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.
Effect is defined by the following key domains: * Upper extremity function and dexterity * Mobility
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
16
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.
Children's Hospital & Research Center Oakland
Oakland, California, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
Universitätsklinikum Hamburg
Hamburg, Germany
University Medical Center Mainz, Center of Pediatric and Adolescent Medicine Villa Metabolica
Percent Change From Baseline in Speed as Measured in Functional Dexterity Test (FDT)
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).
Time frame: Up to 96 weeks
Change From Baseline in Strength as Assessed by Grip and Pinch Test (GPT)
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.
Time frame: Up to 96 weeks
Percent Change From Baseline in Speed as Measured in Timed 25-Foot Walk Test (25FWT)
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.
Time frame: Up to 96 weeks
Percent Change From Baseline in Normalized Urine Keratan Sulfate (uKS)
Urinary keratan sulfate and urinary creatinine were measured through quantitative analysis. uKS is normalized to creatinine.
Time frame: Up to 96 weeks
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Mainz, Germany
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