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Extension Study of Long-term Safety and Efficacy of Myozyme for a Single Patient With Pompe Disease Who Were Previously Enrolled in Genzyme Sponsored ERT Studies.

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00765414
Recruitment Status : Completed
First Posted : October 3, 2008
Last Update Posted : February 5, 2014
Information provided by:

Brief Summary:
This extension study was to monitor the long-term safety and efficacy of rhGAA treatment in a single patient with infantile-onset Pompe disease who were previously treated with rhGAA in a Genzyme study.

Condition or disease Intervention/treatment Phase
Pompe Disease Late-Onset Glycogen Storage Disease Type II GSD II Biological: Myozyme Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 1 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-Label Extension Study of the Long-Term Safety and Efficacy of Recombinant Human Acid α-Glucosidase (rhGAA) Given as Enyzme Replacement Therapy to a Single Patient With Pompe Disease (Glycogen Storage Disease Type II) Who Were Previously Enrolled in Genzyme-Sponsored Enzyme Replacement Therapy Studies
Study Start Date : April 2003
Actual Primary Completion Date : April 2006
Actual Study Completion Date : August 2006

Arm Intervention/treatment
Experimental: 1 Biological: Myozyme
30 mg/kg qow f and 40 mg/kg qow
Other Name: Alglucosidase alfa

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

Information from the National Library of Medicine

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, Learn About Clinical Studies.

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Ages Eligible for Study:   16 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   Female
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Provide written informed consent prior to participating in any study related procedures;
  • Currently enrolled in Protocol AGLU01402
  • Have the ability to comply with the clinical protocol, which required extensive clinical evaluations for an extended period of time.

Exclusion Criteria:

  • Was pregnant or unwilling to use approved birth control during the course of the study;
  • Had experienced any unmanageable AEs under Protocol AGLU01402 (as determined and agreed upon by the Principal Investigator and sponsor) due to rhGAA that would preclude continuing ERT;
  • Was participating in any other investigational study.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00765414

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United States, Texas
University of Texas Health Science Center at San Antonio
San Antonio, Texas, United States
Sponsors and Collaborators
Genzyme, a Sanofi Company
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Study Director: Medical Monitor Genzyme, a Sanofi Company
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Responsible Party: Medical Monitor, Genzyme Corporation Identifier: NCT00765414    
Other Study ID Numbers: AGLU02103
First Posted: October 3, 2008    Key Record Dates
Last Update Posted: February 5, 2014
Last Verified: February 2014
Additional relevant MeSH terms:
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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