EUCTR2011-004800-40-AT进行中（未招募）1 期
An Open-Label Extension Study to Evaluate the Long Term Safety and Efficacy of Migalastat Hydrochloride Monotherapy in Subjects with Fabry Disease.
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 100
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •1. Subject with Fabry disease who completed treatment in a previous study of migalastat HCl given as monotherapy.
- •2. Male or female subjects 16 years of age or older. Note: Subjects under the age of 18 will be enrolled only at sites with all required regulatory and ethics approvals to do so.
- •3. A female subject is eligible to participate if she is:
- •a. Of non-childbearing potential, or
- •b. Of childbearing potential and NOT pregnant or nursing, has a negative urine pregnancy test at the Baseline Visit (Visit 1), and agrees to one of the methods of avoiding pregnancy listed in Appendix 1 from the time of first dose of study medication until 30 days after study completion.
- •A female is considered Non-childbearing potential” if she is status-post hysterectomy, status-post surgical removal of both ovaries, has current, documented tubal ligation, or is postmenopausal and >2 years without menses. Female subjects who are post-menopausal <2 years must be confirmed menopausal by Follicle Stimulating Hormone (FSH) and estradiol levels.
- •A female is considered childbearing potential” if she has functional ovaries, ducts, and uterus with no impairment that would cause sterility. This includes women with oligomenorrhea (even severe), and women who are perimenopausal or who have just begun to menstruate.
- •4. Male subjects must agree to use one of the contraception methods listed in Appendix 1. This criterion must be followed from the time of the first dose of study medication until 30 days after study completion.
- •5. Subject is willing and able to provide written informed consent and authorization for use and disclosure of Personal Health Information (PHI) or has a legally authorized representative who has given written informed consent.
- •Are the trial subjects under 18? no
- •Number of subjects for this age range:
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 100
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •1. The last available estimated glomerular filtration rate (eGFR) in the previous study
- •was <30 mL/min/1.73m2; unless there is measured GFR available within 3 months of Baseline Visit (Visit 1), which is >30 mL/min/1.73m2.
- •2. The subject has undergone, or is scheduled to undergo kidney transplantation or is currently on dialysis.
- •3. The subject is treated or has been treated with another investigational drug (except migalastat HCl) within 30 days of study start.
- •4. Subject is unable to comply with study requirements, or deemed otherwise
- •unsuitable for study entry, in the opinion of the investigator.
- •5. Had a documented transient ischemic attack, stroke, unstable angina, or myocardial infarction within the 12 months before Visit 1.
- •6. Has clinically significant unstable cardiac disease in the opinion of the investigator (e.g., cardiac disease requiring active management, such as symptomatic arrhythmia, unstable angina, or NYHA class III or IV congestive heart failure).
- •7. Has a history of allergy or sensitivity to AT1001 (including excipients) or other iminosugars (e.g., miglustat, miglitol).
- •8. Requires treatment with Glyset® (miglitol) or Zavesca® (miglustat).
- •9. Has any intercurrent illness or condition that may preclude the subject from fulfilling the protocol requirements or suggests to the investigator that the potential subject may have an unacceptable risk by participating in this study.
- •10. Patients with severe or unsuitable concomitant medical condition (cardiovascular, neurological, hepatic, renal, metabolic, hematological, immunological, pulmonary, or gastrointestinal disorder). The medical monitor or designee must be contacted to discuss the stability of a subject’s medical condition(s) and the potential impact of the condition(s) on trial participation.
- •11. Patients with clinically significant abnormal laboratory value(s) and clinically significant electrocardiogram (ECG) findings at baseline. The medical monitor or designee must be contacted to discuss the stability of a subject’s medical condition(s) and the potential impact of the condition(s) on trial participation.
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