2025-521408-24-00RecruitingPhase 3
Low dose corticosteroids adjacent to enzyme replacement therapy or chaperon therapy in patients with cardiac manifestation of Fabry disease
Low dose corticosteroids adjacent to enzyme replacement therapy or chaperon therapy in patients with cardiac manifestation of Fabry disease – prospective randomized controlled phase III trial
Source: EU CTIS (euclinicaltrials.eu) · Last updated 4 Nov 2026
Brief summary (registry, in English)
The main objective of the study is to evaluate whether the use of prednisone - as an adjunctive treatment to standard therapy (ERT or CHT) - can effectively improve cardiac function by reducing myocardial inflammation, considered one of the key pathological mechanisms in Fabry cardiomyopathy.
- Status (registry)
- Recruiting
- Phase (registry)
- Phase 3
- Study type
- Interventional
- Enrollment
- 40 (estimated)
- First submitted
- 30 Jun 2025
- Start date
- 5 Mar 2026
- Lead sponsor
- Medical University Of Lodz
- Diseases
- Fabry disease
- EU CT number
- 2025-521408-24-00
What you commit to
- Study type: Interventional · Phase 3
- Planned participants: 40
- 2 sites in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of EU CTIS (euclinicaltrials.eu). It describes the study design.
AI-kivonat: who the study is looking for
Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.
The trial is looking for adults with Fabry disease who already get enzyme replacement therapy or migalastat and who have signs of heart involvement. It tests low dose prednisone added to that treatment.
- Age
- 18 years or older
- Sex
- All sexes
- Disease stage
- Fabry disease with heart involvement: at least one abnormal finding on ECG, heart ultrasound or heart MRI, and troponin T above 14 ng/l
- Earlier treatment
- Must be on enzyme replacement therapy (agalsidase alfa or agalsidase beta) or chaperone therapy with migalastat. Must not have taken variable-dose corticosteroids for other reasons less than 3 months before joining.
- Biomarker or test result
- High-sensitivity troponin T level above 14 ng/l
Main reasons for not taking part
- Pregnancy or breastfeeding
- eGFR below 30 ml/m2/min
- Diabetes
- Cataracts
- Stomach or duodenal ulcer, or gastrointestinal bleeding in the past 12 months
Read by machine on 9 Nov 2026.
Who cannot take part
Copied verbatim from EU CTIS (euclinicaltrials.eu) and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Concomitant treatment with variable-dose corticosteroids for other indications less than 3 months prior to study inclusion
- Planned attenuated vaccine
- Episode of gastrointestinal bleeding within 12 months prior to starting treatment
- Patient not treated with enzyme replacement therapy or chaperone therapy
Other illness
- Hypersensitivity to any component of the test drug compound
- Diagnosis of mental illness
- Diabetes
- Peptic ulcer disease of the stomach and/or duodenum
- Chronic and/or systemic fungal infection
- Coexistence with autoimmune disease
Lab values
- eGFR<30ml/m2/min
Age
- Age < 18 years at the time of screening
Other
- Reluctance to adhere to a scheduled schedule of visits
- Pregnancy (for women)
- Lactation (for women)
- Cataracts
- Inability to give informed consent to participate in the study
Who the study is looking for
- Giving informed consent
- Age at least 18 years on the day of the screening visit
- Ongoing treatment for Fabry disease: Enzyme replacement therapy with agalsidase alfa or agalsidase beta, or Chaperone therapy with migalastat
- Cardiac involvement in Fabry disease (meeting at least one of the following criteria a-c): a. electrocardiographic abnormalities defined as: - short PR (less than 120 ms) or - long PR (200 ms or more) or - bradycardia (heart rate less than <60 beats per minute) or - chronotropic failure or - repolarization abnormalities. b. echocardiographic abnormalities defined as: - left ventricular hypertrophy (wall thickness of 12 mm or more), or - reduced global longitudinal strain (GLS<-16%), or - dilatation of the aortic annulus >22 mm/m2, or - thickening of the mitral and aortic valve leaflets (>5 mm and 2 mm, respectively) with at least mild valvular regurgitation. c. cardiac MRI abnormalities defined as: - left ventricular hypertrophy defined as LVMI>2SD above the mean based on age- and gender-specific reference ranges, or - papillary muscle hypertrophy, or - the presence of late gadolinium enhancement (LGE).
- High-sensitivity troponin T level above >14 ng/l
- Contraceptive readiness (for women)
Age in the registry: 18 years to - · Sex: All · Does not accept healthy volunteers
Sites (2)
Sites in registry order. Save your city in your profile to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Gornoslaskie Centrum Medyczne Im Prof. Leszka Gieca Sląskiego Uniwersytetu Medycznego W Katowicach | Katowice | Poland | Recruiting |
| Samodzielny Publiczny Zaklad Opieki Zdrowotnej Centralny Szpital Kliniczny Uniwersytetu Medycznego W Lodzi | Lodz | Poland | Recruiting |
This page is information, not medical advice. Discuss any trial with your doctor.