Immune Modulation Therapy for Pompe Disease
Immune Modulation Therapy for Pompe Disease
The purpose of this study is to assess anti-recombinant human acid α-glucosidase (anti-rhGAA)
 antibody titers after treatment with immune modulation therapy in patients of Pompe disease.
Inclusion Criteria:

 -  The patient (and/or patient's legal guardian if patient is < 18years) must provide
 written informed consent prior to any study-related procedures that are performed;

 -  The patient must have a confirmed diagnosis of Pompe disease defined as a documented
 acid α-glucosidase (GAA) enzyme deficiency from blood samples or 2 GAA gene mutations;

 -  The patient (and/or legal guardian) must have ability to comply with clinical
 protocol;

 -  Regimen A only: The patient is receiving enzyme replacement therapy, exhibits clinical
 decline, and has persistent high anti-recombinant human acid α-glucosidase
 (anti-rhGAA) antibody titers and/or tested positive for antibodies that inhibit
 enzymatic activity and/or uptake of Myozyme;

 -  Regimen B only: The patient is cross-reactive immune material (CRIM) -negative AND
 have not received Myozyme infusion prior to enrollment

 Exclusion Criteria:

 -  The patient is at risk of reactivation or is a carrier of Hepatitis B or Hepatitis C;

 -  The patient is at risk of reactivation of tuberculosis or has regular contact with
 individuals who are being actively treated for tuberculosis;

 -  The patient has used any investigational product (other than alglucosidase alfa)
 within 30 days prior to study enrollment;

 -  The patient is pregnant or lactating;

 -  The patient has had or is required to have any live vaccination within one month prior
 to enrollment.