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
Overview
- Phase
- Not Applicable
- Status
- Recruiting
- Sponsor
- Takeda
- Enrollment
- 200
- Locations
- 18
- Primary Endpoint
- Annualized Rate of Change in Left Ventricular Mass Index (LVMI)
Overview
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.
Study Design
- Study Type
- Observational
- Observational Model
- Cohort
- Time Perspective
- Prospective
Eligibility Criteria
- Ages
- 7 Years to — (Child, Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- Not provided
Exclusion Criteria
- Not provided
Outcomes
Primary Outcomes
Annualized Rate of Change in Left Ventricular Mass Index (LVMI)
Time Frame: Up to 18 months
The annualized rate of change in LVMI will be estimated using linear mixed effects model.
Annualized Rate of Change in Estimated Glomerular Filtration Rate (eGFR)
Time Frame: Up to 18 months
Secondary Outcomes
- 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)
- Annualized Rate of Change in Urinary Albumin to Creatinine Ratio (UACR)(Up to 18 months)
- Annualized Rate of Change in Urine Protein Creatine Ratio (UPCR)(Up to 18 months)
- Change From Baseline Over Time in eGFR(Baseline, up to 18 months)
- Change From Baseline Over Time in UACR(Baseline, up to 18 months)
- Change From Baseline Over Time in UPCR(Baseline, up to 18 months)
- Change From Baseline Over Time in 24-hour (h) Urine Protein(Baseline, up to 18 months)
- Change From Baseline Over Time in 36-Item Short Form Health Survey (SF-36)(Baseline, up to 18 months)
- Change From Baseline Over Time in Mainz Severity Score Index (MSSI)(Baseline, up to 18 months)
- Number of Participants With Clinically Significant Adverse Events (AEs) With Agalsidase Alfa Treatment(Up to 18 months)