ClinicalTrials.gov record NCT06880250

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

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Sep 17, 2026

Open-label Comparative Cohort Non-interventional Epidemiological Study of the Efficacy and Safety of Enzyme Replacement Therapy in Russian Patients With Fabry Disease

About this page. This page shows the study's official ClinicalTrials.gov record ↗, with added help to make it easier to understand. We explain medical terms, highlight key details in plain language, and suggest questions you may want to ask the study team.

The record is periodically copied from ClinicalTrials.gov and may lag behind changes. AI summaries are checked by software, not a clinician.Read moreShow less

The official record is written by the study sponsor and published on ClinicalTrials.gov. It may not always reflect the latest changes. Before making any decisions, please confirm important information with the study team and your own healthcare team.

Where you see "AI summary", AI was used to create a plain-language explanation and software checked it against the source. It has not been reviewed by a clinician, so it may contain mistakes or leave out important details.

StatusEnrolling by invitationThe study enrols only people the study team invites, for example patients already at a particular clinic. It is not open to the public.

Study typeObservationalA study in which researchers do not assign participants to a treatment or other intervention. They observe or measure what happens, which may include study visits, tests, questionnaires or a review of medical records.

Age8 years old to 65 years old

SexAll

LocationRussia

AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This observational study follows people with Fabry disease who are already receiving enzyme replacement therapy (ERT). Participants form three cohorts based on the ERT product (Fabagal®, Fabrazyme®, Replagal®). Doctors provide 12 months of past medical data and then observe patients prospectively for 6 months (Fabrazyme and Replagal) or up to 18 months (Fabagal). Data are collected from routine care.

What it is studying. The study measures changes in a blood biomarker called lyso-Gb3 (in dried blood spots) as the primary outcome. It also looks at α‑Galactosidase (α‑GAL) activity, kidney function (GFR), neuropathic pain level (a simple verbal scale), and the occurrence of major clinical events such as cardiac rhythm problems, heart failure, stroke, start of kidney replacement therapy, and death. Patients are observed while continuing the ERT prescribed by their doctors; the three available drugs form separate cohorts for comparison.

What the researchers hope to learn. To describe how enzyme replacement therapy relates to lyso-Gb3 levels over time; to measure changes in α‑GAL activity, kidney function (GFR), neuropathic pain scores, and the rate of significant clinical outcomes (cardiac rhythm disturbances, heart failure, stroke, initiation of renal replacement therapy, and death).

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

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.

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

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.

Study description and what participation involves

The study's detailed description, as published on ClinicalTrials.gov.

Detailed description from the registry

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).

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

Your treating doctor will provide data from your regular care. The study collects 12 months of medical records before enrollment (retrospective) and then follows you prospectively—usually for 6 months, with people on Fabagal® followed up to 18 months. During follow-up the doctor records routine tests and assessments, including dried blood spot measurements (lyso-Gb3 and α‑GAL activity), kidney function (GFR), neuropathic pain scores, and any significant clinical events (heart rhythm problems, heart failure, stroke, start of renal replacement therapy, or death). Data are collected as part of ordinary medical care and documented by the physician.

How the study is structured. An observational, open-label comparative cohort study. Doctors provide retrospective data for the 12 months before enrollment. Patients already on one of three ERT products are then followed prospectively: 6 months of follow-up for the Fabrazyme and Replagal cohorts, and up to 18 months for the Fabagal cohort. No study treatment is assigned—data are collected during routine medical care.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

This is an observational study, so the study itself gives no treatment. What would taking part involve for me, and would it change my usual care?
The registry says participants are enrolled by invitation. Who decides who is invited, and how?
The listed sites are in Russia. Is any of them reachable for me, and does the study help with travel?
Which of the eligibility criteria would need to be checked with tests before I could join?

More on Fabry disease

Clinical trials47 trials listed for Fabry diseaseEvery trial the registry lists for Fabry disease, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in Fabry disease researchLatest monthly update: September 2026. Includes: SGLT2 outcome link. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start: Mar 24, 2025
Primary completion: January 2027 (estimated)
Study completion: January 2027 (estimated)
Target enrollment: 100 participants (estimated)
Last updated on registry: Dec 31, 2025
Data verified: Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor: NPO Petrovax

Terms used on this page · explanations by MedicalUpdates.ai

informed consent: The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
legal representative: A person who is legally allowed to make decisions, including consent to research, on behalf of someone who cannot consent for themselves.
Inclusion Criteria: The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
Exclusion Criteria: The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
enrollment: The number of participants a study plans to include, or has included. 'Estimated' means the planned number; 'actual' means the number who joined.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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