Life-threatening allergic reactions, known as anaphylaxis, can occur during VIMIZIM® (elosulfase alfa) infusions. Typical signs of anaphylaxis include cough, rash, throat tightness, hives, flushing, changes in skin color, low blood pressure, shortness of breath, chest pain, and gastrointestinal symptoms such as nausea, abdominal pain, retching, and vomiting. Contact your doctor or get medical help right away if these symptoms occur during or after VIMIZIM infusions. If you have a respiratory illness, you may be at risk for a sudden worsening of your condition, and you may require additional monitoring.
Current Studies
Study 110-504
A Multicenter, Multinational, Observational Morquio A Registry Study in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
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Indication
MPS IVA (Mucopolysaccharidosis IVA, also known as Morquio A syndrome)
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Investigational Therapeutic
None
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Study Type
Observational, Non-Interventional Registry
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Goal
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)
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Status
Active, Not Recruiting
Completed Studies
Study MOR-005
Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
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Indication
MPS IVA (Mucopolysaccharidosis IVA, also known as Morquio A syndrome)
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Investigational Therapeutic
Vimizim® (elosulfase alfa or BMN 110)
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Study Type
Phase 3
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Goal
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)
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Status
This study has been completed
Study BMN-110
Open-label Phase 2 study of BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome)
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Indication
MPS IVA (Mucopolysaccharidosis IVA, also known as Morquio A syndrome)
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Investigational Therapeutic
Vimizim® (elosulfase alfa or BMN 110)
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Study Type
Phase 2
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Goal
Evaluate the safety and efficacy of weekly 2.0 mg/kg/wk infusions of BMN 110 in pediatric patients, less than 5 years of age at the time of administration of the first dose of study drug, diagnosed with MPS IVA (Morquio A Syndrome) for up to 208 weeks
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Status
This study has been completed
Study MOR-007
Efficacy and Safety Study of BMN 110 for Morquio A Syndrome Patients Who Have Limited Ambulation
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Indication
MPS IVA (Mucopolysaccharidosis IVA, also known as Morquio A syndrome)
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Investigational Therapeutic
Vimizim® (elosulfase alfa or BMN 110)
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Study Type
Phase 2
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Goal
Evaluate the effect of 2.0 mg/kg/week BMN 110 in a patient population that has limited ambulation, in a period of up to 144 weeks
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Status
This study has been terminated
Study MOR-001
A Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)
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Indication
MPS IVA (Mucopolysaccharidosis IVA, also known as Morquio A syndrome)
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Investigational Therapeutic
Vimizim® (elosulfase alfa or BMN 110)
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Study Type
Observational
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Goal
This multicenter, multinational, longitudinal study will quantify endurance and respiratory function in subjects diagnosed with MPS IVA and will better characterize the spectrum of symptoms and biochemical abnormalities in MPS IVA disease over time
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Status
This study has been terminated
Study MOR-100
A Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
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Indication
MPS IVA (Mucopolysaccharidosis IVA, also known as Morquio A syndrome)
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Investigational Therapeutic
Vimizim® (elosulfase alfa or BMN 110)
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Study Type
Phase 2
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Goal
This multicenter, open-label extension study is designed to assess long-term efficacy and safety of 2.0 milligrams (mg)/kilogram(kg)/week of BMN 110 in patients diagnosed with Mucopolysaccharidosis IVA (MPS IVA)
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Status
This study has been terminated
Study MOR-008
Safety and Exercise Study of BMN 110 for Morquio A Syndrome
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Indication
MPS IVA (Mucopolysaccharidosis IVA, also known as Morquio A syndrome)
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Investigational Therapeutic
Vimizim® (elosulfase alfa or BMN 110)
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Study Type
Phase 2
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Goal
Evaluate the safety of a 2.0 mg/kg/week and a 4.0 mg/kg/week of BMN 110 in patients with Morquio A syndrome for up to 192 weeks. Secondary objectives will investigate the effect of the two doses on exercise capacity for up to 192 weeks. In addition, the pharmacokinetic (PK) parameters of both doses of BMN 110 will be assessed.
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Status
This study has been terminated