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NCT01451879 · ClinicalTrials.gov registry record
Observational Study for Subjects With Pompe Disease Undergoing Immune Modulation Therapies
A clinical trial, sponsored by University of Florida.
- Completed
- Registry status
- 11
- Enrollment target
NCT01451879: Completed study, sponsored by University of Florida.
NCT01451879 is a clinical trial that has completed, run by University of Florida. The registered enrollment target is 11 participants. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT01451879 has completed, sponsored by University of Florida.
- COMPLETED
- Registry status
- 11 participants
- Enrollment target
Study 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 immunomodulatory drugs may dampen or eliminate the anti-rhGAA immune response in patients receiving ERT, thereby allowing for greater ERT efficacy. Studying the immune response to rhGAA may provide valuable insight into the role of the immune system in the effectiveness of ERT for Pompe Disease.
Primary Outcome
Antibody titer for anti-rh-GAA will be evaluated at baseline and every 4-8 weeks for 52 weeks of participation in the primary study. For subjects who continue participation in the extension study (\>52 weeks - 5 years), anti-rh-GAA antibody titers will be evaluated every 12 - 24 weeks.
Interventions
- DRUG Rituximab
- DRUG Miglustat
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 11 participants |
| Start Date | 2008-10 |
| Est. Completion | 2017-10 |
What the finished NCT01451879 record still lists
NCT01451879 is an observational study that tracks outcomes without assigning an intervention. The registry caps enrollment at 11 participants, a relatively small participant target.
The record links to 0 conditions, and to 2 interventions - of which Rituximab is the first listed.
NCT01451879 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT01451879 about?
NCT01451879 is a clinical study titled "Observational Study for Subjects With Pompe Disease Undergoing Immune Modulation Therapies". 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 immunomodulatory drugs may dampen or eliminate the anti-rhGAA immune response in p...
What is the current status of trial NCT01451879?
This trial is currently completed. The enrollment target is 11 participants. The study started on 2008-10. Estimated completion is 2017-10.
What interventions are being tested in trial NCT01451879?
The interventions under investigation include: Rituximab (DRUG), Miglustat (DRUG).
Who is sponsoring clinical trial NCT01451879?
This trial is sponsored by University of Florida, which has 1,106 total clinical trials registered on ClinicalTrials.gov.
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