EUCTR2013-003321-28-FR进行中（未招募）不适用
An open-label, multicenter, multinational extension study of the long-term safety and pharmacokinetics of repeated biweekly infusions of neoGAA in patients with Pompe 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
- 18
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
- •Patients with Pompe disease who previously completed a neoGAA study.
- •The patient and/or their parent/legal guardian is willing and able to provide signed informed consent, and the patient, if <18 years of age, is willing to provide assent if deemed able to do so.
- •The patient (and patient’s legal guardian if patient is <18 years of age) must have the ability to comply with the clinical protocol.
- •The patient, if female and of childbearing potential, must have a negative pregnancy test [urine beta-human chorionic gonadotropin] at baseline
- •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 16
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 2
排除标准
- •The patient is concurrently participating in another clinical study using investigational treatment.
- •The patient, in the opinion of the Investigator, is unable to adhere to the requirements of the study.
- •The patient has clinically significant organic disease (with the exception of symptoms relating to Pompe disease), including clinically significant cardiovascular, hepatic, pulmonary, neurologic, or renal disease, or other medical condition, serious intercurrent illness, or extenuating circumstance that, in the opinion of the Investigator, precludes participation in the study or potentially decreases survival.
研究者
相似试验
进行中（未招募）
1 期
Avalglucosidase Alfa Extension StudyPompe disease (acid alpha-glucosidase deficiency)MedDRA version: 20.0Level: LLTClassification code 10036143Term: Pompe's diseaseSystem Organ Class: 10010331 - Congenital, familial and genetic disordersMedDRA version: 20.1Level: PTClassification code 10053185Term: Glycogen storage disease type IISystem Organ Class: 10010331 - Congenital, familial and genetic disordersEUCTR2013-003321-28-DKGenzyme Corporation21
进行中（未招募）
1 期
eoGAA Extension StudyPompe disease (acid alpha-glucosidase deficiency)MedDRA version: 20.0Level: LLTClassification code 10036143Term: Pompe's diseaseSystem Organ Class: 10010331 - Congenital, familial and genetic disordersMedDRA version: 20.1Level: PTClassification code 10053185Term: Glycogen storage disease type IISystem Organ Class: 10010331 - Congenital, familial and genetic disordersEUCTR2013-003321-28-NLGenzyme Corporation21
进行中（未招募）
1 期
Avalglucosidase Alfa Extension StudyPompe disease (acid alpha-glucosidase deficiency)MedDRA version: 24.0Level: LLTClassification code 10036143Term: Pompe's diseaseSystem Organ Class: 10010331 - Congenital, familial and genetic disordersMedDRA version: 20.1Level: PTClassification code 10053185Term: Glycogen storage disease type IISystem Organ Class: 10010331 - Congenital, familial and genetic disordersEUCTR2013-003321-28-DEGenzyme Corporation21
已完成
2 期
An open-label, multicenter, multinational extension study of the long-term safety and pharmacokinetics of repeated biweekly infusions of neoGAA in patients with Pompe diseasePompe disease10021605NL-OMON55395Genzyme Europe BV2
进行中（未招募）
1 期
Evaluation of the Long-term Safety, Pharmacodynamics, and Exploratory Efficacy of GZ/SAR402671 in Treatment-Naïve Adult Male Patients with Fabry DiseaseMedDRA version: 18.0Level: PTClassification code 10016016Term: Fabry's diseaseSystem Organ Class: 10010331 - Congenital, familial and genetic disordersFabry diseaseEUCTR2014-004995-49-GBGenzyme Corporation11