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    Clinical Trials

    BioMarin is committed to the development of new therapies for serious diseases and medical conditions.  Successful clinical trials are required to gain Regulatory Agency (eg FDA in the US) approval for new medications to advance patient care, as well as to support existing products.

    A clinical trial is a research study done to evaluate new potential treatments in people.  During a clinical trial, information is collected to determine if a study drug is safe and effective, as well as to evaluate the risks and benefits of the study drug.  For more information about clinical trials in general, please go to http://www.clinicaltrials.gov/ct2/info/understand

    For the clinical trials that BioMarin is currently conducting, please see:

    PKU

    A Phase 3b Open-Label Study to Evaluate the Effect of Kuvan® on Neurocognitive Function, Maintenance of Blood Phenylalanine Concentrations, Safety, and Population Pharmacokinetics in Young Children With Phenylketonuria More >

    A Double-blind, Placebo-controlled, Randomized Study to Evaluate the Safety and Therapeutic Effects of Sapropterin Dihydrochloride on Neuropsychiatric Symptoms in Subjects With Phenylketonuria More >

    Phase 2, Open-Label Dose-Finding Study to Evaluate the Safety, Efficacy, and Tolerability of Multiple Subcutaneous (SC) Doses of rAvPAL-PEG in Subjects With PKU More >

    A Phase 2, Open-Label Study to Evaluate the Safety, Tolerability, and Efficacy of Subcutaneous Dose Levels of rAvPAL-PEG Administered Daily in Subjects With Phenylketonuria More >

    PKUDOS – Phenylketonuria (PKU) Demographic, Outcomes and Safety Registry
    This study is looking at safety and disease population over time when exposed to Kuvan
    More >

    MPS IVA (Morquio Syndrome)

    A Multicenter, Multinational, Longitudinal Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome) More >

    A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multinational Clinical Study to Evaluate the Efficacy and Safety of 2.0 mg/kg/Week and 2.0 mg/kg/Every Other Week BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome) More >

    Pompe

    A Phase 1/2 Open-label Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamic and Preliminary Efficacy of BMN 701 (GILT-tagged Recombinant Human GAA) in Patients With Late-onset Pompe Disease More >

    Genetically defined cancer

    A Phase 1/2, First in Human, Single-arm, Open-label Study of Once a Day, Orally Administered BMN 673 in Patients With Advanced or Recurrent Solid Tumors More >

    Phase 1, Two-arm, Open-label Study of Once Daily, Oral BMN 673 in Patients With Advanced Hematological Malignancies More >

    MPS VI (mucopolysaccharidosis VI), also known as Maroteaux-Lamy Syndrome

    Mucopolysaccharidosis (MPS) VI Clinical Surveillance Program (CSP)
    This study is looking at the disease population over time. More >

    LEMS

    A Phase 3, Multicenter, Double-blind, Placebo-controlled Randomized Discontinuation Study Followed by an Open-label Extension Period to Evaluate the Efficacy and Safety of Amifampridine Phosphate (3,4-Diaminopyridine Phosphate) in Patients With Lambert-Eaton Myasthenic Syndrome (LEMS). More >