShenZhen Hospital
Shenzhen, Guangdong, 518053, China
The Second Hospital of Hebei Medical University
Shijiazhuang, Hebei, '050000, China
The Second Affiliated Hospital of Harbin Medical University
Harbin, Heilongjiang, 150086, China
Affiliated Drum Tower Hospital, Medical School of Nanjing University
Nanjing, Jiangsu, 210008, China
The First Affiliated Hospital With NanJing Medical University(JiangSu Province Hospital)
Nanjing, Jiangsu, 210029, China
The First Affiliated Hospital Of Soochow University
Suzhou, Jiangsu, 215006, China
The Second Affiliated Hospital of Xi 'an Jiaotong University
Xi'an, Shaanxi, 710004, China
The First Affiliated Hospital of Xi 'an Jiaotong University
Xi'an, Shaanxi, 710061, China
Second Hospital Of ShanXi Medical University
Taiyuan, Shanxi, '030001, China
West China Hospital of Sichuan University
Chengdu, Sichuan, 610041, China
TianJin Chest Hospital
Tianjin, Tianjin Municipality, 300222, China
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Study Director
Takeda
Central Study Contacts
Study Design
- Study Type
- observational
- Observational Model
- COHORT
- Time Perspective
- PROSPECTIVE
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
September 18, 2025
First Posted
September 23, 2025
Study Start
November 1, 2025
Primary Completion (Estimated)
November 30, 2028
Study Completion (Estimated)
November 30, 2028
Last Updated
January 2, 2026
Record last verified: 2025-12
Data Sharing
- IPD Sharing
- Will share
- Shared Documents
- STUDY PROTOCOL, SAP, ICF, CSR
- Access Criteria
- IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.
Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.