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 ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )
ClinicalTrials.gov Identifier:
NCT01597596
First received: May 10, 2012
Last updated: September 11, 2014
Last verified: September 2014
  Purpose

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.


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 as measured by the left ventricular mass Z-score(LVM-Z) [ Time Frame: 52 weeks ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • 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 ]

Estimated Enrollment: 24
Study Start Date: August 2012
Estimated Study Completion Date: August 2017
Estimated Primary Completion Date: July 2017 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: alglucosidase alfa (4000 L material)
Non-US patients will receive alglucosidase alfa (4000 L material)
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
Active Comparator: alglucosidase alfa (160 L material)
US patients will receive alglucosidase alfa (160 L material)
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

  Eligibility

Ages Eligible for Study:   up to 12 Months
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01597596

Locations
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
Study Director: Medical Monitor Genzyme, a Sanofi Company
  More Information

No publications provided

Responsible Party: Sanofi ( Genzyme, a Sanofi Company )
ClinicalTrials.gov Identifier: NCT01597596     History of Changes
Other Study ID Numbers: AGLU07510, 2011-005595-42
Study First Received: May 10, 2012
Last Updated: September 11, 2014
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

ClinicalTrials.gov processed this record on October 01, 2014