Efficacy and Safety Study of BMN 110 for Morquio A Syndrome Patients Who Have Limited Ambulation
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|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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
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|