Pompe Disease Clinical Trial
Official title:
Effects of Immunomodulation Therapy on Anti-rhGAA Immune Response in Subjects With Pompe Disease Receiving rhGAA Enzyme Replacement Therapy
Verified date | December 2017 |
Source | University of Florida |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Observational |
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 enzyme. Treatment with immunomodulatory drugs may dampen or eliminate the anti-rhGAA immune response in patients receiving ERT, thereby allowing for greater ERT efficacy. Studying the immune response to rhGAA may provide valuable insight into the role of the immune system in the effectiveness of ERT for Pompe Disease.
Status | Completed |
Enrollment | 11 |
Est. completion date | October 2017 |
Est. primary completion date | October 2017 |
Accepts healthy volunteers | No |
Gender | All |
Age group | N/A to 65 Years |
Eligibility |
Inclusion Criteria: - 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 Criteria: - 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 |
Country | Name | City | State |
---|---|---|---|
United States | University of Florida | Gainesville | Florida |
Lead Sponsor | Collaborator |
---|---|
University of Florida |
United States,
Elder ME, Nayak S, Collins SW, Lawson LA, Kelley JS, Herzog RW, Modica RF, Lew J, Lawrence RM, Byrne BJ. B-Cell depletion and immunomodulation before initiation of enzyme replacement therapy blocks the immune response to acid alpha-glucosidase in infantil — View Citation
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Anti-rh GAA antibody titers | Antibody titer for anti-rh-GAA will be evaluated at baseline and every 4-8 weeks for 52 weeks of participation in the primary study. For subjects who continue participation in the extension study (>52 weeks - 5 years), anti-rh-GAA antibody titers will be evaluated every 12 - 24 weeks. | 52 weeks | |
Secondary | B-lymphocyte antigen (CD20) level | Reports from clinical lab: B-lymphocyte antigen (CD20) will be added to the study record when available every 4-12 weeks during the primary study and every 12 weeks for subjects who participate in the extension study (>52 weeks - 6 years) | 52 weeks |
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