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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.)
Sponsor:
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )
ClinicalTrials.gov Identifier:
NCT01597596
First received: May 10, 2012
Last updated: December 14, 2015
Last verified: December 2015

May 10, 2012
December 14, 2015
August 2012
December 2014   (Final data collection date for primary outcome measure)
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.
Change from baseline in cardiac function as measured by the left ventricular mass index Z-score(LVMI-Z) [ Time Frame: 52 weeks ]
Complete list of historical versions of study NCT01597596 on ClinicalTrials.gov Archive Site
  • Percentage of Participants With Estimated Probability of Survival [ Time Frame: Up to Week 52 ]
  • 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.
  • 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.
  • Estimated probability of survival [ Time Frame: 52 weeks ]
  • Probability of invasive ventilator-free survival [ Time Frame: 52 weeks ]
  • Change in motor development status as assessed by the Gross Motor Function Measure - 88 Scale (GMFM-88) total percent scores [ Time Frame: 52 weeks ]
  • Number of Treatment-emergent Serious Adverse Events (SAEs) and Adverse Events (AEs) [ Time Frame: 52 weeks ]
Not Provided
Not Provided
 
A Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment Naïve Patients With Infantile-Onset Pompe Disease
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
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.
Not Provided
Interventional
Phase 4
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
  • Pompe Disease (Infantile-Onset)
  • Glycogen Storage Disease Type II (GSD II)
  • Glycogenosis 2
  • Acid Maltase Deficiency
  • Biological: alglucosidase alfa
    Intravenous (IV) infusion of alglucosidase alfa (4000 L material) 20 mg/kg every other week (QOW)
    Other Name: Lumizyme
  • Biological: alglucosidase alfa
    IV infusion of alglucosidase alfa (160 L material) 20 mg/kg QOW.
    Other Name: Myozyme
  • Experimental: Alglucosidase Alfa 4000 L material (Non-US participants)
    Alglucosidase alfa 4000 L material for 52 weeks.
    Intervention: Biological: alglucosidase alfa
  • Active Comparator: Alglucosidase Alfa 160 L material (US participants)
    Alglucosidase alfa 160 L material for 52 weeks.
    Intervention: Biological: alglucosidase alfa
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Terminated
4
December 2014
December 2014   (Final data collection date for primary outcome measure)

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.
Sexes Eligible for Study: All
up to 12 Months   (Child)
No
Contact information is only displayed when the study is recruiting subjects
United States,   Germany,   Taiwan
 
 
NCT01597596
AGLU07510, 2011-005595-42
Yes
Not Provided
Not Provided
Not Provided
Genzyme, a Sanofi Company
Genzyme, a Sanofi Company
Not Provided
Study Director: Medical Monitor Genzyme, a Sanofi Company
Sanofi
December 2015

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP