Clinical Trial: Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
Study Status: Completed
Recruit Status: Completed
Study Type: Interventional
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)
Brief Summary: This Phase 3 extension study will evaluate the long-term efficacy and safety of BMN 110 2.0 mg/kg/week and/or BMN 110 2.0 mg/kg/every other week in patients with mucopolysaccharidosis IVA (Morquio A Syndrome).
Detailed Summary:
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 (1:1 ratio) to one of the 2 BMN 110 dose regimen groups: 2.0 mg/kg/qw or 2.0 mg/kg/qow.
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
Sponsor: BioMarin Pharmaceutical
Current Primary Outcome:
- Change From Baseline in 6-minute Walk (6MW) Test - ITT [ Time Frame: Baseline to week 168 ]Efficacy was assessed by changes from baseline in 6-minute walk test
- Change From Baseline in 6-minute Walk (6MW) Test - MPP [ Time Frame: Baseline to week 168 ]Efficacy was assessed by changes from baseline in 6-minute walk test
Original Primary Outcome: Primary Long-Term Safety/Efficacy Evaluation [ Time Frame: Approximately 240 weeks ]
Current Secondary Outcome:
- Change From Baseline in 3-minute Stair Climb Test - ITT [ Time Frame: Baseline to week 168 ]Efficacy was assessed by changes from baseline in 3-minute stair climb test.
- Change From Baseline in 3-minute Stair Climb Test - MPP [ Time Frame: Baseline to week 168 ]Efficacy was assessed by changes from baseline in 3-minute stair climb test.
- Change From Baseline in Urine Keratan Sulfate - ITT [ Time Frame: Baseline to week 168 ]Efficacy was assessed by changes from baseline in urine keratan sulfate (normalized to urine creatinine.)
- Change From Baseline in Urine Keratan Sulfate - MPP [ Time Frame: Baseline to week 168 ]Efficacy was assessed by changes from baseline in urine keratan sulfate (normalized to urine creatinine.)
Original Secondary Outcome: Long-Term evaluation of changes in biochemical markers of inflammation and bone and cartilage metabolism [ Time Frame: Approximately 240 weeks ]
Information By: BioMarin Pharmaceutical
Dates:
Date Received: August 8, 2011
Date Started: July 2011
Date Completion:
Last Updated: April 13, 2017
Last Verified: April 2017