Study Status: Completed
Recruit Status: Completed
Study Type: Interventional
Official Title: An Open-Label Phase 1/2 Study to Assess the Safety, Efficacy and Dose of UX003 rhGUS Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7)
Brief Summary:
UX003-CL201 is an open-label Phase 1/2 study to assess the safety, efficacy, and dose of UX003 in MPS 7 subjects via IV administration every other week (QOW) for 36 weeks with up to an additional 36 weeks from the optional continuation period. Up to 5 subjects, who are between 5 and 30 years of age inclusive, will be enrolled and treated with UX003.
The initial 12-week treatment period will be followed by a 24-week forced dose titration period to assess the optimal dose. Subjects who complete both the initial treatment and forced dose titration periods will continue treatment in a 36 week continuation period