TKT024 OPEN-LABEL EXTENSION

这个子项目是众多研究子项目之一

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. Mucopolysaccharidosis Type II(MPS II) or Hunter syndrome is a X-linked recessive disorder caused by a deficiency of the lysosomal enzyme iduronate-2-sulfatase that results in the cellular storage of the mucopolysaccharide known as glycosaminoglycans (GAGs). In the most severe form of the disease death occurs before 20 years of age from accumulation of GAGs. Although allogenic bone marrow transplantation for Hunter syndrome has been tried, the results have been unsatisfactory. Therefore, no current treatment for this disease is available. Limited clinical trail data has demonstrated that replacement of the deficient iduronate-2-sulfatase enzyme in the body with an IV administered recombinant form of the enzyme is a safe and effective therapy to reduce the morbidity associated with the disease. A phase I/II trial of the drug sponsored by TKT Corporation has been completed. 12 patients were enrolled in this 6 month double blind placebo controlled study with no unanticipated or significant adverse events. TKT has moved forward to better characterize the safety and efficacy of this treatment by enrolling 90 patients in a randomized 12 month double blind placebo controlled phase II/III study. 10 of those patients are currently enrolled at Wash U and are managed in the Pediatric GCRC. After completing the current one year blinded trial, patients will be moved to this 2 year open label trial. However, only those patients who cannot be moved to a local site closer to home will remain in the trial at Washington University.