CTIS2022-500488-10-00招募中1 期
AN OPEN-LABEL STUDY TO EVALUATE THE SAFETY AND PHARMACOKINETICS OF MIGALASTAT HCL IN SUBJECTS WITH FABRY DISEASE AND AMENABLE GLA VARIANTS AND SEVERE RENAL IMPAIRMENT OR END-STAGE RENAL DISEASE TREATED WITH HEMODIALYSIS - AT1001-025
Amicus Therapeutics Inc.0 个研究点目标入组 15 人开始时间: 2022年7月15日最近更新:
适应症
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 发起方
- 入组人数
- 15
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 18 至 65+（—）
- 性别
- All
入选标准
- •Male or female subjects aged 18 years or older, diagnosed with Fabry disease., Subject is willing and able to provide written informed consent (or assent) and authorization for use and disclosure of Personal Health Information or research-related health information or subject has a legally-authorized representative who has provided written informed consent and authorization, and subject provides assent, if applicable., Subject has a GLA variant recorded in their medical records that is amenable to migalastat. • All subjects will have confirmatory GLA genotyping done at Visit 1. • For subjects without a known GLA variant, GLA genotyping results must be received prior to Visit 2. • For subjects with a GLA variant that has not yet been tested in the Migalastat Amenability Assay, amenability testing results must be received prior to Visit 2., Subject has at least 1 documented eGFR value of < 30 mL/min/1.73 m2 in his/her medical history within the last 3 months and has an eGFRMDRD value of < 30 mL/min/1.73 m2 at Visit 1 per the central laboratory or subject is on HD (standard or HDF)., Subjects with ESRD have been on a stable 2- or 3-times a week HD (standard or HDF) regimen for at least 2 months prior to the screening visit (Visit 1)., Subjects with ESRD must commit to completing at least 4 standard HD or HDF sessions during each 2-week dosing interval., Subjects with ESRD must commit to completing the entire prescribed duration for each dialysis session., If of reproductive potential, both male and female subjects agree to use a medically accepted method of contraception during the study and for up to 30 days after the last dose of migalastat.
排除标准
- •Subject has undergone or is scheduled to undergo kidney transplantation., Subject 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., Subject has a history of allergy or sensitivity to migalastat (including excipients) or other iminosugars (eg, miglustat, miglitol)., Subject requires concurrent treatment with Glyset® (miglitol), Replagal® (agalsidase alfa), or Fabrazyme® (agalsidase beta) or has been treated with these medications within 14 days before Visit 2., In France only, protected persons as defined by the Public Health Code., Subject requires concurrent treatment with Zavesca® (miglustat) or has been treated with Zavesca within the 30 days before Visit 1., Female subject is pregnant or breastfeeding., Subject is unable to comply with study requirements, or deemed otherwise unsuitable for study entry, in the opinion of the investigator., Subject is on peritoneal dialysis., Subject is treated or has been treated with another investigational drug (except migalastat) within the 30 days before Visit 1., Subject has undergone any gene therapy at any time prior to the study or anticipates undergoing gene therapy during the study., Subject has had a documented transient ischemic attack, stroke, unstable angina, or myocardial infarction within the 3 months before Visit 1., Subject has clinically significant unstable cardiac disease in the opinion of the investigator (eg, cardiac disease requiring active management, such as symptomatic arrhythmia, unstable angina, or New York Heart Association Class III or IV congestive heart failure).
研究者
相似试验
招募中
3 期
An Open-label Study to Evaluate the Safety and Pharmacokinetics of Migalastat HCl in Subjects with Fabry Disease and Renal dysfunctionsFabry DiseaseJPRN-jRCT2051220125Koh KyuBum14
进行中（未招募）
1 期
A Study to Evaluate the Safety and Pharmacokinetics of Ataluren in Subjects From =6 Months to <2 Years of Age with Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)onsense Mutation Duchenne Muscular Dystrophy (nmDMD)EUCTR2020-000980-21-Outside-EU/EEAPTC Therapeutics, Inc.
进行中（未招募）
1 期
An Open-Label Study to Evaluate the Safety and Efficacy of Ombitasvir/Paritaprevir/Ritonavir with or without Dasabuvir in Adults with Genotype 1a or Genotype 4 Chronic Hepatitis C Virus (HCV) Infection, with Severe Kidney Impairment or End-Stage Kidney Disease (RUBY-II)EUCTR2015-002012-33-ESAbbVie Deutschland GmbH & Co. KG18
进行中（未招募）
1 期
A Study to Evaluate the Safety and Efficacy of Ombitasvir/Paritaprevir/Ritonavir and Dasabuvir with Ribavirin in Adults with Genotype 1 and Ombitasvir/Paritabprevir/Ritonavir with Ribavirin in Adults with Genotype 4 Chronic Hepatitis C Virus Infection and Decompensated CirrhosisEUCTR2014-001477-13-DEAbbVie Deutschland GmbH & Co. KG60
已完成
1 期
Topical Hidracare Solution in Participants with Mild to Moderate Hidradenitis SuppurativaHidradenitis suppurativaACTRN12619001734156Aerotech Australia Pty Ltd19