Efficacy and Safety of Enzyme Replacement Therapy in Patients With Fabry Disease

Sponsor
NPO Petrovax
Study ID
NCT06880250
Status
Enrolling By Invitation

Conditions

Eligibility Criteria

Sex
ALL
Age
8 Years - 65 Years
Healthy Volunteers
Not accepted

Study Details

The goal of this observational study is to learn how enzyme replacement therapy works in patients with Fabry disease. The main question it aims to answer is: What medical problems do participants have? Participants already on enzyme replacement therapy as part of their regular medical care for Fabry disease will be observed as usual. Their data will be collected by their doctor.

Key Dates

Start date
Mar 24, 2025
Status verified
May 2025
Primary completion
Jan 31, 2027
Completion
Jan 31, 2027

Study Design

Enrollment
100 participants (estimated)

Arms

  • Arm: Fabagal
    Patients taking Fabagal® as the enzyme replacement therapy
  • Arm: Fabrazyme
    Patients taking Fabrazyme® as the enzyme replacement therapy
  • Arm: Replagal
    Patients taking Replagal® as the enzyme replacement therapy

Primary Outcome Measure

Change from Baseline in Lyso-Gb3 Level [ Time Frame: From 12 months before enrollment up to 6 months (for Fabrazyme and Replagal cohorts) or up to 18 months (for Fabagal cohort) of observation ]

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