A Pilot Study of Zavesca® in Patients With Pompe Disease and Infusion Associated Reaction
Status:
Terminated
Trial end date:
2018-07-01
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 Zavesca® prior to infusion may dampen or eliminate the
anti-rhGAA immune response in patients receiving ERT, thereby allowing for greater ERT
efficacy.
Treatment with Zavesca® before a enzyme replacement therapy (ERT) may decrease the severity
of, or eliminate infusion associated reactions (IAR) in people with Pompe Disease receiving
ERT.