EUCTR2011-004800-40-ES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 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 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.
- •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 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 a-galactosidase A.MedDRA version: 17.0Level: SOCClassification code 10010331Term: Congenital, familial and genetic disordersSystem Organ Class: 10010331 - Congenital, familial and genetic disordersEUCTR2011-004800-40-ATAmicus Therapeutics, Inc.100
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
进行中（未招募）
1 期
An extension study to the A083-02 and A083-03 studies that evaluate the long-term effect of the ACE-083 investigational product in patients with Facioscapulohumeral Muscular Dystrophy (FSHD) previously participated in study A083-02 and also in patients with Charcot-Marie Tooth (CMT) disease type 1 and X who previously participated in study A083-03.Facioscapulohumeral Muscular DystrophyMedDRA version: 20.0 Level: PT Classification code 10064087 Term: Facioscapulohumeral muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disordersEUCTR2019-000305-79-ESAcceleron Pharma Inc.150
进行中（未招募）
1 期
An extension of the Phase 2 study with increasing doses of a new medication called ACE-536 in patients suffering from ß-Thalassemiaß-Thalassemia Intermedia and ß-Thalassemia majorMedDRA version: 17.0Level: LLTClassification code 10062923Term: Thalassemia intermediaSystem Organ Class: 100000004850MedDRA version: 17.0Level: LLTClassification code 10054661Term: Thalassemia majorSystem Organ Class: 100000004850MedDRA version: 17.0Level: LLTClassification code 10054660Term: Thalassemia betaSystem Organ Class: 100000004850EUCTR2014-001281-94-GRAcceleron Pharma, Inc.72
进行中（未招募）
1 期
An Study to Evaluate the Long-Term Safety, Tolerability, and Biological Activity of ATYR1940 in Patients with Limb Girdle and Fascioscapulohumeral Muscular Dystrophyimb Girdle Muscular Dystrophy and Facioscapulohumeral Muscular DystrophyMedDRA version: 20.0Level: PTClassification code 10064087Term: Facioscapulohumeral muscular dystrophySystem Organ Class: 10010331 - Congenital, familial and genetic disordersEUCTR2016-000624-25-ITATYR PHARMA, INC.8