The Stride study is designed to collect information about patients with late-onset Pompe (LOPD) disease who are already treated with enzyme replacement therapy (ERT).
Data will be gathered from study participants to evaluate their condition over time and changes related to the therapy. This includes questionnaires about health, lung function, movement, muscle strength, and chemical processes in the body. There will be no investigational drug used in this study. However, the information we collect in this study may be used to design and conduct future late-onset Pompe disease studies with new therapeutic agents.
What should study participants expect?
If you are eligible and decide to join the Stride study, you first have to sign an informed consent, which provides more details about the study. You will continue your current treatment of ERT. At 6-9 study visits that take place over approximately 9 months, you will participate in a variety of tests and evaluations. Some of these will include:
- Muscle function and strength tests
- Lung function test
- Questionnaires about your health
Blood and urine samples will be collected at each visit. The study team will monitor your health and discuss evaluations and lab tests with you throughout the study.
How may participants benefit from the study?
If you participate in this study, you will receive neuromuscular evaluations and lab evaluations at no cost. Patients enrolled in this study will be considered for enrollment in a registrational study evaluating an Advanced and Targeted GAA (AT-GAA), also known as ATB200/AT2221. 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 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.