German Observational Multicenter Study of Patients With Fabry Disease Under Enzyme Replacement Therapy With Pegunigalsidase-alfa
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 60
- 试验地点
- 8
- 主要终点
- eGFR
研究概览
简要总结
Pegunigalsidase-alfa may represent an advance in ERT for FD, based on its unique pharmacokinetics and apparent low immunogenicity. The objective of the study is to document long term data on treatment with pegunigalsidase-alfa under "real world" conditions. 60 patients with FD (therapy-naïve or pretreated with agalsidase-alfa or agalsidase-beta) will be recruited in 8 German Fabry centers. The treatment duration/patient will be 2 years. All patients will be followed-up by the above listed Fabry expert centers.
详细描述
Pegunigalsidase-alfa, a novel PEGylated, covalently crosslinked form of α-galactosidase A developed as enzyme replacement therapy (ERT) for Fabry disease (FD), was designed to increase plasma half-life and reduce immunogenicity, thereby enhancing efficacy compared with available products.
The rationale of the current project is that disease progression of patients with FD can be stabilized comparable to patients under current ERT, leading to a validation of the clinical phase 3-studies and a transfer of these previous outcomes to a nationwide "real world" designed study in Germany.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Other
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
- •Males and females, ≥18 years, diagnosed with Fabry disease.
- •ERT naïve (patients with signs of organ involvement (kidney, heart and/or CNS signs) to be considered for ERT following the European Consensus Guidelines on ERT (Biegstraaten et al. 2015) or patients with neuropathic pain not controlled with pain medication or patients with GI symptoms not relieved with standard medication or ERT switch patients (under ERT for ≥12 months).
- •Subjects taking ACE inhibitors, ARBs, or renin inhibitors on a stable dose for at least 4 weeks before screening.
- •Subjects taking analgesics/antidepressants on a stable dose for at least 4 weeks before screening.
- •Female patients must have a negative pregnancy test and use a medically accepted form of contraception throughout the study.
排除标准
- •Patient is unwilling to give informed consent.
- •Patient is unable to comply with the clinical protocol.
- •Patients on dialysis.
- •Patient has a clinically significant organ disease (e.g., cancer in the past 5 years) that in the opinion of the investigator would preclude participation in the trial.
- •Patients with a history of organ transplantation.
研究组 & 干预措施
Standard of Care
Patient treated with Pegunigalsidase-alfa according to standard of care
干预措施: Pegunigalsidase-alfa (Drug)
结局指标
主要结局
eGFR
时间窗: yearly
eGFR: Change in annualized eGFR slope compared with annualized eGFR slope before treatment start or switch.
次要结局
未报告次要终点