NCT01415427

Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)

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
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 173 (actual)
• Sponsor: BioMarin Pharmaceutical · Industry
• Sex: All
• Age: 5 Years
• Healthy volunteers: Not accepted

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 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 (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

Conditions

• Mucopolysaccharidosis IV A
• Morquio A Syndrome
• MPS IVA

Interventions

Type	Name	Description
DRUG	BMN 110 - Weekly	In Part 1, patients will receive intravenous (IV) infusions of study drug at a dose of 2.0 mg/kg/qw administered over a period of approximately 4 hours once a week. In Part 2, patients will continue to receive 2.0 mg/kg of BMN 110 every week, with no placebo.
DRUG	BMN 110 - Every Other Week	In Part 1, patients will receive intravenous (IV) infusions of study drug at a dose of 2.0 mg/kg administered over a period of approximately 4 hours every other week. Patients randomized to the 2.0 mg/kg/qow arm will receive infusions of placebo on alternating weeks, to mask active drug weeks. In Part 2, patients will receive 2.0 mg/kg of BMN 110 every week, with no placebo.

Timeline

• Start date: 2011-07-01
• Primary completion: 2016-06-16
• Completion: 2016-06-16
• First posted: 2011-08-12
• Last updated: 2021-07-21
• Results posted: 2017-05-19

Locations

43 sites across 20 countries: United States, Argentina, Brazil, Canada, Colombia, Denmark, France, Germany, Italy, Japan, Netherlands, Norway, Portugal, Qatar, Saudi Arabia, South Korea, Spain, Taiwan, Turkey (Türkiye), United Kingdom