Ignite Creation Date:
2024-05-05 @ 11:47 PM
Last Modification Date:
2024-10-26 @ 10:39 AM
Study NCT ID:
NCT01415427
Status:
COMPLETED
Last Update Posted:
2021-07-21
First Post:
2011-08-08
Brief Title:
Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA Morquio A Syndrome
Sponsor:
BioMarin Pharmaceutical
Organization:
BioMarin Pharmaceutical
Study Overview
Official Title:
A Multicenter Multinational Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA Morquio A Syndrome
Status:
COMPLETED
Status Verified Date:
2017-04
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 extension study will evaluate the long-term efficacy and safety of BMN 110 20 mgkgweek andor BMN 110 20 mgkgevery other week in patients with mucopolysaccharidosis IVA Morquio A Syndrome
Detailed Description:
This is a multi-center multinational extension study to evaluate 2 dose regimens of BMN 110 treatment in patients with MPS IVA who completed MOR-004
The last study visit assessments for MOR-004 will constitute Baseline for this study The first study drug dose of this protocol will occur on Week 0 of MOR-005 which is the same as the last visit Week 24 of MOR-004 Initially the study will be double-blind with patients previously randomized to BMN 110 in MOR-004 remaining on their assigned BMN 110 dose regimen qw or qow dosing The MOR-004 placebo patients will be re-randomized 11 ratio to one of the 2 BMN 110 dose regimen groups 20 mgkgqw or 20 mgkgqow
There will be two study parts
Part 1 - randomized double-blind until the optimal BMN 110 dose regimen has been determined based on the final primary efficacy analysis from MOR-004
Part 2 - open-label BMN 110 treatment with the single optimal dose regimen
The last study visit assessments for MOR-004 will constitute Baseline for this study The first study drug dose of this protocol will occur on Week 0 of MOR-005 which is the same as the last visit Week 24 of MOR-004 Initially the study will be double-blind with patients previously randomized to BMN 110 in MOR-004 remaining on their assigned BMN 110 dose regimen qw or qow dosing The MOR-004 placebo patients will be re-randomized 11 ratio to one of the 2 BMN 110 dose regimen groups 20 mgkgqw or 20 mgkgqow
There will be two study parts
Part 1 - randomized double-blind until the optimal BMN 110 dose regimen has been determined based on the final primary efficacy analysis from MOR-004
Part 2 - open-label BMN 110 treatment with the single optimal dose regimen
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