Immune Modulation Therapy for Pompe Disease

Phase 4 Unknown 8 enrolled National Taiwan University Hospital

NaSingle-groupOpen-labelTreatment

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.

Timeline

Start: 2015-08
Primary completion: 2020-07
Completion: 2020-07

Publications

Chen HA, Hsu RH, Fang CY, Desai AK, Lee NC, Hwu WL, Tsai FJ, Kishnani PS, Chien YH. Optimizing treatment outcomes: immune tolerance induction in Pompe disease patients undergoing enzyme replacement therapy. Front Immunol. 2024 Apr 23;15:1336599. doi: 10.3389/fimmu.2024.1336599. eCollection 2024.

Drugs

Evaluation	Drug	Modality	Dose	Route
Background	Bortezomib	Small molecule	—	—
Background	Methotrexate	Small molecule	—	—
Background	Rituximab	Monoclonal antibody	—	—
Background	intravenous immune globulin	Protein / enzyme biologic	—	—

Immune Modulation Therapy for Pompe Disease

Summary

Timeline

Publications

Drugs

Indications