Phase 2 Open-label study of BMN 110 in patients < 5 years old. v1

• Research type

  Research Study

• Full title

  A Phase 2, Open-label, Multinational Clinical Study to Evaluate the Safety and Efficacy of BMN 110 in Pediatric Patients Less Than 5 Years of Age with Mucopolysaccharidosis IVA (Morquio A Syndrome)

• IRAS ID

  88506

• Contact name

  Simon Jones

• Sponsor organisation

  Specialist I, Global Sites Services

• Eudract number

  2011-003197-84

• ISRCTN Number

  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. This study will evaluate the safety and efficacy of intravenous infusions of 2.0 mg/kg BMN 110 once a week for 52 consecutive weeks in patients less than 5 years of age with MPS IVA. As patients may experience hypersensitivity reactions associated with the administration of BMN 110, all patients may be pretreated with an age-appropriate dose of pre medication approximately 30 minutes to 1 hour prior to infusion. Vital signs will be measured just before, during, and following the infusion, and patients will be monitored by continuous pulse oximetry during and following the infusion. Adverse events and changes in concomitant medications will be recorded throughout the study. Clinical laboratory tests and physical examination, including assessment of skeletal deformities and corneal clouding will be performed at Screening and approximately every 13 weeks. Growth assessments, clinical laboratory assessments for blood and urine are conducted every 13 weeks. Study assessments will be completed by caregivers of the patients provided they are appropriate based on the health status and developmental maturity of the individual patient. Clinical laboratory tests and physical examination, including assessment of skeletal deformities and corneal clouding will be performed at Screening and approximately every 13 weeks. Growth measurements will be taken at Baseline and every 13 weeks. Urine samples will be collected at Baseline; Weeks 2, 4, 8 and 13, and every 13 weeks thereafter to measure urinary KS and urinary creatinine. Blood samples will be collected at Baseline; Weeks 4, 8, 12, and every 13 weeks thereafter for immunogenicity testing. The Denver II developmental test and the Clinical Global Impression instruments will be performed at Baseline and every 26 weeks thereafter. A Pediatric Quality of Life assessment will be performed at Baseline and every 26 weeks. X-rays of the spine, legs and hands/wrists will be performed at Baseline and Week 52. Dual-emission x-ray absorptiometry (DXA) scans and magnetic resonance imaging (MRI) scans of the cervical spine will be taken at Screening and Week 52. In addition, echocardiograms and electrocardiograms will be performed at Screening and every 26 weeks and an age appropriate hearing test will be performed at the beginning and the end of the study.

• REC name

  Yorkshire & The Humber - Leeds East Research Ethics Committee

• REC reference

  11/YH/0375

• Date of REC Opinion

  22 Nov 2011

• REC opinion

  Further Information Favourable Opinion