Pompe Disease Clinical Trial
Official title:
A Pilot Study of the Effects of Oral Administration of Zavesca® on Anti-rhGAA Immune Response in Subjects With Pompe Disease Receiving rhGAA Enzyme Replacement Therapy
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.
This Study is designed to assess the effects of Zavesca® as immunomodulatory therapy on
anti-rhGAA immune responses in patients with Pompe disease, as well as their health and
disease progression. Subjects will either receive Zavesca® at 100 mg or 300 mg dosing levels
during study participation (n=3 @ 100 mg dosing; n=3 @ 300 mg dosing).
The first 3 subjects enrolled will be prescribed 100 mg Zavesca® 60 minutes prior to ERT
infusion. The subsequent 3 subjects enrolled will be prescribed 300 mg Zavesca® 60 minutes to
ERT infusion.
Eligible participants are on standard ERT for Pompe disease and have a history of infusion
associated reaction. Travel to the study site in Gainesville, Florida is required for 3
visits. Participants are prescribed medication Zavesca® and have blood tests, punch muscle
biopsy, physical exams, and answer questionnaires over 3 months study participation.
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