MOR006 Efficacy & Safety of BMN110 in Patients with Morquio A Syndrome

• Research type

  Research Study

• Full title

  A Phase 2, Open-label, Multinational Study to Evaluate the Efficacy and Safety of BMN 110 in Patients with Mucopolysaccharidosis IVA (Morquio A Syndrome) Who Have Limited Ambulation

• IRAS ID

  100109

• Contact name

  Christian Hendriksz

• Sponsor organisation

  BioMarin Pharmaceutical Inc.

• Eudract number

  2011-005703-33

• ISRCTN Number

  n.a

• Clinicaltrials.gov Identifier

  n.a

• Research summary

  Mucopolysaccharidosis IVA (Morquio A syndrome, MPS IVA) is a disease resulting in decreased physical endurance, growth, and independence. There is currently no standard accepted treatment for MPS IVA other than supportive care. Enzyme replacement therapy (ERT) with BMN 110 (an experimental drug) may be a potential new treatment option for MPS IVA patients. The purpose of this study is to evaluate the efficacy of weekly intravenous (IV) infusions of 2.0mg/kg BMN 110 for a total of 48 consecutive weeks in an MPS IVA patient population, 5 years of age or older, with limited ambulation. The symptoms of MPS IVA include painful skeletal dysplasia and cardiac abnormalities. The following tertiary objectives will be explored in this study: cardiac function, growth, bone density, spine morphology, biomarkers for bone and cartilage metabolism, analgesic medication use, overall exercise capacity, and endurance. The patient will undergo the following assessments and procedures: Primary efficacy assessments and procedures, patient questionnaires, respiratory function tests, sleep apnea tests (if applicable), urine samples for measurement of urinary KS and urinary creatinine, blood samples, plasma KS levels collected, radiographs, safety assessments, clinical laboratory tests, physical examinations, MRI, measurement of vital signs, adverse event assessment. All assessments and procedures for efficacy are outlined further in the Schedule of Events.

• REC name

  East of England - Cambridge South Research Ethics Committee

• REC reference

  12/EE/0132

• Date of REC Opinion

  29 May 2012

• REC opinion

  Further Information Favourable Opinion