Status:
TERMINATED
A Pilot Study of Zavesca® in Patients With Pompe Disease and Infusion Associated Reaction
Lead Sponsor:
University of Florida
Collaborating Sponsors:
Amicus Therapeutics
Conditions:
Pompe Disease
Hypersensitivity Reaction
Eligibility:
All Genders
18-65 years
Phase:
PHASE1
Brief 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 enzym...
Detailed Description
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. Sub...
Eligibility Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
October 1 2016
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
July 1 2018
Estimated Enrollment :
2 Patients enrolled
Trial Details
Trial ID
NCT02185651
Start Date
October 1 2016
End Date
July 1 2018
Last Update
December 5 2018
Active Locations (0)
Enter a location and click search to find clinical trials sorted by distance.
No Results Found
We couldn’t find results for the location/zipcode entered or within the selected range. Please check your input or adjust your search.