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 ]

To evaluate the long-term safety and efficacy of BMN 110 administration at 2.0 mg/kg/qw and 2.0 mg/kg/qow in patients with MPS IVA. Safety results will be determined by numbers and severity of adverse events as well as descriptive statistic analysis of other safety related assessments. Efficacy will be assessed by changes from baseline in 6-minute walk test and 3-minute stair climb test as well as urine KS concentrations.


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 ]

To evaluate the long-term effect of BMN 110 administration at 2.0 mg/kg/qw and 2.0 mg/kg/qow on changes in biochemical markers of inflammation and bone and cartilage metabolism, in patients with MPS IVA.


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