Immune Modulation Therapy for Pompe Disease

• Status: Unknown status
• Trial end date: 2020-07-01
• Target enrollment: 0
• Participant gender: All

Summary

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.

• Phase: Phase 4
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: National Taiwan University Hospital
• Treatments: Antibodies; Bortezomib; gamma-Globulins; Immunoglobulins; Immunoglobulins, Intravenous; Methotrexate; Rho(D) Immune Globulin; Rituximab

Criteria

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.