Observational Prospective Cohort Study on Long-term Effective and Safe Use of Pegunigalsidase Alfa in Adult Fabry Patients Under "Real-world" Setting
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- 入组人数
- 75
- 试验地点
- 8
- 主要终点
- Assessment of structural abnormalities of the left ventricle
研究概览
简要总结
PEGASO is an observational study designed to collect prospective data on the effectiveness and safety of pegunigalsidase alfa in adult patients with Fabry disease, being treated or planning to start treatment, under real-world setting.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male and female adults (≥ 18 years).
- •Patients with a clinical diagnosis of Fabry disease confirmed by α-Gal A assay and detection of mutation in α-Gal A gene.
- •Patients either taking or planning to take pegunigalsidase alfa as treatment for Fabry disease. The treatment decision must be made independently from participation in this study.
- •Written informed consent to participate in the study and for the processing of personal data.
排除标准
- •History of hypersensitivity reaction to pegunigalsidase alfa.
- •Presence of any medical, emotional, behavioural, or psychological condition that, in the judgment of the physician, could interfere with the ability to participate in the study.
- •Female subjects who are pregnant or planning to become pregnant during the study.
研究组 & 干预措施
Fabry patients
Adult patients whit genetically confirmed diagnosis of Fabry disease being treated or planning to start treatment with Pegunigalsidase alfa according to clinical practice. Participants will be required to meet the inclusion and exclusion criteria and sign informed consent to be enrolled in the study.
干预措施: pegunigalsidase alfa (Drug)
结局指标
主要结局
Assessment of structural abnormalities of the left ventricle
时间窗: 24 months
Changes from baseline in structural abnormalities of the left ventricle assessed by echocardiography and defined by the presence of Left Ventricular Hypertrophy.
Assessment of left ventricular diastolic function
时间窗: 12 and 24 months
Changes from baseline in left ventricular diastolic function by echocardiography. Diastolic dysfunction will be assessed using early to late diastolic trans-mitral flow velocity.
Assessment of renal function
时间窗: 6, 12, 18 and 24 months
Changes from baseline in renal function by estimated Glomerular Filtration Rate.
次要结局
- Assessment of peak oxygen uptake (pVO2)(24 months)
- Assessment of carbon dioxide production (VCO2)(24 months)
- Assessment of NT-pro-BNP and high sensitivity cardiac troponin levels(6, 12,18 and 24 months)
- Assessment of Cardiovascular Magnetic Resonance (CMR)(12 and 24 months)
- Assessment of proteinuria and microalbuminuria(6, 12, 18 and 24 months)
- Assessment of Globotriaosylsphingosine (lyso-Gb3)(3, 6, 12 and 24 months)
- Assessment of QoL outcomes using the Short Form Health Survey 36 (SF-36)(12 and 24 months)
- Assessment of QoL outcomes using the Kansas City Cardiomyopathy Questionnaire (KCCQ)(12 and 24 months)
- Adverse events(24 months)
- Adverse drug reactions to pegunigalsidase alfa(24 months)
- Infusion-Related Reactions (IRRs)(24 months)
- Assessment of severity of neuropathic pain using Chronic Pain Grade (CPG)(12 and 24 months)
- Assessment of severity of neuropathic pain using Short Form of the Brief Pain Inventory (BPI-SF)(12 and 24 months)
- Assessment of specific Fabry disease ocular findings(Baseline, 12 and 24 months)
- Assessment of gastrointestinal symptoms using the Gastrointestinal Symptoms Rating Scale (GSRS)(12 and 24 months)
- Anti-drug antibodies (ADAs) testing(24 months)