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Low dose corticosteroids adjacent to enzyme replacement therapy or chaperon therapy in patients with cardiac manifestation of Fabry disease
EU CTIS ID: 2025-521408-24-00
What this study is testing
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.
- Therapeutic confirmatory (Phase III)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- 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)
You likely can't join if
- Age < 18 years at the time of screening
- Concomitant treatment with variable-dose corticosteroids for other indications less than 3 months prior to study inclusion
- Hypersensitivity to any component of the test drug compound
- Diagnosis of mental illness
- Reluctance to adhere to a scheduled schedule of visits
- Pregnancy (for women)
See the full eligibility criteria
- 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 < 18 years at the time of screening
- Concomitant treatment with variable-dose corticosteroids for other indications less than 3 months prior to study inclusion
- Hypersensitivity to any component of the test drug compound
- Diagnosis of mental illness
- Reluctance to adhere to a scheduled schedule of visits
- Pregnancy (for women)
- Lactation (for women)
- eGFR<30ml/m2/min
- Planned attenuated vaccine
- Diabetes
- Cataracts
- Peptic ulcer disease of the stomach and/or duodenum
- Episode of gastrointestinal bleeding within 12 months prior to starting treatment
- Inability to give informed consent to participate in the study
- Chronic and/or systemic fungal infection
- Patient not treated with enzyme replacement therapy or chaperone therapy
- Coexistence with autoimmune disease
The study team makes the final eligibility decision.
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18-64 years, 65+ years. The study team makes the final eligibility decision.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.