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

NCT ID: NCT06880250

Last Updated: 2025-12-31

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ENROLLING_BY_INVITATION

Total Enrollment

100 participants

Study Classification

OBSERVATIONAL

Study Start Date

2025-03-24

Study Completion Date

2027-01-31

Brief Summary

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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

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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).

Conditions

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Fabry Disease

Keywords

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Fabry ERT enzyme replacement therapy

Study Design

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Observational Model Type

COHORT

Study Time Perspective

OTHER

Study Groups

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Fabagal

Patients taking Fabagal® as the enzyme replacement therapy

No interventions assigned to this group

Fabrazyme

Patients taking Fabrazyme® as the enzyme replacement therapy

No interventions assigned to this group

Replagal

Patients taking Replagal® as the enzyme replacement therapy

No interventions assigned to this group

Eligibility Criteria

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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.
Minimum Eligible Age

8 Years

Maximum Eligible Age

65 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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NPO Petrovax

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Sergey V. Moiseev, MD

Role: STUDY_CHAIR

Sechenov First Moscow State Medical University (Sechenov University)

Locations

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#13

Astrakhan, , Russia

Site Status

#27

Barnaul, , Russia

Site Status

#7

Borovichi, , Russia

Site Status

#6

Chelyabinsk, , Russia

Site Status

#21

Grozny, , Russia

Site Status

#16

Gubkin, , Russia

Site Status

#26

Irkutsk, , Russia

Site Status

#12

Ivanovo, , Russia

Site Status

#19

Kaa-Khem, , Russia

Site Status

#10

Krasnodar, , Russia

Site Status

#18

Kurgan, , Russia

Site Status

#17

Omsk, , Russia

Site Status

#15

Orenburg, , Russia

Site Status

#9

Perm, , Russia

Site Status

#23

Polevskoy, , Russia

Site Status

#2

Rostov-on-Don, , Russia

Site Status

#4

Rostov-on-Don, , Russia

Site Status

#24

Ryazan, , Russia

Site Status

#8

Saint Petersburg, , Russia

Site Status

#3

Simferopol, , Russia

Site Status

#22

Stavropol, , Russia

Site Status

#11

Ufa, , Russia

Site Status

#5

Voronezh, , Russia

Site Status

Countries

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Russia

Other Identifiers

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FAVORIT

Identifier Type: -

Identifier Source: org_study_id