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.
Study Type
OBSERVATIONAL
Enrollment
200
This is a non-interventional study
The First Affiliated Hospital Of USTC (AnHui Provincial Hospital)
Hefei, Anhui, China
NOT_YET_RECRUITINGPeking University First Hospital
Beijing, Beijing Municipality, China
NOT_YET_RECRUITINGPeking University Third Hospital
Beijing, Beijing Municipality, China
RECRUITINGPeking Union Medical College Hospital
Beijing, Beijing Municipality, China
RECRUITINGFuzhou University Provincial Affiliated Hospital
Fuzhou, Fujian, China
RECRUITINGThe Second Affiliated Hospital ZheJiang University School Of Medicine
Hangzhou, Guangdong, China
NOT_YET_RECRUITINGShanTou Central Hospital
Shantou, Guangdong, China
RECRUITINGThe University Of Hong Kong-ShenZhen Hospital
Shenzhen, Guangdong, China
NOT_YET_RECRUITINGThe Second Hospital of Hebei Medical University
Shijiazhuang, Hebei, China
RECRUITINGThe Second Affiliated Hospital of Harbin Medical University
Harbin, Heilongjiang, China
NOT_YET_RECRUITING...and 8 more locations
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.
Time frame: Up to 18 months
Annualized Rate of Change in Estimated Glomerular Filtration Rate (eGFR)
Time frame: Up to 18 months
Annualized Rate of Change in Left Ventricular Posterior Wall Dimensions (LVPWD)
Time frame: Up to 18 months
Annualized Rate of Change in Ejection Fraction (EF)
Time frame: Up to 18 months
Change From Baseline Over Time in LVMI
Time frame: Baseline, up to 18 months
Change From Baseline Over Time in LVPWD
Time frame: Baseline, up to 18 months
Change From Baseline Over Time in EF
Time frame: Baseline, up to 18 months
Annualized Rate of Change in Urinary Albumin to Creatinine Ratio (UACR)
Time frame: Up to 18 months
Annualized Rate of Change in Urine Protein Creatine Ratio (UPCR)
Time frame: Up to 18 months
Change From Baseline Over Time in eGFR
Time frame: Baseline, up to 18 months
Change From Baseline Over Time in UACR
Time frame: Baseline, up to 18 months
Change From Baseline Over Time in UPCR
Time frame: Baseline, up to 18 months
Change From Baseline Over Time in 24-hour (h) Urine Protein
Time frame: Baseline, up to 18 months
Change From Baseline Over Time in 36-Item Short Form Health Survey (SF-36)
SF-36 is a questionnaire that evaluates a person's HRQOL. SF-36 includes 36 questions related to 8 health dimensions: physical functioning, role-physical (role limitations due to physical health problems), bodily pain, general health, vitality (energy/fatigue), social functioning, role-emotional (role limitations due to emotional problems), and mental health. Based on these 4 scales (physical functioning, role-physical, bodily pain, general health), the physical component summary (PCS) score is generated which ranges between 0 and 100, with higher scores indicating a better quality of life. A positive change from Baseline indicates improvement.
Time frame: Baseline, up to 18 months
Change From Baseline Over Time in Mainz Severity Score Index (MSSI)
MSSI is an instrument for quantifying the overall severity of the signs and symptoms of Fabry disease. The MSSI assigns scores based on the presence and severity of signs and symptoms in four areas: general, neurologic, cardiovascular, and renal. Each of the signs and symptoms is weighted in accordance with its relationship to morbidity. MSSI scoring ranges from 0 (healthy) to 76 (maximum severity), and it is divided into severity bands of mild (\<20), moderate (20-40), and severe (\>40) affliction. Higher score indicates more severity.
Time frame: Baseline, up to 18 months
Number of Participants With Clinically Significant Adverse Events (AEs) With Agalsidase Alfa Treatment
Time frame: Up to 18 months
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