What is the PROPEL study?

Pompe study is a randomized, double-blinded, multicenter global study designed to assess the efficacy and safety of an investigational co-administration treatment, AT-GAA1 in adults diagnosed with Late Onset Pompe Disease (LOPD) who have either received Enzyme Replacement Therapy (ERT) with alglucosidase alfa (i.e., ERT-experienced) or who have never received ERT (i.e., ERT–naïve), when compared with alglucosidase alfa / placebo.

To learn more about the PROPEL Pompe study, please visit: https://clinicaltrials.gov/ct2/show/study/NCT03729362

Clinical Trial Overview

To assess the efficacy of AT-GAA co-administration on ambulatory function, as measured by the 6-Minute Walk Test (6MWT), compared with alglucosidase alfa/placebo. The study will consist of a screening period up to 30 days, a 12-month treatment period, and a 30-day safety follow-up period. Participants who complete this study will have the option to participate in an open-label extension study to receive AT-GAA under a separate protocol.

Participant Criteria

You may be able to join the PROPEL study if you are an adult and weigh ≥ 50 kg at screening. Volunteers must have a diagnosis of LOPD based on one of the following: deficiency of acid alpha-glucosidase (GAA) enzyme, GAA genotyping, or muscle biopsy.  Other criteria may apply.

Study Locations

The PROPEL Study is being conducted at sites in the United States and 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 trial location/site information.

The PROPEL Study is designed to assess the efficacy of an investigational co-administration treatment, AT-GAA2, on ambulatory function, as measured by the 6MWT, compared with alglucosidase alfa/placebo.

Amicus Therapeutics is pursuing the development of AT-GAA (also known as ATB200/AT2221) for Pompe Disease.1 ATB200 is an investigational proprietary ERT administered by IV infusion, and AT2221 is an investigational 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.
  2. The safety and efficacy of the agents and/or uses under investigation have not been established. There is no guarantee that AT-GAA will receive health authority approval or become commercially available in any country for the uses being investigated.