Fabry DiseaseFAVORITNCT06880250
Efficacy and Safety of Enzyme Replacement Therapy in Patients With Fabry Disease
An observational study of Fabry Disease, run by NPO Petrovax at 23 sites in Russia.
Trial summary
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… Read more about this study
- Who is it for?
- Ages 8 to 65 years
- Conditions the study is for
- Fabry Disease
- How many people
- 100 participants
- Where
- 23 sitesRussia
- Study dates
- 24 Mar 2025to Jan 2027
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 8 to 65 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Russian patients
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
23 sites in 1 country. Contact the study team or a site near you to ask about taking part.
23 sites in 1 country
Russia23 sites
#13
Astrakhan
#27
Barnaul
#7
Borovichi
#6
Chelyabinsk
#21
Grozny
#16
Gubkin
#26
Irkutsk
#12
Ivanovo
#19
Kaa-Khem
#10
Krasnodar
#18
Kurgan
#17
Omsk
#15
Orenburg
#9
Perm
#23
Polevskoy
#2
Rostov-on-Don
#4
Rostov-on-Don
#24
Ryazan
#8
Saint Petersburg
#3
Simferopol
#22
Stavropol
#11
Ufa
#5
Voronezh
About this study
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
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.
Read the 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).
- Conditions
- Fabry Disease
- Keywords
- Fabry, ERT, enzyme replacement therapy
- Medical subject headings
- Fabry Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| 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 being measured
The primary outcome is the main question the study is designed to answer.
Change from Baseline in Lyso-Gb3 Level
Level of globotriaosylsphingosine (lyso-Gb3) is measured in dried blood spots.
Measured: 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 outcomes (4)
Change from Baseline in α-GAL Activity
Level of α-Galactosidase (α-GAL) activity is measured in dried blood spots.
Measured: 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
Glomerular filtration rate (GFR) is measured by methods available in routine medical use.
Measured: 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
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.
Measured: 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
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.
Measured: From enrollment up to 6 months (for Fabrazyme and Replagal cohorts) or up to 18 months (for Fabagal cohort) of observation
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Mar 2025
- Study start24 Mar 2025
- Primary completionJan 2027 (estimated)
- Study completionJan 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Fabry DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Other
- Enrolment
- 100 participants (estimated)
Sponsor and investigators
- Lead sponsor
- NPO Petrovax
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Sergey V. Moiseev, MD, Study chair, Sechenov First Moscow State Medical University (Sechenov University)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06880250
- Sponsor's protocol ID
- FAVORIT
- Acronym
- FAVORIT
- Status last verified
- May 2025
- First submitted
- 11 Mar 2025
- Last updated
- 31 Dec 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 31 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT06880250
- Acronym / sponsor ID
- FAVORIT / FAVORIT
- Conditions
- Fabry Disease
- Keywords
- Fabry, ERT, enzyme replacement therapy
- Study type
- Observational
- Recruitment status
- Enrolling by invitation
- Enrolment
- 100 participants (estimated)
- Lead sponsor
- NPO PetrovaxIndustry (pharma, biotech, devices)
- Study start
- 24 Mar 2025 (actual)
- Primary completion
- Jan 2027 (estimated)
- Study completion
- Jan 2027 (estimated)
- First posted
- 17 Mar 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Study design
- Observational model
- Cohort
- Time perspective
- Other
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| 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 |
Who may qualify
- Ages
- 8 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Russian patients
Key inclusion criteria (4 listed)
- 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.
Key exclusion criteria (4 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
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.
4 secondary outcomes
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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 Dec 2025.
- Individual participant dataSharing planned: Undecided
Related trials for Fabry Disease
RecruitingPhase 3
A Study to Learn How Well Lucerastat Works and How Safe it is in Untreated Adult Male Participants With Fabry Disease
Fabry Disease
RecruitingObservational study
A Study of Patients With Fabry Disease (US Specific)
Fabry Disease
RecruitingObservational study
A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding
Fabry Disease
Active, not recruiting
Evaluation of the Effect of a Low-FODMAP Diet on Gastrointestinal Symptoms and Inflammatory Markers in Fabry Patients
Fabry Disease
RecruitingPhase 3
A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants
Fabry Disease
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Sergey V. Moiseev, MD study chair
Sechenov First Moscow State Medical University (Sechenov University)
23 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Fabry Disease, run by NPO Petrovax at 23 sites in Russia.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: enrolling by invitation, as of the sponsor's last update on 31 Dec 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 23 sites in Russia. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 31 Dec 2025.