Efficacy and Safety Study of BMN 110 for Morquio A Syndrome Patients Who Have Limited Ambulation
|ClinicalTrials.gov Identifier: NCT01697319|
Recruitment Status : Terminated
First Posted : October 2, 2012
Results First Posted : January 12, 2016
Last Update Posted : January 12, 2016
|Condition or disease||Intervention/treatment||Phase|
|Mucopolysaccharidosis IVA Morquio A Syndrome MPS IVA||Drug: BMN 110||Phase 2|
Effect is defined by the following key domains:
- Upper extremity function and dexterity
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||16 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|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|
|Study Start Date :||August 2012|
|Primary Completion Date :||October 2014|
|Study Completion Date :||October 2014|
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.
- Percent Change From Baseline in Speed as Measured in Functional Dexterity Test (FDT) [ Time Frame: Up to 96 weeks ]FDT assesses the ability to use the hand in daily tasks. The test involves turning 16 wooden pegs over as quickly as possible on a hardwood pegboard with one hand requiring a three-jaw chuck prehension pattern between the fingers and thumb within a two-minute time limit. Hand function is evaluated by how fast a patient can turn over pegs in the given time limit, i.e. speed (number of pegs/minute).
- Change From Baseline in Strength as Assessed by Grip and Pinch Test (GPT) [ Time Frame: Up to 96 weeks ]A grip-strength dynamometer and a pinch meter were used to measure grip strength and pinch strength. The results report change from baseline in strength for dominant and non-dominant hand in a forearm and wrist supported position.
- Percent Change From Baseline in Speed as Measured in Timed 25-Foot Walk Test (25FWT) [ Time Frame: Up to 96 weeks ]The timed 25-Foot Walk Test (25FWT) is an assessment of mobility and performance of leg function. The patient was instructed to walk a marked 25-foot course as quickly as possible in a time limit of 3 minutes and immediately walk back the same distance when reaching one end.The patient is allowed to use any ambulation method to move. The outcome measures the speed (feet / min) of moving.
- Percent Change From Baseline in Normalized Urine Keratan Sulfate (uKS) [ Time Frame: Up to 96 weeks ]Urinary keratan sulfate and urinary creatinine were measured through quantitative analysis. uKS is normalized to creatinine.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01697319
|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|