2025-521408-24-00招募中3 期
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
适应症
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 40
- 试验地点
- 2
- 主要终点
- 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.
研究概览
简要总结
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.
入排标准
- 年龄范围
- 18 years 至 65+ years（65+ Years, 18-64 Years）
- 接受健康志愿者
- 否
入选标准
- •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)
排除标准
- •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
- •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
结局指标
主要结局
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.
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.
次要结局
- 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
研究者
Krzysztof Kaczmarek
Scientific
Medical University Of Lodz
研究点 (2)
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