The objective of this study was to evaluate the efficacy and safety of treatment with 4000 litre (L) alglucosidase alfa (Lumizyme®) in Pompe participants.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
113
4000 L alglucosidase alfa administered by IV infusion at the same dose and dose regimen used for the patient's routine treatment prior to the study for 52 weeks.
Percentage of Participants Who Were Clinically Stable or Improved at Week 52
Clinical stability was defined as absence of death due to disease progression or new dependency on invasive ventilation and; decline in cardiac status, motor function, and pulmonary function from baseline.
Time frame: Week 52
Survival Rate at Week 52
Percentage of participants who were alive at Week 52, were reported. Survival rate was calculated by Kaplan-Meier estimate.
Time frame: Week 52
Invasive Ventilator-Free Survival Rate at Week 52
Percentage of participants, who were invasive ventilator-free at week 52, are reported. Invasive ventilation was defined as mechanical ventilatory support applied with the use of an endotracheal tube or tracheostomy. Invasive ventilator-free survival rate was calculated by Kaplan-Meier estimate.
Time frame: Week 52
Change From Baseline on Left Ventricular Mass Z-Score (LVM-Z) at Week 52
Z-Scores indicate the number of standard deviations (SD) from the mean in a normal distribution. A negative change from baseline indicates an increase in LVM-Z score. The normal range is -2 to 2 and greater than 2 may indicate left ventricular hypertrophy.
Time frame: Baseline, Week 52
Change From Baseline on Gross Motor Function Measure-88 (GMFM-88) at Week 52
GMFM-88 (88-item measure to detect gross motor function) consists of 5 components, each measured on a 4-point Likert scale.The score for each dimension was expressed as a percentage of the maximum score for that dimension.Total score ranges from 0% to 100%, where higher scores indicate better motor functions.
Time frame: Baseline, Week 52
Change From Baseline in Forced Vital Capacity (FVC) at Week 52- At Supine Position
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Unnamed facility
Birmingham, Alabama, United States
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Phoenix, Arizona, United States
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Little Rock, Arkansas, United States
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Oakland, California, United States
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Orange, California, United States
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San Diego, California, United States
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San Jose, California, United States
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Stanford, California, United States
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Aurora, Colorado, United States
Unnamed facility
Washington D.C., District of Columbia, United States
...and 41 more locations
Percent predicted FVC values are reported. FVC is the volume of air that can forcibly be blown out after full inspiration in the upright position, measured in litres. Predicted forced vital capacity is based on a formula using sex, age and height of a person, and is an estimate of healthy lung capacity. Percent of predicted FVC = (observed value)/(predicted value) \* 100%.
Time frame: Baseline, Week 52
Change From Baseline in Forced Vital Capacity (FVC) at Week 52- At Sitting Position
Percent predicted FVC values are reported. FVC is the volume of air that can forcibly be blown out after full inspiration in the upright position, measured in litres. Predicted forced vital capacity is based on a formula using sex, age and height of a person, and is an estimate of healthy lung capacity. Percent of predicted FVC = (observed value)/(predicted value) \* 100%.
Time frame: Baseline, Week 52