Efficacy and Safety Study of BMN 110 for Morquio A Syndrome Patients Who Have Limited Ambulation
The primary objective of this study is to 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.
Morquio A Syndrome
Drug: BMN 110
|Study Design:||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official 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|
- Efficacy will be determined/summarized by percent changes of the domain scores for upper extremity function, dexterity, mobility, pain, and self-care and functional ability testing from baseline. [ Time Frame: Up to 144 weeks ] [ Designated as safety issue: No ]
- Upper extremity function will be tested using the Grip-Pinch Test.
- Dexterity will be tested using the Functional Dexterity Test.
- Mobility will be tested using the 25 Foot Walk test
- Pain will be determined by the Brief Pain Inventory-Short Form questionnaire or the Adolescent Pediatric Pain Tool
- Functional/self care abilities will be determined using Pediatric Outcomes Data Collection Instrument (PODCI)or the SF-36 questionnaire.
Efficacy will be measured at the following timepoints: Baseline, Weeks: 12, 24, 36, 48, 72, 96, 120, 144 / ETV. Weeks: 60, 84, 108, & 132 (Pain Inventory & PODCI only.)
- Change in respiratory function using a summarized analysis of the percentage change in FET, FIVC, FVC, FEV1, MVV and optional TLC values from the Baseline visit. [ Time Frame: Up to 144 weeks ] [ Designated as safety issue: No ]Timepoints: Baseline, Weeks: 24, 48, 96, 144 / ETV
- Change in urinary KS over time as determined by descriptive statistics. [ Time Frame: Up to 144 weeks ] [ Designated as safety issue: No ]Timepoints: Baseline, Weeks: 2, 4, 6, 12, 24, 36, 48, 72, 96, 120, 144 / ETV
- Assessment of effect on sleep apnea results as calculated using the Apnea-hypopnea index(AHI). [ Time Frame: Up to 144 weeks ] [ Designated as safety issue: No ]A subset consisting of any subjects who have abnormal overnight pulse oximetry readings will be assessed for sleep apnea. Only selected sites are participating in the sleep apnea study.
- Descriptive summary of clinical safety assessments [ Time Frame: Continuously for up to 144 weeks or more ] [ Designated as safety issue: Yes ]Incidence of AEs and changes in neurologic examinations, vital signs, ECHOs, ECGs, cervical spine radiographs, immunogenicity tests, clinical laboratory tests, and concomitant medications.
|Study Start Date:||August 2012|
|Estimated Study Completion Date:||July 2016|
|Estimated Primary Completion Date:||July 2016 (Final data collection date for primary outcome measure)|
Experimental: BMN 110 at 2.0 mg/kg/week
Weekly IV infusions of BMN 110 at 2.0 mg/kg/week over a period of approximately 4 hours per infusion for up to 144 weeks.
Drug: BMN 110
Drug will be delivered through a 4 hour (approximate) IV infusion at a dosage amount of 2.0 mg/kg/week for up to 144 weeks of treatment.
Effect is defined by the following domains:
- Upper extremity function and dexterity
- Self care and functional abilities
|United States, California|
|Children's Hospital & Research Center Oakland|
|Oakland, California, United States|
|United States, Illinois|
|Ann & Robert H. Lurie Children's Hospital of Chicago|
|Chicago, Illinois, United States|
|University Medical Center Mainz, Center of Pediatric and Adolescent Medicine Villa Metabolica|
|NIHR/Wellcome Trust Birmingham CRF, Queen Elizabeth Hospital|
|Birmingham, United Kingdom|
|Central Manchester University Hospitals NHS Foundation Trust|
|Manchester, United Kingdom|
|Salford Royal NHS Foundation Trust|
|Salford, United Kingdom|
|Study Director:||Celeste Decker, M.D.||BioMarin Pharmaceutical|