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

Launched by NPO PETROVAX · Mar 11, 2025

Keywords

ClinConnect Summary

This clinical trial is studying how effective and safe enzyme replacement therapy is for people with Fabry disease. Fabry disease is a genetic condition that affects the way your body breaks down certain fats, leading to various health problems. The researchers want to learn more about the medical issues that patients experience while they are receiving this treatment. To do this, they will observe patients who are already receiving enzyme replacement therapy as part of their usual care, gathering information from their doctors.

To participate in this study, you need to be between the ages of 8 and 65 and have a confirmed diagnosis of Fabry disease. You should also be receiving enzyme replacement therapy with medications like FABAGAL, FABRAZIM, or REPLAGAL. However, if you are on kidney dialysis, critically ill, pregnant or breastfeeding, or do not wish to participate, you would not be eligible for this trial. Since this study is not yet recruiting, there’s no immediate action needed, but it’s an important step to learn more about how treatment affects patients with Fabry disease.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • Men and women with a confirmed diagnosis of Fabry disease;
• • Prescribed course of enzyme replacement therapy with any drug (FABAGAL, FABRAZIM, REPLAGAL);
• • Age 8 - 65 years;
• • Signed informed consent of the patient and/or his/her legal representative for participation in the study and processing of personal data.
• Exclusion Criteria:
• • Renal replacement therapy or kidney transplantation;
• • Critical illness unrelated to Fabry disease;
• • Pregnancy or breastfeeding;
• • Refusal of the patient (or his/her legal representative) from further participation in the study or medical care.