A Study of Agalsidase Alfa Enyzme Replacement Therapy in Chinese Children and Adults With Fabry Disease

Sponsor
Takeda
Study ID
NCT07187440
Status
Recruiting
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Conditions

Eligibility Criteria

Sex
ALL
Age
7 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • No intervention — OTHER
    This is a non-interventional study

Study Details

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.

Key Dates

Start date
Nov 1, 2025
Status verified
Dec 2025
Primary completion
Nov 30, 2028
Completion
Nov 30, 2028

Study Design

Enrollment
200 participants (estimated)

Arms

  • Arm: All Participants
    Participants with Fabry disease will receive enzyme replacement therapy (ERT) with agalsidase alfa under routine clinical practice settings.

Primary Outcome Measure

Annualized Rate of Change in Left Ventricular Mass Index (LVMI) [ Time Frame: Up to 18 months ]

Central Contacts

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