A Study of Agalsidase Alfa Enyzme Replacement Therapy in Chinese Children and Adults With Fabry Disease
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: No intervention.
- Who it may be relevant to
- Registry conditions: Fabry Disease. Basic parameters: from 7 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- China
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Official title
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
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.
Interventions
- Other No intervention
This is a non-interventional study
Primary outcome measures
- Annualized Rate of Change in Left Ventricular Mass Index (LVMI) [Time frame: Up to 18 months]
- Annualized Rate of Change in Estimated Glomerular Filtration Rate (eGFR) [Time frame: Up to 18 months]
Secondary outcome measures (12)
- 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) [Time frame: Baseline, up to 18 months]
Eligibility criteria
Inclusion criteria
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.
Exclusion criteria
- 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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Cohort
Study locations
China · 18 centers
- The First Affiliated Hospital Of USTC (AnHui Provincial Hospital) — Hefei
- Peking University First Hospital — Beijing
- Peking University Third Hospital — Beijing
- Peking Union Medical College Hospital — Beijing
- Fuzhou University Provincial Affiliated Hospital — Fuzhou
- The Second Affiliated Hospital ZheJiang University School Of Medicine — Hangzhou
- ShanTou Central Hospital — Shantou
- The University Of Hong Kong-ShenZhen Hospital — Shenzhen
- … and 10 more centers
Identifiers
NCT: NCT07187440 · TAK-675-4013