EUCTR2011-004800-40-IT进行中（未招募）不适用
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
- 不适用
- 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 as per the study protocol 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 ''childbearingpotential'' 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. 6. French subjects: In France, a subject will be eligible for inclusion in this study only if either affiliated to or a beneficiary of a social security category.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 4
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 96
- •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.
研究者
相似试验
进行中（未招募）
1 期
A Study to Evaluate the Long-Term Safety of Inclacumab Administered to Participants with Sickle Cell DiseaseMedDRA version: 21.0Level: PTClassification code 10040644Term: Sickle cell diseaseSystem Organ Class: 10010331 - Congenital, familial and genetic disordersSickle Cell DiseaseEUCTR2020-005289-32-DEGlobal Blood Therapeutics, Inc.520
进行中（未招募）
1 期
A Study using Migalastat to see the safety and usefulness of the drug in patients with Fabry Disease.Fabry disease is a rare X-linked lysosomal storage disorder caused by mutations in the gene (GLA) that encodes the lysosomal enzyme alfa-galactosidase A.MedDRA version: 14.1Level: SOCClassification code 10010331Term: Congenital, familial and genetic disordersSystem Organ Class: 10010331 - Congenital, familial and genetic disordersEUCTR2011-004800-40-ESGlaxoSmithKline Research & Development Limited100
进行中（未招募）
1 期
An extension to the phase 2 study A536-03 for the treatment of anemia in patients with myelodysplastic syndrome of low and intermediate risk groupsMyelodysplastic Syndromes (MDS)MedDRA version: 20.0Level: SOCClassification code 10029104Term: Neoplasms benign, malignant and unspecified (incl cysts and polyps)System Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)MedDRA version: 20.0Level: HLTClassification code 10028536Term: Myelodysplastic syndromesSystem Organ Class: 100000004851EUCTR2014-001280-13-DEAcceleron Pharma Inc.153
已完成
不适用
An Open-Label Extension Study to Evaluate the Long-Term Safety, Tolerability, Biological Activity, and Systemic Exposure of ATYR1940 in Adult Patients with Facioscapulohumeral Muscular Dystrophy (FSHD)Facioscapulohumeral muscular dystrophygenetic myopathy10028396NL-OMON44009aTyr Pharma, Inc.8
尚未招募
3 期
A study to examine the long-term safety, tolerability, and effectiveness of Pozelimab and Cemdisiran combination therapy in adult patients with Paroxysmal Nocturnal HemoglobinuriaParoxysmal Nocturnal HemoglobinuriaJPRN-jRCT2031230013Aurand Lisa11