Status:
COMPLETED
Observational Study for Subjects With Pompe Disease Undergoing Immune Modulation Therapies
Lead Sponsor:
University of Florida
Conditions:
Pompe Disease
Eligibility:
All Genders
Up to 65 years
Brief Summary
Hypothesis: the effectiveness of treatment of Pompe Disease with rhGAA enzyme replacement therapy (ERT) is limited at least in part because patients develop antibodies against the provided rhGAA enzym...
Detailed Description
The purpose of this research study is to determine the effect(s) of medications that alter the immune system on anti-rhGAA immune response in Pompe patients receiving rhGAA enzyme replacement therapy ...
Eligibility Criteria
Inclusion
- patients between the ages of 0 months and 65 years
- diagnosed with early-onset Pompe Disease, confirmed by mutational analysis and/or GAA enzyme assay
- eligible regardless of whether they have begun enzyme replacement therapy prior to enrollment
- all subjects will receive ERT as standard of care during the course of the study, although they may not have begun ERT treatment at the time of enrollment
- subjects may receive an immunomodulatory regimen as part of their standard of care; this may include rituximab, sirolimus, methotrexate, Gamunex, Hizentra, Zavesca or other immunomodulatory agents, alone or in combination, at the discretion of their caregiver(s)
Exclusion
- subject is unable to meet the study requirements
- subjects medical condition contraindicates participation or Study Investigators feel that participation is otherwise not in the subject's best interest
- subject does not receive ERT treatment
Key Trial Info
Start Date :
October 1 2008
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
October 1 2017
Estimated Enrollment :
11 Patients enrolled
Trial Details
Trial ID
NCT01451879
Start Date
October 1 2008
End Date
October 1 2017
Last Update
December 8 2017
Active Locations (1)
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1
University of Florida
Gainesville, Florida, United States, 32610