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

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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.