Efficacy and Safety of Enzyme Replacement Therapy in Patients 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
- This is an observational study: the protocol does not assign a study treatment.
- Who it may be relevant to
- Registry conditions: Fabry Disease. Basic parameters: 8 years — 65 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
- Russia
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
Open-label Comparative Cohort Non-interventional Epidemiological Study of the Efficacy and Safety of Enzyme Replacement Therapy in Russian Patients With Fabry Disease
Overview
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.
Detailed description
All patients on enzyme replacement therapy could be enrolled in the study. Three medicines are currently available in Russia (Fabagal®, Fabrazyme®, Replagal®) so the patients form three cohorts.
For all patients retrospective data are collected for 12 months prior enrollment.
All patients are observed prospectively for 6 months. At the point of 6 months patients taking Fabrazyme or Replagal at the moment finish the study.
Patients on Fabagal are observed for 12 more months (up to 18 months after enrollment).
Primary outcome measures
- 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]
Secondary outcome measures (4)
- Change from Baseline in α-GAL Activity [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]
- Change from Baseline in GFR [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]
- Change from Baseline in Neuropathic Pain Level [Time frame: From enrollment up to 6 months (for Fabrazyme and Replagal cohorts) or up to 18 months (for Fabagal cohort) of observation]
- Incidence Rate of Significant Clinical Outcomes [Time frame: From enrollment up to 6 months (for Fabrazyme and Replagal cohorts) or up to 18 months (for Fabagal cohort) of observation]
Eligibility criteria
Inclusion criteria
- Men and women with a confirmed diagnosis of Fabry disease;
- Prescribed course of enzyme replacement therapy with any drug (Fabagal, Fabrazyme, Replagal);
- Age 8 - 65 years;
- Signed informed consent of the patient and/or patient's 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 patient's legal representative) from further participation in the study or medical care.
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
Russia · 23 centers
- #13 — Astrakhan
- #27 — Barnaul
- #7 — Borovichi
- #6 — Chelyabinsk
- #21 — Grozny
- #16 — Gubkin
- #26 — Irkutsk
- #12 — Ivanovo
- … and 15 more centers
Identifiers
NCT: NCT06880250 · FAVORIT