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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.
Sponsor:
Information provided by:
Genzyme
ClinicalTrials.gov Identifier:
NCT00765414
First received: October 2, 2008
Last updated: July 7, 2009
Last verified: September 2008
History of Changes

October 2, 2008
July 7, 2009
April 2003
April 2006   (final data collection date for primary outcome measure)
The objective of this extension study was to monitor the long-term safety and efficacy of a single patient [ Time Frame: 3 years ] [ Designated as safety issue: No ]
Same as current
Complete list of historical versions of study NCT00765414 on ClinicalTrials.gov Archive Site
Not Provided
Not Provided
Not Provided
Not Provided
 
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.
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

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.
Not Provided
Interventional
Phase 2
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
  • Pompe Disease Late-Onset
  • Glycogen Storage Disease Type II GSD II
Biological: Myozyme
30 mg/kg qow f and 40 mg/kg qow
Other Name: Alglucosidase alfa
Experimental: 1
Intervention: Biological: Myozyme
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
1
August 2006
April 2006   (final data collection date for primary outcome measure)

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.
Female
16 Years and older
No
Contact information is only displayed when the study is recruiting subjects
United States
 
NCT00765414
AGLU02103
Yes
Medical Monitor, Genzyme Corporation
Genzyme
Not Provided
Study Director: Medical Monitor Genzyme
Genzyme
September 2008

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