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A Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment Naïve Patients With Infantile-Onset Pompe Disease

This study has been terminated.
(Study was stopped due to approved label expansion of alglucosidase alfa.)
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company ) Identifier:
First received: May 10, 2012
Last updated: December 14, 2015
Last verified: December 2015
A study to demonstrate comparable safety, efficacy, and pharmacokinetics (PK) of alglucosidase alfa manufactured at the 160 litre (L) and 4000 L scales in participants who had been diagnosed with infantile-onset Pompe disease. Participants were treated with alglucosidase alfa 160 L scale product in the United States (US) and 4000 L scale product in the regions outside the US.

Condition Intervention Phase
Pompe Disease (Infantile-Onset)
Glycogen Storage Disease Type II (GSD II)
Glycogenosis 2
Acid Maltase Deficiency
Biological: alglucosidase alfa
Phase 4

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Phase 3/4, Prospective, Multinational, Open-label, Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment Naïve Patients With Infantile-Onset Pompe Disease

Resource links provided by NLM:

Further study details as provided by Sanofi:

Primary Outcome Measures:
  • Change From Baseline in Cardiac Function at Week 52 [ Time Frame: Baseline, Week 52 ] [ Designated as safety issue: No ]
    Cardiac function was measured by the left ventricular mass Z-score (LVM-Z). Z-Scores indicate the number of standard deviations (SD) from the mean in a normal distribution. A negative change from baseline indicates a decrease and positive 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.

Secondary Outcome Measures:
  • Percentage of Participants With Estimated Probability of Survival [ Time Frame: Up to Week 52 ] [ Designated as safety issue: No ]
  • Number of Participants With Invasive Ventilator-Free Survival [ Time Frame: Up to Week 52 ] [ Designated as safety issue: No ]
    Invasive ventilator-free survival was defined as the time during which the participant is alive and not invasively ventilated. Number of Participants with invasive ventilator-free survival were reported.

  • Change From Baseline in Motor Development Status at Week 52 [ Time Frame: Baseline, Week 52 ] [ Designated as safety issue: No ]
    Motor development status was assessed by the Gross Motor Function Measure - 88 Scale (GMFM-88) total percent scores. GMFM-88 is an 88-item measure to detect gross motor function. It consists of 5 categories: lying and rolling; sitting; crawling and kneeling; standing; walking, running and jumping. Each item was scored on a 4-point Likert scale (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 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.

Enrollment: 4
Study Start Date: August 2012
Study Completion Date: December 2014
Primary Completion Date: December 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Alglucosidase Alfa 4000 L material (Non-US participants)
Alglucosidase alfa 4000 L material for 52 weeks.
Biological: alglucosidase alfa
Intravenous (IV) infusion of alglucosidase alfa (4000 L material) 20 mg/kg every other week (QOW)
Other Name: Lumizyme
Active Comparator: Alglucosidase Alfa 160 L material (US participants)
Alglucosidase alfa 160 L material for 52 weeks.
Biological: alglucosidase alfa
IV infusion of alglucosidase alfa (160 L material) 20 mg/kg QOW.
Other Name: Myozyme


Ages Eligible for Study:   up to 12 Months   (Child)
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • The participant's parent/legal guardian was willing and able to provide signed informed consent.
  • The participant might be less than or equal to 12 months of age.
  • The participant might have documented GAA enzyme deficiency from blood, skin, or muscle tissue.
  • The participant might be naïve to treatment with alglucosidase alfa.

Exclusion Criteria:

  • The participant was cross-reactive immunologic material negative.
  • The participant required invasive ventilator support at the time of enrollment.
  • The participant had decompensated clinical heart failure.
  • The participant had a major congenital abnormality, excluding cardiac hypertrophy.
  • The participant had a clinically significant organ disease (excluding the signs and symptoms of Pompe disease).
  • The participant was currently receiving any investigational product.
  • The participant was participating in another clinical study.
  • The participant and/or the patient's parent/legal guardian was unable to adhere to the requirements of the study.
  Contacts and Locations
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Please refer to this study by its identifier: NCT01597596

United States, Arkansas
Little Rock, Arkansas, United States
United States, California
Oakland, California, United States
United States, Florida
Gainsville, Florida, United States
United States, Georgia
Decatur, Georgia, United States
United States, Illinois
Chicago, Illinois, United States
United States, Massachusetts
Cambridge, Massachusetts, United States
United States, Michigan
Detroit, Michigan, United States
United States, Nevada
Las Vegas, Nevada, United States
United States, New Jersey
New Brunswick, New Jersey, United States
United States, New York
New York, New York, United States
United States, North Carolina
Durham, North Carolina, United States
United States, Ohio
Cincinnati, Ohio, United States
United States, Texas
Fort Worth, Texas, United States
United States, Washington
Seattle, Washington, United States
Geiben, Germany
Mainz, Germany
Taipei, Taiwan
Sponsors and Collaborators
Genzyme, a Sanofi Company
Study Director: Medical Monitor Genzyme, a Sanofi Company
  More Information

Responsible Party: Genzyme, a Sanofi Company Identifier: NCT01597596     History of Changes
Other Study ID Numbers: AGLU07510  2011-005595-42 
Study First Received: May 10, 2012
Results First Received: September 25, 2015
Last Updated: December 14, 2015
Health Authority: United States: Food and Drug Administration
European Union: European Medicines Agency

Additional relevant MeSH terms:
Glycogen Storage Disease Type II
Glycogen Storage Disease
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Carbohydrate Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases processed this record on October 26, 2016