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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 is a clinical trial that has completed, run by University of Florida. The registered enrollment target is 11 participants.

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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.

Interventions

  • DRUG Rituximab
  • DRUG Miglustat

Trial Details

FieldValue
Enrollment Target 11 participants
Start Date 2008-10
Est. Completion 2017-10

Sponsor

University of Florida

1,106 total trials

What the Registry Record Tells You About NCT01451879

The ClinicalTrials.gov registry entry for NCT01451879 describes a study currently listed as completed, categorized as an unspecified phase. The registered enrollment target is 11 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is University of Florida, which has 1,106 total studies on file at ClinicalTrials.gov.

The record links to 0 conditions, and to 2 interventions - of which Rituximab is the first listed.

NCT01451879 reports 0 study locations.

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.

Data sourced from official public datasets. See our methodology for details. Retrieved and formatted by PlainTrial

Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page mirrors this trial's own ClinicalTrials.gov registry record, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.