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
1 other identifier
interventional
1
1 country
1
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_2
Started Apr 2003
Typical duration for phase_2
1 active site
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
Study Start
First participant enrolled
April 1, 2003
CompletedPrimary Completion
Last participant's last visit for primary outcome
April 1, 2006
CompletedStudy Completion
Last participant's last visit for all outcomes
August 1, 2006
CompletedFirst Submitted
Initial submission to the registry
October 2, 2008
CompletedFirst Posted
Study publicly available on registry
October 3, 2008
CompletedFebruary 5, 2014
February 1, 2014
3 years
October 2, 2008
February 4, 2014
Conditions
Outcome Measures
Primary Outcomes (1)
The objective of this extension study was to monitor the long-term safety and efficacy of a single patient
3 years
Study Arms (1)
1
EXPERIMENTALInterventions
Eligibility Criteria
You may qualify if:
- 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.
You may not qualify if:
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (1)
University of Texas Health Science Center at San Antonio
San Antonio, Texas, United States
MeSH Terms
Interventions
Study Officials
- STUDY DIRECTOR
Medical Monitor
Genzyme, a Sanofi Company
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
Study Record Dates
First Submitted
October 2, 2008
First Posted
October 3, 2008
Study Start
April 1, 2003
Primary Completion
April 1, 2006
Study Completion
August 1, 2006
Last Updated
February 5, 2014
Record last verified: 2014-02