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

December 24, 2025 updated by: NPO Petrovax

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

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.

Study Overview

Status

Enrolling by invitation

Conditions

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).

Study Type

Observational

Enrollment (Estimated)

100

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Russia
      • Astrakhan, Russia
        • #13
      • Barnaul, Russia
        • #27
      • Borovichi, Russia
        • #7
      • Chelyabinsk, Russia
        • #6
      • Grozny, Russia
        • #21
      • Gubkin, Russia
        • #16
      • Irkutsk, Russia
        • #26
      • Ivanovo, Russia
        • #12
      • Kaa-Khem, Russia
        • #19
      • Krasnodar, Russia
        • #10
      • Kurgan, Russia
        • #18
      • Omsk, Russia
        • #17
      • Orenburg, Russia
        • #15
      • Perm, Russia
        • #9
      • Polevskoy, Russia
        • #23
      • Rostov-on-Don, Russia
        • #2
      • Rostov-on-Don, Russia
        • #4
      • Ryazan, Russia
        • #24
      • Saint Petersburg, Russia
        • #8
      • Simferopol, Russia
        • #3
      • Stavropol, Russia
        • #22
      • Ufa, Russia
        • #11
      • Voronezh, Russia
        • #5

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

Russian patients

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort
Fabagal
Patients taking Fabagal® as the enzyme replacement therapy
Fabrazyme
Patients taking Fabrazyme® as the enzyme replacement therapy
Replagal
Patients taking Replagal® as the enzyme replacement therapy

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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
Level of globotriaosylsphingosine (lyso-Gb3) is measured in dried blood spots.
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

Outcome Measure
Measure Description
Time Frame
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
Level of α-Galactosidase (α-GAL) activity is measured in dried blood spots.
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
Glomerular filtration rate (GFR) is measured by methods available in routine medical use.
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
Neuropathic pain is assessed according to verbal scale: no pain, mild pain, moderate pain, severe pain, very severe pain, intolerable pain. Corresponding value of 0, 2, 4, 6, 8, 10 is assigned.
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
Significant clinical outcomes include cardiac rhythm disturbances (atrial fibrillation, persistent supraventricular or ventricular tachycardia), heart failure, stroke, initiation of renal replacement therapy and death from any cause.
From enrollment up to 6 months (for Fabrazyme and Replagal cohorts) or up to 18 months (for Fabagal cohort) of observation

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

NPO Petrovax

Investigators

  • Study Chair: Sergey V. Moiseev, MD, Sechenov First Moscow State Medical University (Sechenov University)

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

March 24, 2025

Primary Completion (Estimated)

January 1, 2027

Study Completion (Estimated)

January 1, 2027

Study Registration Dates

First Submitted

March 11, 2025

First Submitted That Met QC Criteria

March 11, 2025

First Posted (Actual)

March 17, 2025

Study Record Updates

Last Update Posted (Actual)

December 31, 2025

Last Update Submitted That Met QC Criteria

December 24, 2025

Last Verified

May 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • FAVORIT

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

UNDECIDED

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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