Ignite Creation Date:
2024-05-05 @ 11:12 PM
Last Modification Date:
2024-10-26 @ 10:30 AM
Study NCT ID:
NCT01275066
Status:
COMPLETED
Last Update Posted:
2014-07-07
First Post:
2011-01-10
Brief Title:
A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA Morquio A Syndrome
Sponsor:
BioMarin Pharmaceutical
Organization:
BioMarin Pharmaceutical
Study Overview
Official Title:
A Phase 3 Randomized Double-Blind Placebo-Controlled Multinational Clinical Study to Evaluate the Efficacy and Safety of 20 mgkgWeek and 20 mgkgEvery Other Week BMN 110 in Patients With Mucopolysaccharidosis IVA Morquio A Syndrome
Status:
COMPLETED
Status Verified Date:
2014-06
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:
This Phase 3 study will evaluate the efficacy and safety of 20 mgkgweek BMN 110 and 20 mgkgevery other week BMN 110 in patients with mucopolysaccharidosis IVA Morquio A Syndrome
There is currently no standard accepted treatment for MPS IVA other than supportive care Enzyme replacement therapy ERT may be a potential new treatment option for MPS IVA patients BMN 110 is administered to MPS IVA patients by IV infusion allowing cellular uptake by the mannose-6-phosphate receptor and transportation to the lysosomes
This enzyme uptake into the lysosomes is hypothesized to promote increased catabolism of keratan sulfate KS in tissue macrophages hyaline cartilage other connective tissues and heart valve and reduce the progressive accumulation of KS which is responsible for the clinical manifestations of the disorders
There is currently no standard accepted treatment for MPS IVA other than supportive care Enzyme replacement therapy ERT may be a potential new treatment option for MPS IVA patients BMN 110 is administered to MPS IVA patients by IV infusion allowing cellular uptake by the mannose-6-phosphate receptor and transportation to the lysosomes
This enzyme uptake into the lysosomes is hypothesized to promote increased catabolism of keratan sulfate KS in tissue macrophages hyaline cartilage other connective tissues and heart valve and reduce the progressive accumulation of KS which is responsible for the clinical manifestations of the disorders
Detailed Description:
None
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
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| 0999935174 | OTHER | Taiwan FDA | None |
| 2010-020198-18 | EUDRACT_NUMBER | None | None |
| 10H130687 | OTHER | None | None |
| 189720213001-0001 | OTHER | None | None |
| 2011_038B201129 | OTHER | None | None |
| 145240 | OTHER | None | None |
| 2011-01-09 | OTHER | None | None |
| 20110012889 | OTHER | None | None |