A Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Patients With Infantile-Onset Pompe Disease Who Have Never Been Treated

This study is currently recruiting participants. (see Contacts and Locations)
Verified August 2014 by Sanofi
Sponsor:
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )
ClinicalTrials.gov Identifier:
NCT01597596
First received: May 10, 2012
Last updated: August 11, 2014
Last verified: August 2014

May 10, 2012
August 11, 2014
August 2012
July 2017   (final data collection date for primary outcome measure)
Change from baseline in cardiac function as measured by the left ventricular mass Z-score(LVM-Z) [ Time Frame: 52 weeks ] [ Designated as safety issue: No ]
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
  • 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 ]
Same as current
Not Provided
Not Provided
 
A Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Patients With Infantile-Onset Pompe Disease Who Have Never Been Treated
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 L and 4000 L scales in patients who have been diagnosed with infantile-onset Pompe disease. Patients will be treated with alglucosidase alfa 160 L scale product in the 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) infusions of alglucosidase alfa (4000 L material) at a dose of 20 mg/kg of body weight every other week (qow)
    Other Names:
    • Myozyme
    • Lumizyme
  • Biological: alglucosidase alfa
    Intravenous (IV) infusions of alglucosidase alfa (160 L material) at a dose of 20 mg/kg of body weight every other week (qow)
    Other Name: Myozyme
  • Experimental: alglucosidase alfa (4000 L material)
    Non-US patients will receive alglucosidase alfa (4000 L material)
    Intervention: Biological: alglucosidase alfa
  • Active Comparator: alglucosidase alfa (160 L material)
    US patients will receive alglucosidase alfa (160 L material)
    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.
 
Recruiting
24
August 2017
July 2017   (final data collection date for primary outcome measure)

Inclusion Criteria:

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

Exclusion Criteria:

  • The patient is cross-reactive immunologic material negative.
  • The patient requires invasive ventilator support at the time of enrollment.
  • The patient has decompensated clinical heart failure.
  • The patient has a major congenital abnormality, excluding cardiac hypertrophy.
  • The patient has a clinically significant organ disease (excluding the signs and symptoms of Pompe disease).
  • The patient is currently receiving any investigational product.
  • The patient is participating in another clinical study.
  • The patient and/or the patient's parent/legal guardian is unable to adhere to the requirements of the study.
Both
up to 12 Months
No
Contact: Medical Information medinfo@genzyme.com
United States,   Germany,   Taiwan
 
NCT01597596
AGLU07510, 2011-005595-42
Yes
Sanofi ( Genzyme, a Sanofi Company )
Genzyme, a Sanofi Company
Not Provided
Study Director: Medical Monitor Genzyme, a Sanofi Company
Sanofi
August 2014

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