A Study to Evaluate the Efficacy and Safety of Alglucosidase Alfa Produced at the 4000 L Scale for Pompe Disease
The objective of this study is to evaluate the efficacy and safety of treatment with 4000 L alglucosidase alfa (Lumizyme®) in Pompe patients.
|Study Design:||Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||A Phase 4 Open Label, Prospective Study in Patients With Pompe Disease to Evaluate The Efficacy and Safety of Alglucosidase Alfa Produced at the 4000L Scale|
- Percentage of participants who are clinically stable or improved at Week 52 [ Time Frame: Week 52 ] [ Designated as safety issue: No ]
Clinical stability defined as the absence of any of the following definitions of clinical worsening:
- death due to disease progression or new dependency on invasive ventilation;
- decline in cardiac status from baseline with an increase in left ventricular mass Z-score of >1 Z-score above a Z-score of 2;
- decline in motor function from baseline as measured by an absolute decrease in Gross Motor Function Measure-88 Total Percent Score of ≥8%;
- decline in pulmonary function from baseline with a decrease of ≥15% in forced vital capacity % predicted in the sitting position.
- Percent survival at Week 52 [ Time Frame: Week 52 ] [ Designated as safety issue: No ]Kaplan-Meier methodology will be applied to estimate the percentage of patients treated with 4000 L alglucosidase alfa who are alive at Week 52.
- Percentage of participants who are invasive ventilator-free at Week 52 [ Time Frame: Week 52 ] [ Designated as safety issue: No ]Invasive ventilation is defined as mechanical ventilatory support applied with the use of an endotracheal tube or tracheostomy. Invasive ventilator dependence will be defined as the time to the first use of invasive ventilation which is not followed by an invasive ventilator-free period of at least 14 consecutive days. Kaplan-Meier methodology will be applied to estimate the percentage of patients who are alive and free of invasive ventilator dependence at Week 52.
- Change from baseline on left ventricular mass Z-score (LVM-Z) [ Time Frame: Baseline and Week 52 ] [ Designated as safety issue: No ]
- Change from baseline on Gross Motor Function Measure-88 (GMFM-88) Total Percent Score [ Time Frame: Baseline and Week 52 ] [ Designated as safety issue: No ]The GMFM-88 consists of 88 items organized into 5 categories: lying and rolling, sitting, crawling and kneeling, standing, walking, running and jumping. Each item is scored on a 4-point Likert scale (i.e., 0=cannot do; 1=initiates [<10% of the task]; 2=partially completes [10 to <100 % of the task]; 3=task completion). The score for each dimension is expressed as a percentage of the maximum score for that dimension. The total score is obtained by adding the percentage scores for each dimension and dividing the sum by the total number of dimensions.
- Change from baseline in forced vital capacity (FVC) % predicted [ Time Frame: Baseline and Week 52 ] [ Designated as safety issue: No ]FVC % predicted in a sitting position will be measured in accordance with the American Thoracic Society guidelines.
|Study Start Date:||March 2012|
|Estimated Study Completion Date:||March 2015|
|Estimated Primary Completion Date:||March 2015 (Final data collection date for primary outcome measure)|
|Experimental: Alglucosidase alfa||
Drug: Alglucosidase alfa
4000 L alglucosidase alfa administered by intravenous infusion at the same dose and dose regimen used for the patient's routine treatment prior to the study for 52 weeks.
Other Name: Lumizyme®
|Contact: Medical Informationfirstname.lastname@example.org|
|Contact: Medical Informationemail@example.com|
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|Study Director:||Medical Monitor||Genzyme, a Sanofi Company|