CLINICAL TRIAL: AGLU02704: LATE-ONSET POMPE DISEASE TREATMENT STUDY

临床试验：AGLU02704：迟发性庞贝病治疗研究

• 批准号: 7731283
• 负责人: KATHERINE B SIMS
• 金额: $ 1.1万
• 依托单位: MASSACHUSETTS GENERAL HOSPITAL
• 依托单位国家: 美国
• 财政年份: 2008
• 资助国家: 美国
• 起止时间: 2008-04-01 至 2008-05-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7731283
• 关键词: Acids; Clinical Trials; Computer Retrieval of Information on Scientific Projects Database; Drug Kinetics; Enzymes; Funding; Glycogen; Glycogen storage disease type II; Grant; Infusion procedures; Institution; Patients; Pharmaceutical Preparations; Placebos; Rare Diseases; Research; Research Personnel; Resources; Safety; Site; Source; United States National Institutes of Health; glucosidase

This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. Pompe is a rare disease caused by the deficiency of acid a glucosidase, an enzyme that breaks down lysosomal glycogen. The overall aim is to evaluate the safety, efficacy, and pharmacokinetics of Myozyme in patients with late-onset Pompe as compared to placebo. Myozyme is an investigational enzyme made by Genzyme Corp. to replace the missing enzyme in Pompe patients. We are applying as a secondary site. All subjects will begin the study at a primary site & transfer after receiving study drug infusions for 6 mos.

该副本是利用众多研究子项目之一 由NIH/NCRR资助的中心赠款提供的资源。子弹和 调查员（PI）可能已经从其他NIH来源获得了主要资金， 因此可以在其他清晰的条目中代表。列出的机构是 对于中心，这不一定是调查员的机构。 庞贝是酸性葡萄糖酶缺乏引起的罕见疾病，糖苷酶是一种分解溶酶体糖原的酶。 总体目的是评估与安慰剂相比，肌酶疗法的安全性，有效性和药代动力学。 Myozyme是Genzyme Corp.生产的研究酶，以取代庞然患者中缺失的酶。我们正在作为次要站点申请。所有受试者将在接受研究药物输注6个MOS后在主要部位开始研究并转移。