Ignite Creation Date:
2024-05-05 @ 11:30 AM
Last Modification Date:
2024-10-26 @ 9:09 AM
Study NCT ID:
NCT00069641
Status:
COMPLETED
Last Update Posted:
2021-06-10
First Post:
2003-09-29
Brief Title:
Iduronate-2-sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II MPS II
Sponsor:
Shire
Organization:
Takeda
Study Overview
Official Title:
A Phase IIIII Randomized Double-Blind Placebo-Controlled Study Evaluating the Safety and Efficacy of Weekly and Every Other Week Dosing Regimens of Iduronate-2-Sulfatase Enzyme Replacement Therapy in Patients With MPS II
Status:
COMPLETED
Status Verified Date:
2021-05
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
The purpose of this study is to determine whether the administration of iduronate-2-sulfatase enzyme in a weekly or every other week therapy frequency is safe and efficacious in patients with MPS II
Detailed Description:
MPS II is a rare X-linked lysosomal storage disorder caused by a deficiency in the enzyme iduronate-2-sulfatase Because of this deficiency glycosaminoglycans GAG accumulate in multiple tissues and organs resulting in progressive cellular and organ system dysfunction The purpose of this study is to determine if one year of therapy with iduronate-2-sulfatase enzyme replacement therapy at a dose of 05mgkg weekly or every other week is safe and results in clinically meaningful improvement in multiple organ function compared with a placebo group Upon completion of the study patients will be eligible to enroll in an open-label maintenance study
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None