Observational Study for Subjects With Pompe Disease Undergoing Immune Modulation Therapies
Status:
Completed
Trial end date:
2017-10-01
Target enrollment:
Participant gender:
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 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.