A Study of Agalsidase Alfa Enyzme Replacement Therapy in Chinese Children and Adults With Fabry Disease
ConditionFabry Disease
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age7+
SponsorTakeda
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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.
Disqualifiers
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.
Trial population
Participants with Fabry disease who receive ERT with agalsidase alfa in routine clinical practice settings in China.
Trial design
Design model
Cohort
Time perspective
Prospective
Treatments tested in this trial
No intervention
Other interventionThis is a non-interventional study
Treatment groups
200 Participants
are divided into 1 treatment groupGroup A: All Participants1 intervention
Trial outcomes
Primary outcomes
1
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 months2
Annualized Rate of Change in Estimated Glomerular Filtration Rate (eGFR)
Time frame
Up to 18 monthsSecondary outcomes
1
Annualized Rate of Change in Left Ventricular Posterior Wall Dimensions (LVPWD)
Time frame
Up to 18 months2
Annualized Rate of Change in Ejection Fraction (EF)
Time frame
Up to 18 months3
Change From Baseline Over Time in LVMI
Time frame
Baseline, up to 18 months4
Change From Baseline Over Time in LVPWD
Time frame
Baseline, up to 18 months Source ClinicalTrials.gov