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

• Conditions: Pompe disease (acid alpha-glucosidase deficiency); MedDRA version: 17.1Level: LLTClassification code 10036143Term: Pompe's diseaseSystem Organ Class: 100000004850; Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]

• Registration Number: EUCTR2015-000512-18-Outside-EU/EEA
• Lead Sponsor: Genzyme Corporation Inc

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2015-03-02
• Last Update Posted: 9 March 2015

Recruitment & Eligibility

• Status: A
• Sex: All
• Target Recruitment: 114

Inclusion Criteria

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

Are the trial subjects under 18? yes
Number of subjects for this age range: 113
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 1
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

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

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: The objective of this study is to evaluate the efficacy and safety of treatment with 4000 L alglucosidase alfa (Lumizyme®) in Pompe patients.<br><br><br>;Secondary Objective: Not applicable;Primary end point(s): Percentage of participants who are clinically stable or improved at Week 52;Timepoint(s) of evaluation of this end point: Percentage of participants who are clinically stable or improved at Week 52 [ Time Frame: Week 52 ]

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): Percent survival <br><br>Percentage of participants who are invasive ventilator-free<br><br>Change from baseline on left ventricular mass Z-score (LVM-Z)<br><br>Change from baseline on Gross Motor Function Measure-88 (GMFM-88) Total Percent Score <br><br>Change from baseline in forced vital capacity (FVC) % predicted ;Timepoint(s) of evaluation of this end point: Percent survival at Week 52 [ Time Frame: Week 52 ]<br>Percentage of participants who are invasive ventilator-free at Week 52 [ Time Frame: Week 52 ]<br>Change from baseline on left ventricular mass Z-score (LVM-Z) [ Time Frame: Baseline and Week 52 ]<br>Change from baseline on Gross Motor Function Measure-88 (GMFM-88) Total Percent Score [ Time Frame: Baseline and Week 52 ] <br>Change from baseline in forced vital capacity (FVC) % predicted [ Time Frame: Baseline and Week 52 ] <br><br>