AN OPEN-LABEL MAINTENANCE STUDY OF THE ENZYME REPLACEMENT THERAPY (ERT)

酶替代疗法 (ERT) 的开放标签维持研究

• 批准号: 8167321
• 负责人: Kenneth Rosenbaum
• 金额: $ 2.5万
• 依托单位: CHILDREN'S RESEARCH INSTITUTE
• 依托单位国家: 美国
• 财政年份: 2010
• 资助国家: 美国
• 起止时间: 2010-01-20 至 2010-06-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8167321
• 关键词: Body Weight; Clinical Research Protocols; Computer Retrieval of Information on Scientific Projects Database; Data; Disease; Dose; Fabry Disease; Funding; Grant; Human Genetics; Institution; International; Maintenance; Monitor; National Institute of Neurological Disorders and Stroke; Outcome; Outcome Measure; Patients; Pharmaceutical Preparations; Protocols documentation; Registries; Research; Research Personnel; Resources; Safety; Source; Surveys; United States National Institutes of Health; enzyme replacement therapy; gene therapy; open label

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. The objectives of this study are to continue treatment of three patients with Fabry disease with enzyme replacement therapy (ERT) using Replagal (agalsidase alfa) at a dose of 0.2mg/kg of body weight administered every 2 weeks. These patients were previously on clinical research protocol 04-N-0027 at the National Institute of Neurological Disorders and Stroke (NINDS) under P.I. Dr. Raphael Schiffmann. With the closure of protocol 04-N-0027 the patients are being transitioned to this study. Dr. Tifft will assume Dr. Schiffmann's IND for Replagal. This open-label study will monitor safety and outcome measures of disease stability as part of the Fabry Outcome Survey (FOS), an international registry of outcome data on patients treated with Replagal. 04-N-0027 was an investigator-sponsored protocol funded by Transkaryotic Therapies (TKT), now Shire, Human Genetic Therapies (HGT), and all TKT protocols and obligations were assumed by Shire HGT. The hypothesis is that patients currently stable on over 5 years of Replagal infusiona at 0.2mg/kg body weight every other week will remain stable. Safety profile of the drug will continue to be favorable.

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。 这项研究的目的是继续治疗三名Fabry病患者 酶替代疗法（ERT），使用重质（agalsidase alfa）以0.2mg/kg的剂量为0.2mg/kg 体重每2周一次进行一次。 这些患者以前从事临床研究 国立神经疾病与中风研究所（NINDS）的协议04-N-0027 在P.I.拉斐尔·席夫曼（Raphael Schiffmann）博士。 随着协议的关闭04-N-0027，患者是 过渡到这项研究。 Tifft博士将假设Schiffmann博士的IND居住。 这 开放标签研究将监测疾病稳定性的安全性和结果指标 Fabry结果调查（FOS），《治疗患者的结果数据》的国际注册表 与乡土。 04-N-0027是由跨卡罗尼特资助的研究者赞助的协议 疗法（TKT），现在的郡，人基因疗法（HGT）以及所有TKT方案和 Shire HGT承担了义务。 假设是，目前在5年以上的Replagal Infusiona上稳定的患者 每隔一周的体重0.2mg/kg体重将保持稳定。 该药物的安全性将会 继续有利。