A Study of Agalsidase Alfa Enyzme Replacement Therapy in Chinese Children and Adults With Fabry Disease
- Conditions
- Fabry Disease
- Registration Number
- NCT07187440
- Lead Sponsor
- Takeda
- 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- NOT_YET_RECRUITING
- Sex
- All
- Target Recruitment
- 200
Not provided
Not provided
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Annualized Rate of Change in Left Ventricular Mass Index (LVMI) 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) Up to 18 months
- Secondary Outcome Measures
Name Time Method 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 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.
Change From Baseline Over Time in Mainz Severity Score Index (MSSI) Baseline, up to 18 months 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.Number of Participants With Clinically Significant Adverse Events (AEs) With Agalsidase Alfa Treatment Up to 18 months
Trial Locations
- Locations (18)
Peking University First Hospital
🇨🇳Beijing, China
Peking University Third Hospital
🇨🇳Beijing, China
Peking Union Medical College Hospital
🇨🇳Beijing, China
West China Hospital of Sichuan University
🇨🇳Chengdu, China
Fuzhou University Provincial Affiliated Hospital
🇨🇳Fuzhou, China
The Second Affiliated Hospital ZheJiang University School Of Medicine
🇨🇳Hangzhou, China
The Second Affiliated Hospital of Harbin Medical University
🇨🇳Harbin, China
The First Affiliated Hospital Of USTC (AnHui Provincial Hospital)
🇨🇳Hefei, China
Affiliated Drum Tower Hospital, Medical School of Nanjing University
🇨🇳Nanjing, China
The First Affiliated Hospital With NanJing Medical University(JiangSu Province Hospital)
🇨🇳Nanjing, China
Scroll for more (8 remaining)Peking University First Hospital🇨🇳Beijing, ChinaSite ContactContact13681050902kmmawei@sina.comWei MaPrincipal Investigator