Pompe Disease Clinical Trial #2

Pompe Disease is a genetic disorder that causes glycogen to build up in your body. As the enzyme builds up on your muscles and organs, it causes them to deteriorate. It often results in weak muscles, an enlarged heart and liver, and breathing difficulties, making it a dangerous condition.

In this trial we will study a new combination treatment (an investigational enzyme replacement therapy and oral drug ) for Pompe Disease in adolescents.

Who can take part in the study?

We are conducting a new combination treatment for adolescents diagnosed with Pompe Disease. The trial alternates between enzyme replacement therapy and oral medication.

Fabry-Disease-Clinical-BALANCE-Study

Age

12-18 years old

Patient Compensation:

$200 per visit (about 30 visits total)

Key Inclusion and Exclusion Criteria:

  • Diagnosis of Late Onset Pompe Disease
  • May be ERT naïve or experienced
  • You are able to walk at least 75 meters (82 yards)

Patient Commitment Duration:

About 60 weeks with the option to enroll in an open-label extension study

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I always actually enjoy my time at Infusion Associates. The nursing staff there are so kind and considerate that I am as comfortable as if I were at home in my own living room. Thank you Infusion Associates for your loving care

 

 

Louann Reisbig

 

Interested in Other Enrolling Clinical Trials?

All of our studies are approved by the Food and Drug Administration (FDA). To find out more, click on the links below.

Fabry Disease Trial #2