What is the Stride study?

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).

To learn more about the Stride Pompe study, please visit: http://clinicaltrials.gov/ct2/show/NCT03347253

Clinical Trial Overview

Data will be gathered from study participants to evaluate their condition over time and changes related to the therapy, including questionnaires about health, lung function, movement, muscle strength, and chemical processes in the body. There will be no investigational drug used in this study, though information collected may be used to design and conduct future late-onset Pompe disease studies with new therapeutic agents.

Participant Criteria

You may be able to join the Stride study if you are between 18 and 75 years of age, you are currently receiving standard-of-care ERT, and you have been on ERT for the past two years or longer (other criteria will apply).

Study Locations

The STRIDE Study is being conducted at sites in the United States in countries around the world. In the United States, the study will be conducted in various states from coast-to-coast. Additional locations/sites are continually being explored. Check back periodically for updated site information.

The STRIDE Study is a Prospective Non-interventional Study in Subjects with Late-onset Pompe Disease who are Currently Being Treated with Enzyme Replacement Therapy.

Amicus Therapeutics is pursuing the development of an Advanced and Targeted GAA (AT-GAA, also known as ATB200/AT2221) for Pompe Disease.1 ATB200 is an investigational proprietary ERT administered by IV infusion, and AT2221 is a pharmacological chaperone administered orally.

Pompe disease is an inherited lysosomal storage disorder that results from a deficiency in acid alpha-glucosidase (GAA) and is characterized by progressive accumulation of lysosomal glycogen primarily in cardiac and skeletal muscles. Enzyme replacement therapy (ERT) using recombinant human GAA (rhGAA) is the only approved treatment available for Pompe disease.

  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.