EUCTR2013-003321-28-DE
进行中（未招募）
1 期
An open-label, multicenter, multinational extension study of the long-term safety and pharmacokinetics of repeated biweekly infusions of Avalglucosidase Alfa (NeoGAA, GZ402666) in patients with Pompe disease
适应症Pompe 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 disordersTherapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
概览
- 阶段
- 1 期
- 干预措施
- 未指定
- 疾病 / 适应症
- Pompe disease (acid alpha-glucosidase deficiency)
- 发起方
- Genzyme Corporation
- 入组人数
- 21
- 状态
- 进行中（未招募）
- 最后更新
- 2年前
概览
简要总结
暂无简介。
研究者
入排标准
入选标准
- •Patients with Pompe disease who previously completed an avalglucosidase alfa 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 17
- •F.1\.3 Elderly (\>\=65 years) yes
- •F.1\.3\.1 Number of subjects for this age range 4
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.
结局指标
主要结局
未指定
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