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Trial record 1 of 1 for:    aglu07510
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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

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ClinicalTrials.gov Identifier: NCT01597596
Recruitment Status : Terminated (Study was stopped due to approved label expansion of alglucosidase alfa.)
First Posted : May 14, 2012
Results First Posted : January 18, 2016
Last Update Posted : January 18, 2016
Sponsor:
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )

Study Description
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Brief Summary:
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 or disease Intervention/treatment Phase
Pompe Disease (Infantile-Onset) Glycogen Storage Disease Type II (GSD II) Glycogenosis 2 Acid Maltase Deficiency Biological: alglucosidase alfa Phase 4

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 4 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (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
Study Start Date : August 2012
Actual Primary Completion Date : December 2014
Actual Study Completion Date : December 2014

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
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


Outcome Measures
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Primary Outcome Measures :
  1. Change From Baseline in Cardiac Function at Week 52 [ Time Frame: Baseline, Week 52 ]
    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 :
  1. Percentage of Participants With Estimated Probability of Survival [ Time Frame: Up to Week 52 ]
  2. Number of Participants With Invasive Ventilator-Free Survival [ Time Frame: Up to Week 52 ]
    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.

  3. Change From Baseline in Motor Development Status at Week 52 [ Time Frame: Baseline, Week 52 ]
    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.


Eligibility Criteria
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Ages Eligible for Study:   up to 12 Months   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01597596


Locations
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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
Germany
Geiben, Germany
Mainz, Germany
Taiwan
Taipei, Taiwan
Sponsors and Collaborators
Genzyme, a Sanofi Company
Investigators
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Study Director: Medical Monitor Genzyme, a Sanofi Company
More Information
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Responsible Party: Genzyme, a Sanofi Company
ClinicalTrials.gov Identifier: NCT01597596    
Other Study ID Numbers: AGLU07510
2011-005595-42 ( EudraCT Number )
First Posted: May 14, 2012    Key Record Dates
Results First Posted: January 18, 2016
Last Update Posted: January 18, 2016
Last Verified: December 2015
Additional relevant MeSH terms:
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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