Trial Entry Criteria

Rallying towards a better future for Pompe Disease

Who can participate in the study?

You may be able to join the Stride study if you meet these criteria:

  • 18 to 75 years of age
  • Diagnosis of Pompe disease based on documented deficiency of acid α-glucosidase (GAA) activity and a documented GAA mutation
  • Currently receiving standard-of-care Enzyme Replacement Therapy (ERT) (alglucosidase alfa) at a dose of 20 mg/kg every 2 weeks
  • Has been on ERT for the past 2 years or more
  • Able to perform pulmonary testing and muscle function testing in a seated position
  • Able to walk at least 100 meters in a 6-minute walk test

Up to 124 patients will be enrolled in the study for approximately 9 months and will attend approximately 6-9 clinic visits. Information gained in this study may be used in the design and conduct of future studies in Late Onset Pompe Disease (LOPD) patients. Patients enrolled in the study will be considered for enrollment in a registrational study evaluating an Advanced and Targeted GAA (AT-GAA), also known as ATB200/AT2221.1 To be enrolled in the registrational study, patients must have completed the current Stride study and meet all eligibility criteria for the planned study.

 

  1. AT-GAA is an investigational treatment paradigm that consists of a ATB200, a recombinant human acid alpha-glucosidase (rhGAA) enzyme with optimized carbohydrate structures, particularly mannose-6 phosphate (M6P), designed to enhance uptake, co-administered with AT2221, a pharmacological chaperone