A Study to Evaluate the Efficacy and Safety of Alglucosidase Alfa Produced at the 4000 L Scale for Pompe Disease

Phase 4

Terminated

• Conditions: Pompe Disease
• Interventions: Drug: Alglucosidase alfa

• Registration Number: NCT01526785
• Lead Sponsor: Genzyme, a Sanofi Company

Brief Summary

The objective of this study was to evaluate the efficacy and safety of treatment with 4000 litre (L) alglucosidase alfa (Lumizyme®) in Pompe participants.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2012-02-02
• Study First Submitted QC: 2012-02-03
• Study First Posted: 2012-02-06
• Study Start: 2012-03
• Primary Completion: 2014-12
• Study Completion: 2014-12
• Results First Submitted: 2015-09-29
• Results First Submitted QC: 2015-09-29
• Results First Posted: 2015-10-27
• Status Verified: 2015-11
• Last Update Submitted: 2015-11-09
• Last Update Posted: 2015-12-08

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 113

Inclusion Criteria

A participant might meet all of the following criteria to be eligible for this study.

1. The participant and/or their parent/legal guardian was willing and able to provide signed informed consent.
2. The participant might be at least 1 year of age at the time of informed consent.
3. The participant had a diagnosis of Pompe disease and might have received treatment with 160 L alglucosidase alfa prior to screening.
4. The participant, if female and of childbearing potential, might have a negative pregnancy test (urine beta-human chorionic gonadotropin) at baseline. Note: all female participants of childbearing potential and sexually mature males might agree to use a medically accepted method of contraception throughout the study.

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Exclusion Criteria

A participant who met any of the following criteria were to be excluded from this study.

1. The participant had within the past 3 months received or was currently receiving any investigational product other than 160 L alglucosidase alfa or was currently participating in another clinical treatment study.
2. The participant, in the opinion of the Investigator, was clinically unstable and would not be expected to survive to completion of the 52-week treatment period.
3. The participant and/or their parent/legal guardian, in the opinion of the Investigator, was unable to adhere to the requirements of the study.

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Alglucosidase alfa	Alglucosidase alfa	Alglucosidase alfa (4000 L scale) intravenous (IV) infusion administered for 52 weeks as per physician's routine practice.

Primary Outcome Measures

Name	Time	Method
Percentage of Participants Who Were Clinically Stable or Improved at Week 52	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.

Secondary Outcome Measures

Name	Time	Method
Survival Rate at Week 52	Week 52	Percentage of participants who were alive at Week 52, were reported. Survival rate was calculated by Kaplan-Meier estimate.
Change From Baseline on Gross Motor Function Measure-88 (GMFM-88) at Week 52	Baseline, 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.
Change From Baseline in Forced Vital Capacity (FVC) at Week 52- At Supine Position	Baseline, Week 52	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%.
Change From Baseline in Forced Vital Capacity (FVC) at Week 52- At Sitting Position	Baseline, Week 52	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%.
Invasive Ventilator-Free Survival Rate at Week 52	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.
Change From Baseline on Left Ventricular Mass Z-Score (LVM-Z) at Week 52	Baseline, 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.