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Your journey
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More info
You can access this
clinical trial
if you have
Pompe Disease
and you are
under 65
years old
This is an observational trial.
You are contributing to medical knowledge about your condition.
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The purpose

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.

Provided treatments

  • Drug: Rituximab
  • Drug: Miglustat

Locations near you

Unfortunately, there are no recruiting locations near you. Please check the list with all locations below.
Tris trial is registered with FDA with number: NCT01451879. The sponsor of the trial is University of Florida and it is looking for 11 volunteers for the current phase.
Official trial title:
Effects of Immunomodulation Therapy on Anti-rhGAA Immune Response in Subjects With Pompe Disease Receiving rhGAA Enzyme Replacement Therapy