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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.

This study has been completed.
Information provided by:
Sanofi Identifier:
First received: October 2, 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 a single patient with infantile-onset Pompe disease who were previously treated with rhGAA in a Genzyme study.

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

Study Type: Interventional
Study Design: 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

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 of a single patient [ Time Frame: 3 years ]

Enrollment: 1
Study Start Date: April 2003
Study Completion Date: August 2006
Primary Completion Date: April 2006 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: 1 Biological: Myozyme
30 mg/kg qow f and 40 mg/kg qow
Other Name: Alglucosidase alfa


Ages Eligible for Study:   16 Years and older   (Child, Adult, Senior)
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.
  Contacts and Locations
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Please refer to this study by its identifier: NCT00765414

United States, Texas
University of Texas Health Science Center at San Antonio
San Antonio, Texas, United States
Sponsors and Collaborators
Genzyme, a Sanofi Company
Study Director: Medical Monitor Genzyme, a Sanofi Company
  More Information

Responsible Party: Medical Monitor, Genzyme Corporation Identifier: NCT00765414     History of Changes
Other Study ID Numbers: AGLU02103
Study First Received: October 2, 2008
Last Updated: February 4, 2014

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