AN OPEN-LABEL EXTENSION OF STUDY TKT024 EVALUATING LONG-TERM SAFETY AND CLINI

本子项目是利用由NIH/NCRR资助的中心赠款提供的资源的众多研究子项目之一。子项目和研究者（PI）可能已经从另一个NIH来源获得了主要资金，因此可以在其他CRISP条目中表示。列出的机构是中心的，不一定是研究者的机构。该扩展研究旨在允许研究TKT024双盲期的依杜硫酶受体继续接受长期治疗。同样重要的是，它也将允许研究TKT024中的安慰剂接受者交叉并开始使用idursulase进行积极治疗。这项扩展研究TKT024EXT的主要目的是收集接受伊杜硫酶治疗的MPS II患者的长期安全性和临床结果数据。为了实现这一目标，在研究的第一年将每隔4个月进行一次安全性评估和临床结果评估，这与研究TKT024中的测试一致，然后在第二年每隔6个月进行一次评估。安全性和有效性终点将与TKT024研究中评估的终点相同。此外，将监测和记录反映患者长期MPS II疾病进展的临床事件。本研究的第二个目的是获得商业化生产的伊杜硫酶的安全性数据。该药物一旦可用于临床使用，将立即给试验中的患者使用。该材料的药代动力学数据也将从第一年进行的PK研究中产生。商业规模药品的PK和安全性数据将单独分析，也将作为完整数据集的一部分进行分析。

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. This extension study is designed to allow idursulfase-recipients in the double-blind phase of Study TKT024 to continue to receive therapy on a long-term basis. Equally important, it will also allow the placebo-recipients in Study TKT024 to cross-over and begin active treatment with idursulfase. The primary objective of this extension study TKT024EXT is to collect long-term safety and clinical outcome data in MPS II patients who are receiving idursulfase therapy. To achieve this objective, safety evaluations and clinical outcomes will be conducted at 4-month intervals during the first year of the study, which is consistent with the testing in Study TKT024, and then at 6-month intervals in the second year. Safety and efficacy endpoints will be identical to those evaluated in Study TKT024. In addition, clinical events reflecting long-term MPS II disease progression in patients will be monitored and documented. The secondary objective of this study is to obtain safety data on idursulfase manufactured at commercial-scale. This drug product will be administered to patients in the trial as soon as it is available for clinical use. Pharmacokinetic data on this material will also be generated from the PK studies to be conducted during the first year. The PK and safety data on the commercial-scale drug product will be analyzed separately as well as part of the complete data set.