Low dose corticosteroids adjacent to enzyme replacement therapy or chaperon therapy in patients with cardiac manifestation of Fabry disease
Key facts
- Where
- Katowice és Lodz, LengyelországSee the 2 sites
- Phase
- 3. fázis
Where in Lengyelország
- Gornoslaskie Centrum Medyczne Im Prof. Leszka Gieca Sląskiego Uniwersytetu Medycznego W KatowicachKatowice, LengyelországTaking people
- Samodzielny Publiczny Zaklad Opieki Zdrowotnej Centralny Szpital Kliniczny Uniwersytetu Medycznego W LodziLodz, LengyelországTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Medical University Of Lodz
- Trial code
- EU 2025-521408-24-00
- Record updated
- 2026. november 4.
- Original description
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.Read full original descriptionShow less
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
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.
Inclusion: 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).
Exclusion: 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; 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; eGFR<30ml/m2/min; Age < 18 years at the time of screening; 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.