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
Verified date | December 2018 |
Source | University of Florida |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
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.
Status | Terminated |
Enrollment | 2 |
Est. completion date | July 2018 |
Est. primary completion date | July 2018 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 18 Years to 65 Years |
Eligibility |
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 |
Country | Name | City | State |
---|---|---|---|
n/a |
Lead Sponsor | Collaborator |
---|---|
University of Florida | Amicus Therapeutics |
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Evaluate Pharmacodynamics of ERT with pre-medication Zavesca | Change in GAA antibody level from Baseline to week 0: GAA antibody titer is reported at timepoints: pre-infusion, and 6, 12, and 24 hours post-infusion start. Change in GAA antibody level from Baseline to week 6: GAA antibody titer is reported at time points: pre-infusion and post-infusion. |
Baseline, week 0, week 6 | |
Secondary | Evaluate pharmacokinetics of ERT with pre-medication Zavesca® | Change in ERT half life at baseline (without Zavesca®) compared to week 0 (ERT with pre-medication Zavesca®). Change in Maximum plasma concentration (Cmax) at baseline (without Zavesca®) compared to week 0 (ERT with pre-medication Zavesca®). |
Baseline, week 0 | |
Secondary | Evaluate biodistribution of ERT with pre-medication Zavesca®. | Punch muscle biopsy is performed at week 0 and week 7 for biodistribution: Change in glycogen content and muscle fiber morphology is reported. | Week 0, week 7 |
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