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 ] [ 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.
Change from baseline in cardiac function as measured by the left ventricular mass index Z-score(LVMI-Z) [ Time Frame: 52 weeks ] [ Designated as safety issue: No ]
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 ] [ 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.
  • Estimated probability of survival [ Time Frame: 52 weeks ] [ Designated as safety issue: No ]
  • Probability of invasive ventilator-free survival [ Time Frame: 52 weeks ] [ Designated as safety issue: No ]
  • Change in motor development status as assessed by the Gross Motor Function Measure - 88 Scale (GMFM-88) total percent scores [ Time Frame: 52 weeks ] [ Designated as safety issue: No ]
  • Number of Treatment-emergent Serious Adverse Events (SAEs) and Adverse Events (AEs) [ Time Frame: 52 weeks ] [ Designated as safety issue: Yes ]
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
Endpoint Classification: Safety/Efficacy Study
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.
Both
up to 12 Months
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
Sanofi ( 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