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Extension Study of Long-term Safety and Efficacy of Myozyme in Patients With Pompe Disease Who Were Previously Enrolled in Genzyme Sponsored Enzyme Replacement Therapy (ERT) Studies

This study has been completed.
Information provided by:
Sanofi Identifier:
First received: September 30, 2008
Last updated: February 4, 2014
Last verified: February 2014
This extension study was to monitor the long-term safety and efficacy of rhGAA treatment in patients with infantile-onset Pompe disease who were previously treated with rhGAA derived from the Synpac cell line

Condition Intervention Phase
Pompe Disease Infantile-Onset Glycogen Storage Disease Type II Biological: Myozyme Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Multicenter, Open-Label Extension Study of the Long-Term Safety and Efficacy of Recombinant Human Acid α-Glucosidase (rhGAA) in Patients With Pompe Disease (Glycogen Storage Disease Type II) Who Were Previously Enrolled in Genzyme-Sponsored Enzyme Replacement Therapy Studies

Resource links provided by NLM:

Further study details as provided by Sanofi:

Primary Outcome Measures:
  • The objective of this extension study was to monitor the long-term safety and efficacy [ Time Frame: 3 years ]

Enrollment: 7
Study Start Date: April 2003
Study Completion Date: July 2006
Primary Completion Date: June 2006 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: 1 Biological: Myozyme
10 mg/kg or 20 mg/kg qw OR 20 mg/kg or 40 mg/kg qow
Other Name: Alglucosidase alfa


Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • The patient was enrolled in Protocol AGLU-008-01, AGLU-009-02, or AGLU01502
  • The patient's legal guardian(s) provided written informed consent prior to any study related procedures being performed
  • The patient and his/her guardian(s) were able to comply with the clinical protocol, which required extensive clinical evaluations for an extended period of time.

Exclusion Criteria:

  • Patients were excluded from this study if they did not meet the specific inclusion criteria, or if the patient experienced any unmanageable AE in Protocol AGLU-008-01, AGLU-009-02, or AGLU01502 (as determined and agreed upon by the Principal Investigator and Genzyme Corporation), due to Synpac rhGAA therapy, that would preclude continuing therapy
  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 identifier: NCT00763932

United States, California
Children's Hospital of Oakland
Oakland, California, United States
Children's Hospital of Orange Country
Orange, California, United States
United States, Nebraska
University of Nebraska Medical Center
Omaha, Nebraska, United States
United States, New Jersey
Institute for Genetic Medicine, Saint Peter's University Hospital
New Brunswick, New Jersey, United States
United States, New York
New York University [NYU] School of Medicine
New York, New York, United States
Pediatrique Hospital Debrousse
Lyon, France
Hôpital Porte Madeleine
Orleans, France
South Africa
The Morningside Clinic
Johannesburg, South Africa
Sponsors and Collaborators
Genzyme, a Sanofi Company
Study Director: Medical Monitor Genzyme, a Sanofi Company
  More Information

Additional Information:
Responsible Party: Medical Monitor, Genzyme Corporation Identifier: NCT00763932     History of Changes
Other Study ID Numbers: AGLU02003
Study First Received: September 30, 2008
Last Updated: February 4, 2014

Additional relevant MeSH terms:
Glycogen Storage Disease Type II
Glycogen Storage Disease
Metabolic Diseases
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 processed this record on September 21, 2017