Safety and efficacy of BMN110 in patients with Morquio A syndrome

• Research type

  Research Study

• Full title

  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)

• IRAS ID

  61852

• Contact name

  Christian Hendriksz

• Sponsor organisation

  BioMarin Pharmaceutical Inc

• Eudract number

  2010-020198-18

• Clinicaltrials.gov Identifier

  N/A

• Research summary

  A Phase 3, Randomized, Double blind, Placebo-Controlled, Multinational Clinical study which will evaluate the efficacy and safety of BMN110 in patients with mucopolysaccharidosis IVA (Morquio A Syndrome).Eligible patients will be randomized 1:1:1 to one of three treatment arms and will receive either:1)2.0 mg/kg/week, 2)2.0 mg/kg/every other week, BMN 110 and infusion of placebo on alternating weeks3)PlaceboPatients will return to the study center each week to receive infusions of BMN 110 for 24 consecutive weeks. As patients may experience hypersensitivity reactions associated with administration of BMN 110, antihistamine will be administered prior to infusions at the investigators discretion. Vital signs will be measured just before, during and immediately following the infusion. Adverse Events and changes in concomitant medication will be recorded throughout the study.Laboratory tests will be performed at Screening, Baseline and every 6 weeks. At Screening and/or baseline and every 12 weeks duplicate endurance testing including 6-minute walk (6MW) test, 3-minute stair climb (3MSC) test and physical examinations will be performed, MPS Health assessment Questionnaire will be completed, and blood samples for evaluating exploratory biomarkers will be collected. Blood samples for immunogenicity testing and urine samples for urine KS and urine creatinine will be collected at Baseline, Week 2, Week 4 and every 4 weeks thereafter. Respiratory function testing and anthropometric measurements will be performed at Baseline and Week 24. Echocardiograms and Electrocardiograms (ECG) will be performed at Screening and Week 24. Cervical spine radiographs will be performed Screening. Radiographs of the lumbar spine will be performed at Baseline. Patients 20 years old and younger will undergo radiographs of the lower extremity at Baseline and Week 24.Patients may withdraw voluntarily from receiving study drug at any time yet remain in the study and continue to undergo study assessments. Patients may also withdraw entirely from participation in the study at any time.

• REC name

  Yorkshire & The Humber - Leeds East Research Ethics Committee

• REC reference

  10/H1306/87

• Date of REC Opinion

  30 Dec 2010

• REC opinion

  Further Information Favourable Opinion