NCT05054387已完成4 期
A Phase 4, Open Label, Safety and Efficacy Study of Fabrazyme® (Agalsidase Beta) as Enzyme Replacement Therapy in Chinese Participants With Fabry Disease
Genzyme, a Sanofi Company6 个研究点 分布在 1 个国家目标入组 22 人开始时间: 2021年10月13日最近更新:
适应症
干预措施
相关药物
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 发起方
- 入组人数
- 22
- 试验地点
- 6
- 主要终点
- Incidence of treatment-emergent adverse events (AEs)
研究概览
简要总结
This is a 54-week Phase 4, open label, single arm study to evaluate the safety and the efficacy of Fabrazyme (agalsidase beta) as enzyme replacement therapy (ERT) in Chinese participants with Fabry Disease.
详细描述
Study participation for each patient will be total of 54 weeks which will include 4 weeks of screening, 48 weeks of treatment period and 2 weeks of post study treatment observation
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 8 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participant must be 8 years of age or older, at the time of signing the informed consent
- •Participants naive to agalsidase beta and agalsidase alpha
- •Chinese participants diagnosed with Fabry disease and with documented plasma or leukocyte αGAL activity deficient below laboratory's reference range, and/or documented diagnosis by genotyping
- •Participants must have one or more symptoms and signs consistent with manifestations of Fabry disease (not limited to neuropathic pain, chronic kidney disease, hypertrophic cardiomyopathy, cardiac rhythm disturbances, cerebrovascular involvement, cornea verticillata, angiokeratoma, gastrointestinal symptoms, hypo- or anhydrosis)
- •A female participant is eligible to participate if she is not pregnant or breastfeeding and use an acceptable contraceptive method
- •Participants and/or participant's legal representative capable of giving signed informed consent.
排除标准
- •The participant has undergone kidney transplantation.
- •The participant has a clinically significant organic disease (with the exception of symptoms relating to Fabry disease) in the opinion of the Investigator, would preclude participation in the trial.
- •Received an investigational drug, or device, other than Fabrazyme, within 30 days of anticipated IMPs administration or 5 half-lives of the previous investigational drug, whichever is longer.
- •The patient has current evidence of kidney failure or renal insufficiency, as defined by eGFR <30 mL/min/1.73 m
- •Individuals who have life threatening hypersensitivity (anaphylactic reaction) to the active substance or any of the excipients included.
- •The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.
研究组 & 干预措施
Agalsidase beta
Experimental
Agalsidase beta treatment at approved dose and regimen, administered once every 2 weeks as an IV infusion
干预措施: Agalsidase beta (Drug)
结局指标
主要结局
Incidence of treatment-emergent adverse events (AEs)
时间窗: Baseline to week 50
Including TEAE, SAEs, and adverse events of special interest (AESIs) including infusion associated reactions (IARs) and change of clinical laboratory, vital signs and ECG
次要结局
- The percentage of participants with abnormal plasma GL3 values per central lab reference range(at Week 6, Week 12, Week 24 and Week 48)
- The percent changes of plasma GL3(from baseline to Week 6, Week 12, Week 24 and Week 48)
- The absolute changes of plasma globotriaosylsphingosine (lyso-GL3)(from baseline to Week 6, Week 12, Week 24 and Week 48)
- The change of Fabry disease symptoms(from baseline to Week 24 and Week 48)
- The absolute change of estimated glomerular filtration rate (eGFR) by chronic kidney disease epidemiology collaboration (CKD-EPI) for adult (≥18 years)(from baseline to Week 12, Week 24, Week 36 and Week 48)
- The absolute change of estimated glomerular filtration rate (eGFR) by Schwartz for children (8 ≤age <18 years)(from baseline to Week 12, Week 24, Week 36 and Week 48)
- The percent changes of plasma lyso-GL3(from baseline to Week 6, Week 12, Week 24 and Week 48)
- The absolute changes of plasma globotriaosylceramide (GL3)(from baseline to Week 6, Week 12, Week 24 and Week 48)
- The number of participants with abnormal plasma GL3 values per central lab reference range(at Week 6, Week 12, Week 24 and Week 48)
研究者
研究点 (6)
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