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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

Recruiting

A Multicenter, Multinational, Observational Morquio A Registry Study in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)

  • Indication

    MPS IVA (Mucopolysaccharidosis IVA, also known as Morquio A syndrome)

  • Investigational Therapeutic

    None

  • Study Type

    Observational, Non-Interventional Registry

  • 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)

  • Status

    This program is recruiting participants

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Completed Studies


 

Study MOR-005

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

  • Indication

    MPS IVA (Mucopolysaccharidosis IVA, also known as Morquio A syndrome)

  • Investigational Therapeutic

    Vimizim® (elosulfase alfa or BMN 110)

  • Study Type

    Phase 3

  • 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)

  • Status

    This study has been completed

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Study BMN-110

Open-label Phase 2 study of BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome)

  • Indication

    MPS IVA (Mucopolysaccharidosis IVA, also known as Morquio A syndrome)

  • Investigational Therapeutic

    Vimizim® (elosulfase alfa or BMN 110)

  • Study Type

    Phase 2

  • 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

  • Status

    This study has been completed

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Study MOR-007

Efficacy and Safety Study of BMN 110 for Morquio A Syndrome Patients Who Have Limited Ambulation

  • Indication

    MPS IVA (Mucopolysaccharidosis IVA, also known as Morquio A syndrome)

  • Investigational Therapeutic

    Vimizim® (elosulfase alfa or BMN 110)

  • Study Type

    Phase 2

  • 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

  • Status

    This study has been terminated

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Study MOR-001

A Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)

  • Indication

    MPS IVA (Mucopolysaccharidosis IVA, also known as Morquio A syndrome)

  • Investigational Therapeutic

    Vimizim® (elosulfase alfa or BMN 110)

  • Study Type

    Observational

  • 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

  • Status

    This study has been terminated

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Study MOR-100

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

  • Indication

    MPS IVA (Mucopolysaccharidosis IVA, also known as Morquio A syndrome)

  • Investigational Therapeutic

    Vimizim® (elosulfase alfa or BMN 110)

  • Study Type

    Phase 2

  • 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)

  • Status

    This study has been terminated

Visit Study MOR-100 Now
 

Study MOR-008

Safety and Exercise Study of BMN 110 for Morquio A Syndrome

  • Indication

    MPS IVA (Mucopolysaccharidosis IVA, also known as Morquio A syndrome)

  • Investigational Therapeutic

    Vimizim® (elosulfase alfa or BMN 110)

  • Study Type

    Phase 2

  • 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.

  • Status

    This study has been terminated

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