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 48 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 48 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, Week 12, Week 24, Week 36,and Week 48/ ETV
- 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 48 weeks ] [ Designated as safety issue: No ]Timepoints: Baseline, Week 24, and Week 48
- Change in plasma and urinary KS over time as determined by descriptive statistics. [ Time Frame: Up to 48 weeks ] [ Designated as safety issue: No ]Timepoints: Baseline, Week 2, Week 4, Week 6, Week 12, Week 24, Week 36, Week 48/ ETV
- Assessment of effect on sleep apnea results as calculated using the Apnea-hypopnea index(AHI). [ Time Frame: Up to 48 weeks ] [ Designated as safety issue: No ]A subset of approximately 5 patients at selected sites who have abnormal overnight pulse oximetry readings and who provide additional informed consent will be assessed for sleep apnea.
- Descriptive summary of clinical safety assessments [ Time Frame: Continuously for up to 48 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:||May 2014|
|Estimated Primary Completion Date:||May 2014 (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 48 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 48 weeks of treatment.
Effect is defined by the following domains:
- Upper extremity function and dexterity
- Self care and functional abilities
|Contact: Celeste Decker, M.D.||866-961-8212|
|Contact: Adam Shaywitz, M.D., Ph.D.|
|United States, California|
|Children's Hospital & Research Center Oakland||Recruiting|
|Oakland, California, United States|
|Contact: Jo Ann Johnson 510-428-3885 ext 5421 firstname.lastname@example.org|
|United States, Illinois|
|Ann & Robert H. Lurie Children's Hospital of Chicago||Recruiting|
|Chicago, Illinois, United States|
|Contact: Paul Kogut 312-227-6766 email@example.com|
|United States, Texas|
|Not yet recruiting|
|Houston, Texas, United States|
|Contact: Celeste Decker, M.D. 866-961-8212|
|Not yet recruiting|
|Contact: Celeste Decker 866-961-8212|
|University Medical Center Mainz, Center of Pediatric and Adolescent Medicine Villa Metabolica||Recruiting|
|Contact: Dr. Eugen Mengel, MD 0049 6131 17 5754|
|NIHR/Wellcome Trust Birmingham CRF, Queen Elizabeth Hospital||Recruiting|
|Birmingham, United Kingdom|
|Contact: Mrs. Farfia Capper 01213713170 Farfia.Capper@uhb.nhs.uk|
|Salford Royal NHS Foundation Trust||Recruiting|
|Salford, United Kingdom|
|Contact: Dominic Sexton 0161 206 7879 Dominic.Sexton@srft.nhs.uk|
|Study Director:||Celeste Decker, M.D.||BioMarin Pharmaceutical|