A Pilot Study of Zavesca® in Patients With Pompe Disease and Infusion Associated Reaction
Pompe Disease, Hypersensitivity Reaction
About this trial
This is an interventional treatment trial for Pompe Disease focused on measuring Pompe Disease, Enzyme Replacement Therapy, Infusion Associated Reaction
Eligibility Criteria
Inclusion Criteria:
Subjects will be patients between the ages of 18 years and 65 years who have been diagnosed with Pompe Disease, confirmed by mutational analysis and/or GAA enzyme activity assay.
- Receiving rhGAA ERT
- Willing to travel to the study site for study assessments
- Willingness of local medical treatment provider to continue treating study participant with addition of Zavesca® to treatment plan.
- Willingness of study participant to modify dietary intake on day of infusion *All Subjects will continue enzyme replacement therapy as standard of care, as prescribed by local medical treatment provider during the course of the Study.
Exclusion Criteria:
- Subject is unable to meet the study requirements
- Subject's 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
- Participation in other interventional studies at the time of enrollment that may interfere with this study (at the investigator's discretion)
- Unable to travel to the University of Florida for study visits
Sites / Locations
Arms of the Study
Arm 1
Arm 2
Active Comparator
Active Comparator
Zavesca® 100 mg
Zavesca® 300 mg
3 study participants are given Zavesca® prescription 100 mg for administration before ERT infusion. Week 0 infusion is completed at study site, with blood collection for anti-GAA antibody level before, during and after the ERT infusion. A punch muscle biopsy is completed the day after ERT infusion with pre-medication Zavesca®. Health Survey is completed. Week 2, 4, and 6 ERT infusion with pre-medication are completed at local/home infusion center. Travel to site for week 7 study visit includes physical exam, blood collection and punch muscle biopsy. Health survey is completed.
3 study participants are given Zavesca® prescription 300 mg for administration before ERT infusion. Week 0 infusion is completed at study site, with blood collection for anti-GAA antibody level before, during and after the ERT infusion. A punch muscle biopsy is completed the day after ERT infusion with pre-medication Zavesca®. Health Survey is completed. Week 2, 4, and 6 ERT infusion with pre-medication are completed at local/home infusion center. Travel to site for week 7 study visit includes physical exam, blood collection and punch muscle biopsy. Health survey is completed.