NCT04049760已完成3 期
A Long-term, Open-label Study to Evaluate the Safety, Pharmacodynamics, and Efficacy of Migalastat in Subjects > 12 Years of Age With Fabry Disease and Amenable GLA Variants
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 16
- 试验地点
- 6
- 主要终点
- Incidence of Treatment-emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and AEs (Adverse Events) Leading to Discontinuation of Study Drug
研究概览
简要总结
This is a long-term, Open-label Study to Evaluate the Safety, Pharmacodynamics, and Efficacy of Migalastat in Subjects > 12 Years of Age With Fabry Disease and Amenable GLA Variants
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
- 12 Years 至 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female subjects diagnosed with Fabry disease > 12 years of age who completed Study AT1001-020
- •Subject's parent or legally-authorized representative is willing and able to provide written informed consent and authorization for use and disclosure of personal health information or research-related health information, and subject provides assent, if applicable
- •If of reproductive potential, both male and female subjects agreed to use a medically accepted method of contraception throughout the duration of the study and for up to 30 days after their last dose of migalastat
排除标准
- •Subject's last available estimated glomerular filtration rate (eGFR) in the previous study was < 60 mL/min/1.73 m2
- •Subject had advanced kidney disease requiring dialysis or kidney transplantation
- •Subject received any investigational/experimental drug, biologic, or device within 30 days before baseline, with the exception of migalastat
- •Subject anticipated starting gene therapy during the study period
- •Subject had any intercurrent illness or condition at Visit 1 that may have precluded the subject from fulfilling the protocol requirements or suggested to the investigator that the potential subject may have an unacceptable risk by participating in this study
- •Subject had a history of allergy or sensitivity to migalastat (including excipients) or other iminosugars (eg, miglustat, miglitol)
- •Subject required treatment with Replagal® (agalsidase alfa) or Fabrazyme® (agalsidase beta)
- •Subject required treatment with Glyset® (miglitol) or Zavesca® (miglustat)
- •Female subject was pregnant or breast-feeding, or was planning to become pregnant during the study period
- •In the opinion of the investigator, the subject and/or parent or legally-authorized representative was unlikely or unable to comply with the study requirements
研究组 & 干预措施
migalastat HCl 150 mg
Experimental
One migalastat 123 mg capsule equivalent to 150 mg migalastat HCl will be administered every other day (QOD) during the treatment period.
干预措施: migalastat HCl 150 mg (Drug)
结局指标
主要结局
Incidence of Treatment-emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and AEs (Adverse Events) Leading to Discontinuation of Study Drug
时间窗: Entire study
Number of subjects with TEAE, SAE, and AE leading to discontinuation during the study period
次要结局
- Change From Baseline to Month 24 in Estimated Glomerular Filtration Rate (eGFR)(Baseline, Month 24)
- Change From Baseline to Month 24 in Urine Protein Levels(Baseline, Month 24)
- Change From Baseline to Month 24 in Urine Albumin(Baseline, Month 24)
- Change From Baseline to Month 24 in Left Ventricular Mass Index (LVMi)(Baseline, Month 24)
- Change From Baseline to Month 24 in Pediatric and Quality of Life Inventory™ (PedsQL™) Scores(Baseline, Month 24)
- Change From Baseline to Month 24 in Fabry-Specific Pediatric Health and Pain Questionnaire (FPHPQ) Score for Pain Intensity(Baseline, Month 24)
- Number of Subjects Who Experienced Sudden Onset of Pain As Assessed Using the Fabry-Specific Health and Pain Questionnaire (FPHPQ)(Month 24)
- Change From Baseline to Month 24 in Plasma Levels of Lyso-Gb3(Baseline, Month 24)
研究者
研究点 (6)
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