An Open-Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Migalastat Hydrochloride Monotherapy in Subjects With Fabry Disease
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 84
- 试验地点
- 1
- 主要终点
- Number Of Participants Experiencing Adverse Events (AEs)
研究概览
简要总结
This is an open-label extension study intended to provide continued treatment with migalastat hydrochloride (HCl) for participants with Fabry disease who completed treatment of a previous migalastat HCl study. The study assessed the long-term safety and effectiveness of migalastat HCl.
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
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participant had completed treatment in a previous study of migalastat HCl given as a monotherapy
- •Male and female participant agreed to use protocol-identified acceptable contraception
- •Participant was willing to provide written informed consent and authorization for use and disclosure of Personal Health Information (PHI)
排除标准
- •Participant's last available estimated glomerular filtration rate (eGFR) in the previous study was <30 milliliter (mL)/minute (min)/1.73 meters squared (m^2); unless there was measured GFR available within 3 months of Baseline Visit, which was >30 mL/min/1.73 m^2
- •Participant had undergone, or was scheduled to undergo kidney transplantation or was currently on dialysis
- •Participant had a documented transient ischemic attack, stroke, unstable angina, or myocardial infarction within the 3 months before Baseline Visit
- •Participant had clinically significant unstable cardiac disease in the opinion of the investigator (for example, cardiac disease requiring active management, such as symptomatic arrhythmia, unstable angina, or New York Heart Association class III or IV congestive heart failure)
- •Participant had a history of allergy or sensitivity to AT1001 (including excipients) or other iminosugars (for example, miglustat, miglitol)
- •Participant required treatment with Glyset® (miglitol) or Zavesca® (miglustat)
- •Participants with severe or unsuitable concomitant medical condition
- •Participants with clinically significant abnormal laboratory value(s) and/or clinically significant electrocardiogram (ECG) findings
研究组 & 干预措施
Migalastat HCl 150 mg
Migalastat HCl 150 milligram (mg).
干预措施: migalastat HCl 150 mg (Drug)
结局指标
主要结局
Number Of Participants Experiencing Adverse Events (AEs)
时间窗: Day 1 after first dose to approximately 30 days after last treatment, median duration of 3.1 years
An AE was defined as any untoward medical occurrence in a participant administered migalastat that did not necessarily have a causal relationship with the treatment. Each AE was recorded at time of reporting; visits typically occurred every 6 months. Serious AEs were life threatening or resulted in death, resulted in disability/incapacity, hospitalization or prolonged hospitalization, or a congenital anomaly. The criteria for AE severity were: Mild: awareness of sign or symptom, does not interfere with normal everyday activities; Moderate: discomforting, interferes with normal everyday activities, but able to function; Severe: incapacitating, prevents normal everyday activities or significantly affects clinical status and requires medical intervention. A summary of serious and all other non-serious AEs regardless of causality is located in the Reported Adverse Events module.
次要结局
- Annualized Rate Of Change In The Estimated Glomerular Filtration Rate (eGFR)(Baseline to approximately 30 days after last treatment, median duration of 3.1 years)
- Change From Baseline In eGFR At End Of Study(Baseline to approximately 30 days after last treatment, median duration of 3.1 years)
- Change From Baseline In Plasma Globotriaosylsphingosine (Lyso-Gb3) To End Of Study(Baseline to approximately 30 days after last treatment, median duration of 3.1 years)
- Change From Baseline In White Blood Cell α-Gal A Activity To End Of Study(Baseline to approximately 30 days after last treatment, median duration of 3.1 years)
- Change From Baseline In 24-hour Urine Protein To End Of Study(Baseline to approximately 30 days after last treatment, median duration of 3.1 years)
- Change From Baseline In Left Ventricular Mass (LVM) To End Of Study(Baseline to approximately 30 days after last treatment, median duration of 3.1 years)
- Change From Baseline In Left Ventricular Mass Index (LVMi) To End Of Study(Baseline to approximately 30 days after last treatment, median duration of 3.1 years)
- Change From Baseline In Patient Reported Quality Of Life To End Of Study, As Assessed By The Short Form-36 (SF-36) Questionnaire(Baseline to approximately 30 days after last treatment, median duration of 3.1 years)