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

Conditions

Fabry disease

Brief summary

Reduction in high-sensitivity troponin T (hsTnT) levels by 50% at the end of GCS therapy compared to levels at the visit before starting GCS.

Detailed description

Combined clinical outcome (death + cardiac arrest + unplanned cardiac hospitalization), Quality of life measured by SF-36v.2; EQ-5D and KCCQ, Fabry disease progression assessed by dedicated Mainz Severity Score Index (MSSI), DS3 and FASTEX scores, Assessment of functional capacity using cardiopulmonary exercise testing (CPET), 6-minute walk test (6MWT) and NYHA classification, Electrocardiographic abnormalities (brady- and tachyarrhythmias, autonomic tension), Echocardiographic assessment (morphological and functional evaluation of the heart), Cardiac MRI abnormalities (left ventricular mass, inflammation and fibrosis), Biochemical biomarkers of the heart (CK-MB, NT-pro-BNP), Substrate levels of FD-specific biomarkers ( Gb3 in urine and Lyso-Gb3 in serum), Antidrug antibody (ADA) levels in patients on ERT, Levels of inflammatory cytokines (CRP, IL-1beta, IL-6, IL-8, IL-10, IL-12, TNF alpha, SAA), Assessment of renal function with glomerular filtration rate (eGFR), albumin/creatinine ratio (ACR), FGF2 and VEGFA

Related papers

No related papers found yet.

Interventions

DRUGPREDNISONE

Eligibility

Sex/Gender

All

Age

18 Years to No maximum

Design outcomes

Primary

Measure	Time frame
Reduction in high-sensitivity troponin T (hsTnT) levels by 50% at the end of GCS therapy compared to levels at the visit before starting GCS.	—

Secondary

Measure	Time frame
Combined clinical outcome (death + cardiac arrest + unplanned cardiac hospitalization), Quality of life measured by SF-36v.2; EQ-5D and KCCQ, Fabry disease progression assessed by dedicated Mainz Severity Score Index (MSSI), DS3 and FASTEX scores, Assessment of functional capacity using cardiopulmonary exercise testing (CPET), 6-minute walk test (6MWT) and NYHA classification, Electrocardiographic abnormalities (brady- and tachyarrhythmias, autonomic tension), Echocardiographic assessment (morphological and functional evaluation of the heart), Cardiac MRI abnormalities (left ventricular mass, inflammation and fibrosis), Biochemical biomarkers of the heart (CK-MB, NT-pro-BNP), Substrate levels of FD-specific biomarkers ( Gb3 in urine and Lyso-Gb3 in serum), Antidrug antibody (ADA) levels in patients on ERT, Levels of inflammatory cytokines (CRP, IL-1beta, IL-6, IL-8, IL-10, IL-12, TNF alpha, SAA), Assessment of renal function with glomerular filtration rate (eGFR), albumin/creatinine ra	—

Measure

Time frame

Combined clinical outcome (death + cardiac arrest + unplanned cardiac hospitalization), Quality of life measured by SF-36v.2; EQ-5D and KCCQ, Fabry disease progression assessed by dedicated Mainz Severity Score Index (MSSI), DS3 and FASTEX scores, Assessment of functional capacity using cardiopulmonary exercise testing (CPET), 6-minute walk test (6MWT) and NYHA classification, Electrocardiographic abnormalities (brady- and tachyarrhythmias, autonomic tension), Echocardiographic assessment (morphological and functional evaluation of the heart), Cardiac MRI abnormalities (left ventricular mass, inflammation and fibrosis), Biochemical biomarkers of the heart (CK-MB, NT-pro-BNP), Substrate levels of FD-specific biomarkers ( Gb3 in urine and Lyso-Gb3 in serum), Antidrug antibody (ADA) levels in patients on ERT, Levels of inflammatory cytokines (CRP, IL-1beta, IL-6, IL-8, IL-10, IL-12, TNF alpha, SAA), Assessment of renal function with glomerular filtration rate (eGFR), albumin/creatinine ra

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Countries

Poland

Outcome results

None listed